What I Learned as A Volunteer

On Sunday, December 9, 2012, I was awarded the Lola Hanzel Courageous Advocacy Award by the American Civil Liberties Union of Northern California (ACLU-NC) at their annual Bill of Rights Day Celebration. Below is my acceptance speech for the award.

I delivered the speech using text -to-speech software on my iPad. If you are interested in speech technology, you might be interested my blog post on that topic, entitled Having a

That's me.

That’s me.

Voice, Communicating and Somewhere in Between or the post entitled You Don’t Have to Talk Like Stephen Hawking.

 I have put in brackets explanatory information for folks who are not familiar with ACLU-NC.

When Micky Walsh [Chair of the ACLU-NC Board of Directors] emailed to tell me that I was to receive this award today, I was ACLUNCincredulous, and humbled. I know a lot of the people who have gotten this award in previous years, and I am honored to be included in this august company.

I embarked on my relationship with the ACLU when I volunteered for the New Jersey affiliate in 1975. Little did I know that the work I did then on prison reform was just the beginning of many years of working with the ACLU.

I won’t bore you with all of the volunteer jobs I’ve had at various ACLU affiliates and the National office. Though I think it’s important to point out, that the ACLU always had the wisdom not to appoint me to a finance committee. What I want to talk about is not how I helped the ACLU, but how I was helped by the many roles I had with ACLU over time, one of the two organizations that are closest to my heart.

My experience with the ACLU confirms that volunteering is its own reward. I’m sure that my work with the ACLU — as a board member, a legal intern, a member of the Legal

Lend A Hand When You Can

Lend A Hand When You Can

Committee, a member of the board nominating committee, a representative to the Biennial Conference, a member of the National board, a fundraiser — was of value to the organization. At the same time, I learned so much — about civil liberties, about justice, about organizing, about effective campaigns and about how great non-profits are run — and I met some lifelong friends. At the ACLU, I learned how to be an effective activist. And, much to my surprise, I developed skills that would allow me to be an effective staff leader of another organization.

I was in my third term on the ACLU N C board (not 3 consecutive terms, that would be a no no) [ACLU-NC policy requires board members to cycle off the board after 2 consecutive terms], when I was diagnosed with breast cancer. I was 42 years old. While I was in treatment, I resigned from the board, and decided to stop practicing law to look for a job in health advocacy.

I ended up as the Executive Director of Breast Cancer Action, a tiny organization trying to tell the truth about breast cancer. There were already a lot of organizational players BCA logomaking their voices heard with a pretty pink and deeply misleading story. I was the first full time employee of Breast Cancer Action –or as I referred to it, BCA — with no experience in running or building an organization except what I’d learned at the ACLU. It turned out that that was plenty.

I had learned at the ACLU that an important part of getting your organizational message out was an effective press strategy. And one of my good friends from the ACLU was Elaine Elinson, the mistress of [ACLU-NC] press relations — I think she had a slightly less grand official title. I asked Elaine, who had a connection to breast cancer, to join the Breast Cancer Action board of directors and guide me in a media strategy. That may have been the smartest thing I ever did at BCA.

It was also at the the ACLU that I learned about board term limits as a way of balancing board members knowledgeable about organizational history with new people with fresh  energy and ideas. We modeled Breast Cancer Action’s board term limits on those of ACLU-NC.

I also thought, based on my experience with the ACLU, that having written policy statements was important as a guide for people working for the organization either as staff or volunteers, and as educational tools for others. So I worked with the board to write policies on topics on which Breast Cancer Action was involved.

But the biggest thing I brought to BCA was a social justice perspective that had been honed through my work with the ACLU. It’s a rare issue that can be successfully addressed without understanding the context in which it occurs. But there were no breast cancer organizations operating at the national level who addressed breast cancer through a social justice lens. It was fine to talk about new treatments, but we also needed to focus attention on who could get them, and how much that depended  on patients being able to find out about them, on the doctors they saw or the clinics they went to, and having  money to pay for them.

And the information people got about treatments was often prepared by the drug manufacturer, focusing on the benefits and downplaying the risks.

The talk about differences in breast cancer incidence and mortality among different racial groups is always labeled as a focus on disparities. But disparity just means difference. BCA called these differences inequities, and raised questions about the social, cultural, physical, and economic realities of different racial groups that go a long way to explaining the incidence and mortality differences. Only by addressing inequities can we hope to minimize the differences.

When places like Marin County got attention about its high breast cancer rates, Breast Cancer Action would point out that there were a lot of black women dying at young ages in Bay View Hunters Point that needed at least as much attention.

On the subject of environmental links to breast cancer, BCA called for studying the usually poor communities that are often situated near pollution sources and therefore at highest risk.

When it came to programs providing mammography screening for poor women, Breast Cancer Action took the position that if the government was going to pay for breast  screening for poor women, then women diagnosed with the disease should also have their treatment paid for by the government. Remarkably, that was not the law. It is now.

And there were areas where, as the leader of Breast Cancer Action I took a different course from the one that the ACLU national organization had adopted when I was on the board. I had been in the dissenting minority at the national board when I and others urged the organization to endorse limiting corporate contributions in elections. This affiliate [ACLU-NC] endorses these limits. The area of corporate influence in cancer advocacy isn’t about elections so much as it is about the reality or the perception that corporations that make drugs and devices for cancer use donations to influence the advocacy that cancer organizations do around treatment issues.

Breast Cancer Action was the first cancer advocacy organization to make it a matter of policy not to accept funding from corporations profiting from cancer or contributing to cancer by environmental harm.

It was the corporate contributions policy that in many ways enabled BCA to have, in a small way, an impact on breast cancer advocacy similar to that of the ACLU on a wide range of civil liberties issues. We launched our Think Before You Pink campaign in 2002, TB4UPraising questions about all the products sold with pink ribbons on them. We called for more transparency in these sales efforts. After all, if shopping could cure breast cancer, shouldn’t it be cured by now.? We also called out “pink washers,” companies that sold some product to raise money for breast cancer while at the same time making products that were likely contributing to the breast cancer epidemic. There’s now a documentary film about the pinking of breast cancer and pink washers. It’s called Pink Ribbons, Incorporated, and you saw a clip from it here  this afternoon. The film is available on DVD and Netflix.

There’s one more area of social justice where Breast Cancer Action’s goals overlapped completely with those of the ACLU. That issue is the patenting of human genes, in this case genesthe breast cancer genes known as BRCA1 and 2. BCA had tried unsuccessfully to get someone in Congress to address this issue when the patents on these two human genes were first issued. When the ACLU started examining the issue, they contacted us, and when the lawsuit was prepared to challenge the patents, Breast Cancer Action was the only national organization to sign on as a plaintiff. We could do that because we didn’t accept funding from the patent holder, Myriad Genetics.

Sooner or later, all issues of social justice are connected. And we as individuals can  advance the arc of history towards justice by volunteering. The world changes because we work for change. I am deeply grateful for the privilege of volunteering for the ACLU, and very honored to be the recipient of this Lola Hanzel Courageous Advocacy Award.

© Barbara A. Brenner 2012

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One More Time With Feeling: Can We Be Done With Mammography Screening?

October was long over when the New York Times ran an op-ed about mammography screening and over-diagnosis. The op-ed makes a compelling case for ending

Why are all these doctors here?

population-based mammography screening for breast cancer, based on the on the numbers of women who will be treated unnecessarily as a result.

 

This happens because screening mammograms: that is, mammograms given when there is no evidence of a breast lump or any other problem in the breast find cancers that, untreated, will never be life-threatening. In the medical field, this is referred to as over- diagnosis. Women experience it as unnecessary treatment.

Neither the op-ed nor I is talking about the need for diagnostic mammograms, mammograms given when a woman has a lump or another breast condition that needs to be figured  out.

I wrote briefly on this topic a year ago in blog called Is October Over Yet? at. In that post, I talk about Komen’s responsibility for push mammography screening, and their refusal to educate people on the risks of screening.

The New York Times op-ed prompted the usually number of outraged letters from members of what I call “A Mammogram Saved My Life” chorus. No one who has undergone surgery, and possibly radiation and drug treatment or chemotherapy wants to believe they didn’t need treatment.

As I explained in the film Pink Ribbons, Inc.at the time of a breast cancer diagnosis, women fall into one of the three categories:

— They have a kind of cancer that, left untreated, will never be life threatening. If they are treated, the treatments will very likely make them feel bad. Cancer won’t.

— They have a kind of cancer that can be effectively treated with currently available treatments. If they get treatment in a timely manner, the treatment may make them feel bad for a while, but it will keep them from dying of breast cancer.

— They have a kind of cancer that is so aggressive, no matter how small the tumor is, that no currently available treatment can stop it. They will be made very sick by the treatments, seriously affecting the quality of their remaining lives.

Of course, everyone diagnosed who is treated wants to believe that they are in the second group. If their cancer comes back after treatment, they will learn that they were in the third group. But people in the first group will never learn that they are in that group: they will continue to believe that are in the second group.

Folks, these realities are about biology, not mammography. Mammograms have always had their problems: they are radiation based, they miss things that might be life threatening and find things that aren’t. We have been promised a better technology for more than 20 years, and we’re not close. What we need are diagnostic tools that can tell us whether a cancer needs treatment. And we need to reduce the amount of over-treatment while not jeopardizing the availability of treatment for cancers that can and should be treated.

We should stop doing population-based mammography screening and focus on screening those at highest risk of breast cancer.

What we know now is that mammograms alone never save a life. If a life is saved, it’s saved by treatment, no matter how the cancer is found. The chorus notwithstanding and Komen are damned.

© Barbara A. Brenner 2012

 

 

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Sitting Down to Call Out Stand Up To Cancer (SU2C)

I’m a baseball fan. And a San Francisco Giants fan. I watched a lot of baseball in the post-season. (If you’re wondering, Giants won the World Series, sweeping the Detroit Tigers in 4 straight games. But that’s not really the subject of this blog.) During many of those games, viewers were pummeled with ads for Stand Up to Cancer (SU2C), an effort to raise money for cancer research. One night during the game, MasterCard presented a check to SU2C for $4 million, and many fans and even the game’s announcers held up SU2C signs showing names of people with cancer on them. Mine might have been the only dry eyes in the country when the presentation was over. Here’s why.

The Origin of SU2C

The story as I first heard it about how SU2C came to be has disappeared from the historical annals, replaced by the “official” version. Here’s what I heard at the time. Seems someone high up in the television business had a wife with cancer. He wondered to himself why there wasn’t a big effort to connect highly-watched TV to the cancer cause. He knew, if I remember correctly, the baseball commissioner, who agreed that the TV guy was onto something. Baseball endorsed the effort. And, since the TV guy had access to a lot of TV stars, SU2C was born.

The effort is a charitable program of the Entertainment Industry Foundation (EIF),

An industry it certainly is.

established by media, entertainment and philanthropic leaders who have been affected by cancer. The EIF describes itself as Hollywood’s leading charity. They describe their mission as “to fund ground breaking cancer research through innovation and collaboration, and to accelerate translational research to new target treatments to patients sooner.” Founded May, 2008, SU2C has so far has raised $180 million, $100 million that with its first telethon.

SU2C is so bold as to say of itself: This is where the end of cancer begins.

Really?

Where the Money Goes

Of course, EIF isn’t qualified to do cancer research, let alone innovative and collaborative research that will translate quickly in new targeted treatments of patients. So they subbed that task out. To whom, you might wonder, since nothing in the publicity about SU2C tells you.

The organization that gets these gobs of money is called AACR — American Association for Cancer Research. Unless you’re in the cancer field, you’ve probably never heard of them because, until now, they didn’t do any glitzy public campaigns.

Promising cures for over a century. What’s to brag about?

AACR was founded in 1907. (That’s the beginning of the last century. You would be perfectly justified in wondering what is taking so long.) Its mission is “to prevent and cure cancer through research, education, communication and collaboration.”

They describe what they do with the SU2C funds as giving “cutting edge” grants to “great scientists” doing “innovative high-risk hi-reward research.”

Same Old, Same Old

Of course, most cancer research organizations (and there are tons of them) make the very same claims that AACR and EIF do about the kind of research they fund. They will cure cancer. Where have we heard that before?  The proof should be in the outcomes of the research. What new discoveries have been made by AACR/EIF-funded scientists that are benefiting patients today with targeted therapies?  Answer: None, zero, zip.

And I don’t think that is only a matter of time. As I understand AACR, their focus is basic cancer research, not translational research. They fund science that looks at cells and sub-parts of cells, and sub-parts of the sub-parts of cells ad nauseum, not patients. AACR is mired in the reductionism that has characterized cancer research for decades, and produced very little of use to people with cancer, relative to the money and time invested.

As for MasterCard, why do you think they spent $10 million to advertise their $4 million

Goodwill goes a long way.

donation to SU2C? Cynical me thinks they were mostly in for the corporate good will.

Don’t be Fooled

Stand Up to Cancer is a highly successful effort to pull at people’s heartstrings and purse strings to get them to contribute to “life saving” cancer research. If you rearrange the letters, you can think of it as a campaign 2 SUC(K) off of peoples’ fear of cancer.

With billions being spent on cancer research, it’s time to re-think how we do cancer research. If money could solve the cancer the problem, it should be solved already. But it can’t, and it hasn’t been. It’s way past time for a new  approach.

© Barbara A. Brenner 2012

Posted in Breast Cancer, Medical Science | Tagged , , , , , | 10 Comments

NBCC — The Promise, the Process and the Problems

I have a reputation as an expert on the breast cancer and breast cancer advocacy organizations, gained during my 15 years as the (now former) Executive Director of Breast Cancer Action. Since it’s once again October — Breast Cancer Industry Month — I thought I should use that expertise to talk about an organization that does a lot of good, but seems to be misguided in a couple of important ways. That organization calls itself the National

NBCC’s logo. A little arcane from my point of view. Not to be confused with the National Board of Certified Counselors.

Breast Cancer Coalition, or NBCC.

NBCC promotes nuanced messages about breast cancer, does not overhype treatment news, and encourages an evidence-base approach to treatments. It also encourages people to move beyond breast cancer “awareness,” which Lord knows we have enough of.

But, despite its name, NBCC does not represent all breast cancer organizations. No group does. It is the brainchild and under the close control of Fran Visco, its founding Executive Director and now President. Like many charismatic leaders, Fran surrounds herself with people and groups who agree with her, declining to confer or

Fran Visco in a happy moment.

collaborate with those who disagree or don’t sing her praises. Unfortunately, that limits NBCC’s effectiveness.

A Breast Cancer Deadline?

NBCC’s current focus is on a “deadline” to end breast cancer by January 1, 2020. When they field tested this campaign with some of the breast cancer researchers with whom they work, they were told in no uncertain terms that setting a deadline would be counter- productive to research. But set a deadline they did.

Of course, they are not the first to set a deadline for solving the breast cancer problem. Susan Love set one in the mid-90’s. For what happened to that see, Susan Love: Time to

Deadlines can be very troubling.

Think Before You Pink. And how many times have we been told by doctors and researchers  that, in the next 5 or 10 years, the problem will be solved?

One problem with deadlines is that they assume that you have control over the problem you’ve identified. NBCC does not have that control. It’s an advocacy organization. It doesn’t do or fund research, though a lot of researchers work with them. NBCC also doesn’t control the legislative agenda for research, try as they might to do so. Under these circumstances, how might they expect to meet their deadline? They have a stated strategy, but it doesn’t seem to include working with other groups that know a thing or two on the topics being addresses. The NBCC strategy is: leave it to us.

I also should say that I think ending breast cancer is a promise that no one can keep. As I wrote in Breast Cancer Action’s Cancer Policy Perspective, breast cancer has been with us since before the Greeks. I think it will be with us always. The questions are (1) can the incidence be reduced and (2) can the treatments be improved?

What will happen when 1/1/20 rolls around and breast cancer persists? I assure that you NBCC will find some other mission to pursue that will allow them to continue to raise funds. Will they acknowledge failure? I doubt it. Barring a miracle, I won’t be around to witness that date, but I hope some people who read this will be here and will remember.

NBCC’s Legislative Strategy

NBCC has been active in lobbying Congress, and has had some success getting bills addressing breast cancer research passed. As far as I know, their efforts have focused on the Department of Defense (DOD) Breast Cancer Research Program, environmental breast

Congress: Where OUR representatives work

cancer research, and, most recently, an Act to Accelerate the End of Breast Cancer.

The DOD program — which drives funding through the DOD: it does not restrict its funding to the military — is NBCC’s longest-standing success. For background on the program and some of its challenges, see ERA of Hope or Hype?

What all these legislative efforts have in common is a commission of some sort to guide  the program efforts. The commissions, set up by the laws for which NBCC advocates, are composed of lay people and scientific experts. The participants are selected with NBCC approval.

These are efforts funded with taxpayer dollars. Why should NBCC and Fran Visco get to decide who the deciders are? Who elected them? And who will NBCC veto who is important

This guy said he was the decider, and we elected him.

to the discussions?

Time for New Leadership?

The competition among breast cancer organizations — for funding and for influence — is fierce. It leads to deadlines and promises that can’t be met and to steps that are more effective at achieving influence and dominance than at addressing the incidence and treatment of the disease. Witness the Komen Foundation, for example. It’s time for leaders who can bring people and organizations together. We need new approaches. In fact, I think the movement needs new leaders.

© Barbara A. Brenner 2012

 

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Who Shall Live and Who Shall Die? — A Yom Kippur Reflection

 

Introduction

This past week marked the end of what we Jews call the Days of Awe, the 10 days between Rosh Hashanah, the Jewish New Year, and Yom Kippur, the Day of Atonement, which is the holiest day in the Jewish Calendar. Susie, my beloved partner, and I attended services

The Shofar is blown on Rosh Hashanah and Yom Kippur

at the Mendocino Coast Jewish Community, where we have been members for many years.

This year, our friend and Rabbi Margaret Holub asked me to do a teaching on a prayer that we say at Yom Kippur, called the Unataneh Tokef. The prayer’s most famous lines, at least to Jews, are: “On Rosh Hashanah it is written; on Yom Kippur it is sealed, who shall live and who shall die . . .”

The Days for Awe are so named because it is during the time between Rosh Hashanah and Yom Kippur that Jews are called upon to examine their lives, their relationships with people, and their relationship with God. They are called to turn, through these examinations, towards God.

Below is the text of the talk I gave on Yom Kippur. I’ve decided that images will distract from the text. I promise to return to them next post.

Thoughts on Unataneh Tokef

Our High Holiday mohzor (prayer book) is filled with reminders — as if we needed to be reminded — that life is cyclical. As Emmy Lou Harris sings, we are all born to live; we are all bound to die.

Some of these reminders are relatively gentle. That cannot, however, be said of the best known part of Unataneh Tokef, a prayer that many of us know at least a part of by heart. That part is:  “On Rosh Hashanah it is written, on Yom Kippur it is sealed, how many will leave this world and how many shall be born into it, who will live and who will die . . . .”

After a long litany of the many ways we might die, we are told that, while we cannot change the decree, tshuvah (turning), tefillah (study) and tzedakah (charity) will make our fate easier.

If tshuvah, tefillah and tzedakah could reverse the decree, I suspect the world would already be filled with many more devout and very old Jews.

I think it’s odd that this prayer is a central part of the Yom Kippur liturgy, because by the time it rolls around, it’s time for the ledger to be sealed and may be too late to do anything about it. At the same time, I don’t find any indication in the mohzor that what is written on Rosh Hashanah ever changes by the time Yom Kippur arrives. Nonetheless, like others more steeped in our tradition than I am, I have been struggling to understand what this prayer means, how to take it in.

I wonder if anyone still believes that God actually has a ledger book in which all our names appear, and that God makes an entry each year for each of us. But even if we don’t believe this, this prayer captures our attention and imagination, prompting us to wonder and pray that we end up on the living side of the ledger on Yom Kippur.

Because of this hope, it is during these days of awe that we ask ourselves questions about how we live our lives, ask whether our lives have purpose and meaning. Yom Kippur drives us to examine our lives, not because we’re necessarily going to die in the next year, but because doing so may result in tshuvah — turning towards God. Since we can’t know (not even I know despite my illness) whether we are written and sealed for another year, we strive to turn towards God so that, whenever our time comes, we have done our best to lead meaningful lives.

In a perfect reflection of Judaism, I think that what’s important in the Days of Awe are the questions we ask ourselves, not necessarily the answers to those questions. Because questions prod us to examine our selves and our lives deeply. The questions aren’t just for the Days of Awe; they are for everyday.

It’s not about getting to heaven, especially if you don’t believe there is one. It’s about examining ourselves to be sure we are living our lives to the fullest, with purpose and meaning. I think this is part of what Margaret was talking about in her drosh on Erev Rosh Hashanah (the evening service of Rosh Hashanah). While being inscribed in the book of life is a thing to pray for, it’s how we live the lives we are given  — however short or long — that indicates how we incorporate tshuvah, tefillah and tzedakah.

The singer song writer Keven Welch, who I doubt is Jewish, has lyrics that go like this: There’ll be two dates on your tombstone. And all your friends will read ‘em. But all that’s gonna matter is that little dash between ‘em.

The poet Mary Oliver, in a poem called The Summer Day, expresses it a little differently:

Tell me, what is it you plan to do

With your one wild and precious life.

The point of personal, self-reflective questions is to focus us on how we live now and how we need to change. If we do this, it does not matter a lot whether we’re not inscribed this year or next or ten years from now. Because we all die. The question is: are the lives we’re leading ones of connection, contemplation and good deeds?

The poet Rilke offers this: Have patience with everything that is unresolved in your heart. And try to love the questions themselves. Don’t search for answers which couldn’t be given to you now, because you wouldn’t be able to live them. In poetic terms,

And the point is to live everything.

Live the questions now.

Perhaps, then, someday far in the future,

You will gradually, even without noticing it,

Live your way into the answer.

Rilke also offers this poem, which I think speaks to how we live our lives in relationship to God.

 

God speaks to each of us as he makes us,

then walks with us silently out of the night.

 

These are words we dimly hear:

 

You, sent out beyond your recall,

go to the limits of your longing.

Embody me.

 

Flare up like flame

and make big shadows I can move in.

 

Let everything happen to you: beauty and terror.

Just keep going.  No feeling is final.

Don’t let yourself lose me.

 

Nearby is the country they call life.

You will know it by its seriousness

Give me your hand.

In this day and age, I think Unataneh Tokef’s  true significance is not whether God in fact sits in judgment of each of us, but whether we believe that, in some meaningful way, our lives depend on our power to change, to take God’s hand, to engage in tshuvah, tefillah and tzedakah. These things make it easier to bear what God may decree. I think they do so by helping us to live lives of learning, connection and good deeds that benefit the communities in which we live. That brings us closer to God in all of God’s many manifestations.

Since we cannot know where our names appear in the book, maybe the purpose of acknowledging that on Rosh Hashanah it is written and on Yom Kippur it is sealed is to remind us that, whenever and however we die, our obligation is to notice — ourselves, our relationships with others, the good we do in the world. We are finite, but we transcend death by the way we live our lives and connect with each other and with God. The good we do lives on through the lives we’ve touched.

Life, death, and birth are mysteries that are in God’s hands. We cannot control them. But we can control our attitude towards them. I don’t think my having ALS is a way that God is punishing me. We’re all going to die of something. My challenge is to live whatever life I have left in tshuvah, tefillah and tzedakah. I think that is the challenge for all of us, even those of us who are perfectly healthy today.

As I explained to someone else with ALS who was asking why I call myself a “practicing Jew,” I see myself as constantly striving toward a meaningful life and, through that life, a relationship with God. It’s a practice. Some people may get it perfectly right, but I think most of us — myself included — keep working to achieve tshuvah. I think that is the message of Unataneh Tokef.

© Barbara A. Brenner 2012

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Not So Funny Things Happened on the Way to a Diaphragm Pacer: When “Informed” is Not “Consent” and Related Adventures of an ALS Activist

Regular readers of this blog have read a lot about my adventures trying to get Biogen Idec, the makers of dexipramipexole (dex), to let me get a diaphragm pacer without being dropped out of the dex trial in which I’ve been participating for the last 15 months. When the company proved intransigent, I turned my efforts and readers’ considerable energies to the trying to get the FDA to “do the right thing.” This blog post updates those efforts, and, for me, brings to close, at least for now, this chapter of my ALS activism.

What the FDA Says Now: A Chance to Make Changes?

A number of people at the FDA actually read my blog, and I guess the title of the last one (FDA to ALS Patients: Fuck You), as well as the comments posted to it, got their attention. On September 6, I got the following message from the Deputy Director of the Division of Drug Information for FDA’s Center for Drug Evaluation and Research (CDER):

Dear Barbara,

The FDA would like to assure you that we are taking your concerns seriously.                  The issues that you have raised have been under discussion internally, and the                     Agency is considering your arguments about how to design and conduct trials in                  ALS that put the patient first.

Additionally, we would like to let you know that the Agency is in the process of                planning a public meeting that will address your general concerns as well as the                 more specific concerns you and others have raised, such as an independent                         patient ombudsman. Once a meeting date has been set, I would be happy to               provide you with the details.

             The passion for ALS treatment that you and the readers of your blog have is                    obvious from your posts and the comments you receive. Please know that                      patients’ perspectives are an integral part of the drug approval  process, and     we value and appreciate these comments.

              I understand that the scheduled date for your pacer implant surgery is quickly                   approaching. I want to extend our best wishes to you for a successful surgery.              Please email me afterwards to let me know how you are doing.                                       Best regards,

Catherine Y. Chew, PharmD

Deputy Director, Division of Drug Information

Center for Drug Evaluation and Research

Food and Drug Administration

While this doesn’t help in my current circumstances (see below: Treatment Delayed is Treatment Denied), the prospect of a public meeting at which people affected by ALS will have an opportunity to influence the future structure of ALS trials is heartening.

I hope everyone concerned about these issues — and everyone should be — will contact the FDA to be sure they get notice of when this meeting will take place. Normally, the FDA accepts written comments as well as in-person testimony, and I have asked if that will be the case with this meeting. Write to DRUGINFO@fda.hhs.gov.

Activism can work. Try it. You’ll see.

The IRB Should Be Called the CYA

In the earlier response from the FDA, which I described as “fuck you,” it was suggested that I approach the IRB (note: that stands for “Institutional Review Board,” not “Independent Review Board”) at CPMC, where the Forbes Norris clinic is based. I did so.

This is a covered ass.

The basic position of the IRB was that, since the research folks had followed all the informed consent procedures, they did not need to take action. The ethical issue of holding me — and others with ALS — hostage to irrational rules was not considered.

Interestingly, CPMC has a medical ethicist on staff, but that person said the ethical issue in this setting was for the IRB to address. Evidently, the IRB didn’t think so.

The response from the IRB, and my contact with other ALS folks about this topic, led me back to look at the informed consent I signed. There was not one word in it about the diaphragm pacer. The rules of the trial changed in the middle. I explained this to the IRB, and told them that, while I know I had signed one or two consent forms after the first one, no one had mentioned to me whether subsequent forms precluded a diaphragm pacer.

The IRB thought this might be problem, so they asked the ALS research team for documentation of what I supposedly knew. (Nothing like asking the folks who may have dropped the ball whether they did or not, eh?) After reviewing what the ALS research team provided, the IRB again turned me down. They acknowledged that I had never signed a consent form precluding the pacer, but since I had acknowledged receiving a email from Forbes Norris informing me of the pacer limitation, I had no basis to complain.

Before I get to my rant on “informed consent,” I should say this about my email communication with Forbes Norris about the diaphragm pacer. I did get an email from the research coordinator saying that a pacer would bounce me from the trial. In response, I told the coordinator that I would spare her my views of the ruling since she had already heard my rant about how irrational that was. Consent?

Informed” v. “Consent”

I deeply appreciate all the people who stood up some years ago to say that people in trials need to understand for what they are signing up. There was a time when people got no information. (A terrific book about one aspect of this is The Immortal Life of Henrietta Lacks by Rebecca Skloot.) Unfortunately, the noble effort to change that reality has turned into another sort of nightmare for patients.

Is there really such a thing anymore?

Informed consent documents for trials, if the Biogen forms are any indication, now run 20 to 30 pages long. While they included information about the trial and the risks and benefits, they also include things like the follow up schedule, the information sharing  rules, and a myriad of other things. The notion that being informed amounts to consenting to all the things these forms contain is folly.

And consider the patient’s option. S/he can sign the consent form as written, or not join the trial. There is no give and take in the process of “informed consent.” So is it really consent?

Add to these problems what happens when drug company sponsor like Biogen Idec  decides to change the trial rules after the trial has started. Patients are then “re-consented,” asked to sign a new altered consent form. Again, they can sign or be dropped from the trial. The drug company holds all the cards.

And, evidently, the company can play those cards without even getting a signed  consent document. An email stating the change in trial rules evidently binds the patients.

It is a misnomer to call this process “informed consent.” The law has name for it: unilateral contract.

Treatment Delayed is Treatment Denied — No Diaphragm  Pacer for Me

While all this effort to try to stay in the dex trial if I got a diaphragm pacer was going on, I was trying to get set up to get a pacer implanted.

Here are a few dates to consider as you read about this. The pacer was approved by the FDA in late September, 2011. On October 11, 2011, I let the clinic know I was interested in getting a pacer.

I had been in discussions with my neurologist at Forbes Norris since early April about getting the tests I need to establish my eligibility. I had respiratory tests at the clinic in February, 2012, which I passed. On April 30,  2012, after arguing with my neurologist about which other tests to get, I had a SNIFF test (a flouroscopic view of my diaphragm). I had  to argue with my neurologist to get that test.

I heard nothing about pacers from the clinic again until early June, 2012, when I was asked the clinic folks if I was willing to be the first person at Forbes Norris to get a pacer. I said yes.

I assumed — incorrectly — that things  would move along with scheduling the surgery.

The only hold up I heard about  was in June,  2012. MediCare approval had not yet been confirmed. For some reason, the clinic was unwilling to start scheduling the surgery until coverage was confirmed, even though it involved several doctors with busy schedules. MediCare coverage was obtained in late July, 2012. At that point, the earliest available date for surgery was September 12.

ALS, as the neurologists certainly know, is a progressive disease that effects respiratory capacity. Since there is  no effective treatment, the respiratory numbers go only in one direction: down. Yet the doctors at Forbes Norris did nothing that I know of to expedite getting me a diaphragm pacer while my respiratory numbers were good enough to be eligible.

By the time I got to the clinic on September 10 and had my respiration checked again,  my function had dropped below the eligible criteria. So, I cannot get the pacer.

In ALS, as in many diseases, treatment delayed is treatment denied. The people at Forbes Norris know this. I would love to see them act like it mattered.

© Barbara A, Brenner 2012

Posted in ALS, ALS Treatment, Health Policy | Tagged , , , , , , , , | 18 Comments

FDA to ALS Patients: Fuck You

If you like this blog, please pass it on to your friends. Let’s keep the conversation going.

I will spare my readers the long saga of my fight with Biogen Idec about the dex trial and my plans to get a diaphragm pacer. If you want all the background, you can read it in three installments: here, here, and here.

When the company refused to budge, I appealed to the FDA Ombudsman both for help with my situation and to assure that every large trial had an ombudsman to resolve disputes between trial participants and the drug company running the trial.

Great friend to drug companies everywhere.

The FDA has spoken. In short they turned me down flat on both requests — apparently without seriously considering my arguments or the letters that many of you sent on my behalf. Here’s what the FDA said.

****************************************************************************************************

Dear Barbara,

Thank you for writing to the Food and Drug Administration (FDA). This is in response to your email to Dr. Janet Woodcock, Dr. Russell Katz, and Ms. Virginia Behr concerning access to dexpramipexole. The issues you raised have been carefully considered, and I’ve been asked to explain our thinking to you.

We understand that Amyotrophic lateral sclerosis (ALS) is a very serious disease which deeply affects patients and their loved ones. We appreciate you sharing your concerns

No thanks to the FDA

about your possible lack of continued access to the study drug, dexpramipexole, if you begin using a diaphragm pacer before Biogen Idec’s EMPOWER trial ends in September.

FDA works with drug developers like Biogen Idec to ensure that patients volunteering to take part in studies are treated fairly and ethically. We understand your argument for being allowed to start use of a diaphragm pacer while staying in the dexpramipexole trial. We also understand Biogen Idec’s argument that making exceptions to the rules of the study would jeopardize the ability of the study to show if dexpramipexole is effective. We wish we had a way to resolve dilemmas like this, but we simply do not. We are acutely aware of the immediate need for effective treatments for ALS, but the only way we know how to learn if a drug is effective is to require sponsors to conduct studies with rules about the types of treatments patients receive, including, of course, rules that assign one group of patients to the experimental drug and a comparison group to placebo for some set period of time.  The rules for these studies must be consistent with established ethical and scientific principles, but, within these standards, the specific elements are generally chosen by the sponsors for reasons that are specific to the particular study.

In response to your request for an ombudsman and independent review of each clinical trial, please be aware that in addition to FDA’s responsibility to ensure that clinical trials are conducted ethically, clinical trial procedures are reviewed by institutional review boards (IRBs) which essentially act as an independent ethics committee for clinical trials. These boards are composed of at least five members who include scientists, doctors, and lay people, and they must approve every clinical trial taking place within their jurisdiction- generally a hospital or clinic.

We wish you the best with your continued treatment.

Best regards,

Catherine Y. Chew, PharmD

Deputy Director, Division of Drug Information

Center for Drug Evaluation and Research

Food and Drug Administration

This communication is consistent with 21CFR10.85(k) and constitutes an informal communication that represents our best judgment at this time but does not constitute an advisory opinion, does not necessarily represent the formal position of the FDA, and does not bind or otherwise obligate or commit the agency to the views expressed.

****************************************************************************************

Essentially, the message is patients have no recourse once the trial design has been approved, no matter what the circumstances are. As I understand it, the IRB’s to which the FDA refers consider issues before the trial begins, not after.

Here’s what I wrote back to Dr. Chew:

********************************************************************************************

Dear Dr.  Chew,

This is an outrageous response.

You take Biogen’s argument hook, line and sinker.

I guess I know now who the FDA works for.

I see nothing here that addresses the issue a subset analysis for participants who have been in the trial at least 12 months.

I’m now in a situation where, with this decision,  I will be forced out of the trial 11 days before it ends.

This decision makes ALS patients hostage to an irrational rule. If I am forced out of the trial by Biogen, I want to bar them from using any data gathered from me during the course of the trial. But I’m betting you will say that I cannot do that.

***************************************************************************

This would have been a shorter and clearer message from the FDA

Bottom line here is that ALS patients are screwed, first by their disease, and then by the companies and agencies who claim to want to help them. For shame.

 

© Barbara A. Brenner 2012

Posted in ALS, ALS Treatment, Health Policy | Tagged , , , , | 28 Comments

Fool Me Once, Shame on You. Fool Me Twice . . . : Nothing New at Komen

If you like this blog, pass it on to your friends. Let’s keep the conversation going.

Maybe you were wondering what I would have to say about the Chick-fil-A. It’s a good story, but not the subject of this blog.

Sorry, not writing about this.

Besides, since that story broke, the Susan G. Komen for the Cure Foundation has been in the news twice. The first time was a story about the dangers and misleading nature of Komen’s messages about screening mammography. The second time was a story about leadership changes at the Foundation. I want to comment briefly on the first story, and more extensively about the second, though they may well be related.

One More Time With Feeling:  Komen Sends the Wrong Messages on Mammography Screening

If you’ve seen Pink Ribbons, Inc., or you are a member of Breast Cancer Action, you will not be surprised to read here that Komen oversells mammography, to the detriment of women’s health. I’ve said it for years, but I’m not the only one saying it. Most recently, in an article in the British Medical Journal, two health professors pointed out in detail how Komen distorts statistics to encourage women to get mammograms, ignoring the realities

Not as a good a technology as Komen says it is

of what screening mammograms can and cannot do. In an article entitled “How A Charity Oversells Mammography,” the professors analyze a prominent Komen ad that reads “What’s the key to surviving breast cancer? You — get screened.” It’s a short article, but available on line only to British Medical Journal subscribers. If you search the internet the title of the article, you’ll find quite a few stories discussing it.

What’s remarkable is not the conclusion that Komen distorts statistics to overstate the benefits of mammography screening, while ignoring the risks. Like I said, I’ve been reading or saying things like it for many years. But Komen’s response is simply astonishing. Chandini Portteus, is Komen’s VP of research, evaluation and scientific programs (who, unlike many other VP’s has not resigned or been forced out over the Planned Parenthood mess). Ms. Portteus actually said the

Maybe Ms. Portteus knows something we don’t.

following to a t.v. reporter covering the story: “We have long advocated for women to be informed about the benefits and risks of early detection . . .”

I challenge anyone at Komen or anywhere else to point to anything that Komen has ever said, let alone promoted, that talks about risks from mammography screening. (I will send this blog to Ms. Portteus. Maybe she’ll be able to meet the challenge, but I doubt it.)

The public needs to have balanced information about screening so we can make informed choices about our care. What will it take to make sure they have it? It’s clear that an occasional news story won’t get us there, because there have been many stories over the years that haven’t done the trick. Komen won’t change its message (see below). So maybe it’s time for Komen to go out of business (following the lead of the  strangely named Y ME Breast Cancer Organization which recently did). On mammography (and several other things), Komen is doing more harm than good.

A PR Stunt Masquerading As A Leadership Change

My email was about to explode recently when the news hit of leadership changes announced by Komen. The President, Liz Thompson will soon be gone, and Nancy Brinker will step down as CEO as soon as they fill current leadership vacancies for President, Chief Executive Officer, and Chief Operating Officer. The Foundation’s statement announcing these changes neglects to mention the recent departures of the VP of Communications VP, VP for Policy, Executive VP and Chief Marketing Officer, VP of Global Networks, and Director of Affiliate Planning and Strategy.

That Brinker will not leave her CEO post until leadership vacancies have been filled is a

30 years at the helm at Komen. Time to move on, Nancy..

clear indication that she will be deciding who fills these positions. And, when she does step out of the CEO job, Brinker will continue on the Board and as chair of its Executive Committee. Her new role, as yet untitled, will be a “management” role focused on global growth, fundraising, and overall strategy. New title; same control.

For the story on what Nancy Brinker really wants and how she is the Komen Foundation to get it, see this story in New York Magazine

I sure wouldn’t want to be the CEO if someone else in the organization has that portfolio. And, as a breast cancer activist, I am horrified at the prospect of Brinker spreading Komen’s misleading messages (mammography screening is the tip of the ice berg) all over the world.

Nancy Brinker has done this before. Several years ago, she took a “step back” when she was given the Ambassadorship to Hungary in exchange for her fundraising for George W. Bush. She hired Hala Moddelmog to be CEO of Komen. But Brinker was still in charge, which she proved by firing  Moddelmog and returning to the CEO position herself when her Ambassadorship ended.

How About Some Real Change at Komen

What we seem to have here is founder syndrome run amok: a founder who did great work getting the organization going and growing, but cannot imagine how the organization could survive without her. So she surrounds herself with people who feed that myth, and ignores the reality that it’s time for new leadership. On the subject of leadership, see my previous post entitled Thoughts on Leadership.

Komen is always delighted to tell us how many billions of (your) dollars it has invested in breast cancer research. It would be nice to hear about the return on that massive investment, but, in the meantime, maybe Komen should spend some of that money on a consultant to help Nancy Brinker figure out her exit strategy. Someone has to do it. It sure won’t be her hand-picked board chair.

When is change not change: when it involves Nancy Brinker and the Komen Foundation. If you agree, tell Nancy Brinker so. Her email address is NBrinker@Komen.org

If you would like to join others calling on Brinker to step down — really down — from Komen now, you can do so at here.

© Barbara A. Brenner 2012

Posted in Breast Cancer, Health Policy | Tagged , , , , , , | 2 Comments

Broadening ALS Research to Help Patients III: Make Biogen Idec Walk Its Talk

Like many readers of this blog, I have been exchanging emails with Dr. George Scangos, CEO of Biogen Idec, the company running the clinical trial in which I am a participant. The trial is testing the efficacy of a drug called “dex” for short. (Thanks so much to all of you who wrote to him for your support.)

The upshot of our “conversation” is that company will not allow me to remain in the trial if

Because of a drug company, this is where I’m living

get a diaphragm pacer before the trial ends sometime in September. If I do so, they will exclude me from eligibility for dex if and when it becomes open label.

In the interest of transparency, my email exchange with Dr. Scangos is copied here. I have a few observations, and a new call to action for those who care about these issues.

EMPOWER?

The dex trial being run by Biogen Idec is called EMPOWER (in all caps, no less). Given how the company is interpreting the rules of this trial, I think there is little doubt that only the company and its shareholders will be empowered if this trial is a success. The power of ALS patients, including those who, like me, have given more than a year of time and effort (and considerable amount of bodily fluids) to the trial, seems to be way down the list.

My Crap Shoot

It’s possible that this trial will be a failure, in which case my concerns about access to dex if I’m forced out of the trial will be moot.

If the trial is positive, I may be in trouble, or I may not be. If I’m currently on placebo, the drug might help me. That’s trouble if I have to wait to get it.

If I’m currently on the drug, I may have gotten all the benefit possible from it already, in which case my being booted out of the trial won’t matter. It’s also possible, that, if I’m currently taking the drug, stopping it has adverse effects. Again, that’s trouble.

In the midst of this dilemma, I have to make some decisions about what is best for my health. Against these various outcomes, do I get a diaphragm pacer now in hopes that will provide me more benefit than the drug might if proves effective, or do I wait until the trial becomes open label — maybe an extra two months — so I can be eligible to the obtain the drug on open label if the trial is a success? If I do the latter, at what cost to my health from delaying the pacer?

The bigger question is why any patient with ALS should be faced with this kind of dilemma. Biogen Idec has the power to remove this question from the debate, but it refuses to do so.

What Biogen Says versus What It Does

As you will see from the emails below and others in previous posts on this topic, Biogen Idec says it is committed to finding effective treatments for ALS and getting them quickly into the hands of patients.

Biogen Idec, through its CEO, also says this: “It is an ethical consideration to allow patients who have participated in the trial to benefit from the drug, should it prove to be efficacious.”

And this: “If you decide to have the pacemaker and come off the trial, the data from your participation will be counted up until the time that you leave the trial. You still will have contributed to the trial and to the development of what we hope will be an important therapy for ALS patients.”

So here’s the deal Biogen Idec is “giving” me and patients like me: if we are booted out of the trial by seeking other treatments, the data from our participation (in my case, at least 14 months of data for a trial where minimum participation is 12 months) will be analyzed as part of the evaluation of drug efficacy. If the drug proves to be effective, however, we will not automatically receive it, we will have to plead for it. Others who decide to remain hostage to these trial rules will be automatically given it as part of trial design.

In other words, Biogen Idec will receive all the benefit it possibly can from our participation, but our benefit, if any, will be limited to the effects we may have received from the drug — if we were on it  — while we were included in the trial.

Time to Make Drug Trials Work for Patients

The FDA has a role in setting drug trial rules. Biogen Idec claims to be hampered in part by those rules. While I continue to believe that the company in this case is unnecessarily using these rules to the disadvantage of patients like me, they are not budging.

So it’s time to go to the FDA to demand change. If you agree that these trial rules, as interpreted, are unfair to ALS patients, let the FDA know it. Tell the FDA to make Biogen Idec do the right thing.

And suggest to the FDA that they require drug companies in large ALS trials to have an independent ombudsman to address patient concerns. The CEO of a company cares about the company; patients needs are secondary.

>The Director of the office of the Center for Drug Evaluation and Research is Dr. Janet Woodcock. Her email address is Janet.Woodcock@fda.hhs.gov

> The FDA Commissioner is Margaret Hamburg. Her email address is margaret.hamburg@fda.hhs.gov.

> Please send a copy of your message to the FDA to Dr. George Scangos at Biogen. His email address is: george.Scangos@biogenidec.com.

> And please send a copy of your message to the FDA to my neurologist, Dr. Jonathan Katz. His email address is katzjs@sutterhealth.org.

Please copy anything you send as comment to this post. And thanks for caring what happens to people.

©  Barbara A. Brenner 2012

************************************

Email exchange July 23, 2012

Dear Dr. Scangos,

Thanks for your message. See my reply below [in italics] in the interest of what I hope will be clear communications.

Barbara

Dear Ms. Brenner,

We would love to honor your request, but I am very sorry to say that we can not do so. The reasons are complicated, and perhaps Doug and I have not been clear in our explanations why, so I’m going to give it one more try.

1.  The trial protocol does not mandate that each patient stay on the trial for one year. The trial protocol requires that all patients stay on the trial until the last patient completes one year of treatment.  It is designed that way so that most patients will be on study for more than one year to satisfy the views of the regulatory agencies here and in Europe.  We can not change those rules in the middle of the trial.

BB: When you say “so that most patients will be on study for more than one year,” I cannot  believe the regulatory agencies where vague what constitutes “most patients.” Can you share that information with me (and those copied on this message), as well as  the number of patients  that have been one the study for more than a year and  for how long; and the total number of patients  in the study?

2.  The subgroup analyses that you suggest are not feasible for a range of statistical, medical, and regulatory reasons.

BB: I would like an explanation  of these reasons. But I think  it requires knowing  the information about the number of patients in the trial and how many have been  in the trial for more than a year, and for how long.

3.  The trial protocol does not allow the type of treatment that you are seeking.

BB: The  treatment I am seeking was not,  if my memory serves,  approved by the FDA when this trial  started. If I’m correct about this, it’s not that protocol  excludes the treatment; it  wasn’t considered one  way or another. And  why are you allowing  this treatment  on the open label phase of the study but not for patients  who have been  on the study more than a year.

4.  Exceptions to these rules jeopardize the power of the trial and thus the availability of Dexpramipexole to all patients with ALS if it is indeed shown to provide an acceptable risk/benefit profile.

Please believe me that we have no reason to want to deny your request.  We have devoted ourselves to trying to help patients with ALS and other neurodegenerative diseases and would love to grant your request and help you.  I have tried to respond personally to your email  and to the emails of your supporters rather than sending a rote response because of the obvious sincerity and depth of the requests.  However, I don’t want to stretch this issue out.  As heart-wrenching and difficult as it is, we must stand by our decision in the interest of all patients with ALS.

On a personal note, I want to you to know that my thoughts and prayers are with you.  We obviously don’t know each other, but you must be an extraordinary person to have such a large collection of people who care so deeply about you.  And just so you know, the reply I sent to your supporters was not canned, as you state on your blog.  I wrote it myself on Saturday trying to explain the reasoning for our decision.  I’m sorry if it appeared canned, but I was just trying to get the same information to all of the people who wrote.

BB: Thank you  for your thoughts  and prayers. What  I need is more thought by people like you as  to how to best meet the needs of ALS patients for whom time  is so short. 

I’m sorry for offending  you  by saying your response was canned. I’m should  have said “identical.”

********************************

Message sent July 24, 2012

Dear Dr. Scangos,

In thinking more about our exchange, I wonder if you perceive as I do that my situation, like the dex trial in general, has the capacity to provide information that will benefit all ALS patients? If others, like  me, spend many months in the trial, and then go one to take advantage of another therapy, what might that tell us about mutiple treatment modalities and their effectiveness? Since, in most health settings, this is how treatment works, I suspect and hope that, as more treatments become available for ALS, we will get more information like this of value of patients. Why not start here?

I was also wondering whether, if I’m forced out of the trial before the open label period by a decision to get a pacer, will the data the company has collected from my participation nonetheless be evaluated in the trial results?

Thank you.

Barbara

*********************************************************************************************

Reply received July 24, 2012

Hello Barbara,

You are right in saying that in most health settings, at least in the US, patients and physicians take advantage of multiple modes of treatment to maximize the benefit they receive.  There are many things about our health care system that can be improved, but that flexibility is one of the good aspects of our system.  In a clinical trial, the situation is different, of course.  The purpose of the trial is to determine if a particular treatment, in this case Dexpramipexole, is safe and provides a benefit to patients.  For that reason, the effect of the tested treatment has to be isolated from other treatments that affect outcome. The reason that you want the pacemaker is that there is a good chance that it will improve your breathing and make you feel and function better.  That potential improvement as a result of the pacemaker can mask the impact that Dex has.  If you are on the drug arm, it can make the drug look artificially good, and if you are on placebo, it can make the drug look less good than it actually is.  What we are trying to accomplish is a clear outcome that will accurately determine the safety and effectiveness of Dex, so that if it is positive, we can get it to the maximum number of patients in the shortest possible time.

If you decide to have the pacemaker and come off the trial, the data from your participation will be counted up until the time that you leave the trial.  You still will have contributed to the trial and to the development of what we hope will be an important therapy for ALS patients.

There is something else that you should know.  We obviously don’t know at this point if Dexpramipexole is working or not, but we will know later this year.  In the case where it does turn out to be positive, we want to be able to make it available to some ALS patients even before it is approved by the FDA.  We are making enough of the drug to be able to do so, and we are having discussions with the FDA about how to ethically and legally conduct such a program.  Although our plans are not yet finalized, we probably will seek to make the drug available through centers that participated in the clinical trial.  Since you are obviously being treated at one of those centers, should you come off the trial, the drug may still be available to you through that program.  My best guess is that the program will be available early next year.  Of course if the outcome of the trial is not positive, then this is all moot.

I talked today with the head of the Massachusetts Chapter of the ALS Association and with an ALS patient here in Boston.  We had them in to talk with our employees at lunchtime as part of our ongoing program to make sure that all of our employees understand what is at stake.  I hope that for their sake, for yours, and for all patients with ALS that our trial is successful and that we are able to offer at least some relief to patients.

Sincerely,

George Scangos

***************************************************************************************

My reply to the above message, July 24

Hello, George,

Thanks  for this.

I understand  the difference between general health settings and clinical trials.

I appreciate that if I get a pacer and am forced out of trial, you will still count the data received from me up until that point and that you recognize that my participation will have contributed to the knowledge about the effectiveness of dex.

What I’m still puzzled by is the timing issue and why it makes so much difference. If I get the pacer in the next month, before the September trial cut off, that means you will have one  less month of data for me for the trial because you don’t want to run  the risk that, in month’s time, the pacer could mask the benefits of the drug. But, after September, when I assume you will continue to follow patients after the drug goes to open label, you’re not concerned about that masking effect because you will allow patients in the trial to then have pacers. Why does it matter in August, but not September?

You have addressed a lot of my concerns here. I now understand that your goal is to make the drug, if it shows benefit, available to centers that participated in the trial, including Forbes Norris, hopefully by early next year. If I were to remain the trial, would I have access to the drug sooner?

Thank you.

******************************************************************

Scagnos message July 25, 2012

Hello Barbara,

The September date is important from a regulatory standpoint.  The trial officially ends one year after the last patient was enrolled, which as you know is in September.  At that point, the data are “locked” and analyzed, and will comprise the package submitted to the FDA and European authorities for approval.  Patients on the trial will then all be switched to drug.  Patients will continue to be followed to gather longer-term data on safety and efficacy, but although the data gathered beyond September are supportive (or not) of approval, they are not included in the primary analyses comparing drug to placebo. That’s the reason why August is a problem and September is not.

Since you are familiar with breast cancer, you may recognize this trial design.  This “crossover” design, in which all patients are allowed access to the drug after the trial is done, is often employed in oncology trials.  It is an ethical consideration to allow patients who have participated in the trial to benefit from the drug, should it prove to be efficacious. Allowing this crossover complicates a determination of whether or not the drug provides a longer-term survival benefit, but in cancer, and in ALS, it is the only ethical way to proceed and therefore is a part of our trial.  I hope that this answers your question.  Also, if your physician has questions about the trial or treatment, I would be happy to have someone from our medical affairs team talk with him or her.

George

*************************************************************************************

My reply to the above message, July 25

Hi, George,

Thanks  for this explanation.

I have, I think, only one more question: if I’m forced out of the trial before the trial officially ends because I get a diaphragm pacer, will be I be among nonetheless considered to be “a patient who has participated in the trial” allowed access to the trial if it proves efficacious?

I think the answer this should be “yes,” but I want to be sure.

I’m very familiar with the cross-over design of cancer trials, and have argued with others about for years. But ALS is not cancer, as you know.

Thanks again.

Barbara

*******************************************************************************

Follow up message from me, July 25

Hi, again George,

I have one more question, and I thought I could save you multiple email repsonses.

If the answer to my question below about access to the drug if the trial proves effective is “no,” (which I don’t think it should be, given our exchange to date), will your company consider a compassionate (expanded) access program?

Thanks again.

Barbara

*******************************************************************

Response to above two messages,  July 26. 2012

Hi Barbara,

I’m sorry not to have responded earlier.  I had a family emergency and have been off line for the past 2 days (and will be only sporadically checking in for the next 3 or 4 days). However, I do appreciate the need to get you an answer.  Unfortunately I believe that I wasn’t clear.  In order to receive drug at the conclusion of the trial in September, you have to complete the trial, for all the reasons that we’ve been discussing.  We will have an expanded access program and will make drug available as quickly as we can.  We will make it available through sites that participated in the trial, so you should have access to the program.  I’ll check in with our clinical people to give you more insight into the timing of drug availability through that program (assuming that the trial is positive) and get back to you.  I believe that it will be around year-end, but I will get you a more accurate answer.

Sincerely,

George

***************************************************************************************

Thanks for getting back to me. This answer is clear. I will update my blog today or tomorrow. You might want to take a look in your copious spare time.

Barbara

Posted in ALS, ALS Treatment, Health Policy, Medical Science | Tagged , , , , , | 4 Comments

Broadening ALS Research to Help Patients — II

Late last week I posted here a message I sent I had sent to the CEO of Biogen Idec, the company running the dex drug trial I’m in. I encouraged people to write to the CEO, Dr. George Scangos, about the company’s plan to drop from the trial anyone who gets a diaphragm pacer before the trial goes to open label in September of this year. That post is the one immediately below this one, called “Let’s Broaden ALS Research to Help Patients.”

Dr. Scangos has not replied to my message. But he did send a canned reply to the many people who emailed him on my behalf.

Based on that reply to others, I have sent another — more pointed — message to Dr. Scangos. That message is here. Also here is a copy of the doctor’s canned reply.

It’s not too late to email Dr. Scangos and tell him what you think. I hope you will. If you’ve already emailed and were unhappy with his response, my message here might give you ideas of what more to say to him. His email address is: gscangos@biogenidec.com, unless he changes it to avoid these messages.

This is not just about me, or just about dex. It is about all health research and whose interests it serves: when is it important to let patients decide what is in their best interest without being forced to make impossible choices? When being forced to make those choices makes no sense either in science or ethics.

© Barbara A. Brenner 2012

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My response to Dr. Scangos’s messages about my request.

July 22, 2012

Dr. George A. Scangos

CEO

Biogen Idec

Via email:

RE: Continued participation in the dex trial

Dear Dr. Scangos:

As you no doubt know, I have not received from you the courtesy of a reply to my message to you of July 19, 2012. But people who contacted you on my behalf– some of whom I don’t even know — have forwarded to me the response you sent to them. I write to address what you say in that response in hopes of getting a prompt resolution to this very important matter.

I hope that, at some point, we can discuss ways to better design drug trials for the benefit of ALS patients, but what we need to resolve now is why Biogen Idec’s policies are inconsistent with the rules set up for this trial. You say in your message, which I have copied below, that each patient has to be followed for a minimum of a year. As I pointed out in my first message to you, I have been in the trial for 14 months. Under these circumstances, I fail to see — and you do not pinpoint — the exact violation of the trial protocol that my obtaining a diaphragm pacer at this stage would represent.

I am not asking for an exception to your protocol. I am asking that you implement policies consistent with it for the benefit of patients with ALS. To do otherwise is to hold ALS patients hostage to your trial. That is inhumane.

I fail to see — and nothing you say addresses — what inhibits you from doing a subgroup analysis of patients in the trial for more than a year who undertake other treatments thereafter, or cutting off analysis of their results after a year.

I understand that you need to protect the comparison between the placebo group and the treatment group. What I don’t understand is how, given the one year participation requirement of the trial, a diaphragm pacer in patients who have been in the trial more than a year would jeopardize that comparison.

This is not 1950. You have the capacity to do a staggered analysis of the data from this trial based on when patients entered it and one year has passed. As I understand  it, that is what your protocol requires.

I am entitled to and expect a response to this message.

Sincerely,

Barbara A. Brenner

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Dr. Scangos’s message about my request

I have received 18 emails requesting that Biogen Idec allow Barbara Brenner to stay on the trial while she receives a diaphragm pacer, and they are still coming.  I am touched by the letters and it is obvious that Barbara has a great number of people who care deeply about her.  Our mission at Biogen Idec is to bring better treatments to people with ALS and with other neurodegenerative disorders.  We have physicians working for us who still treat patients with ALS, and we have employees with MS, ALS, and other degenerative diseases.  I, and all the employees of our company are keenly aware how devastating the disease can be, and that these diseases affect not only the patient, but their family, friends, and colleagues.  It is the desperate need for better care for these patients that motivates us to work the long hours that we do.

We are conducting the phase 3 trial of Dexpramipexole under an agreement with the FDA. The trial is intended to demonstrate whether or not Dexpramipexole has the ability to reduce the rate of disability progression and/or prolong the lives of patients with ALS.  Half of the patients are receiving drug, and half are receiving placebo. Neither the company nor the treating physicians know whether a patient is receiving drug or placebo, and we won’t know until the end of the trial when it is unblinded.   Each patient is being followed for a minimum of one year.  The last patient was enrolled in the trial last September and therefore the trial will be completed this September, after the 1 year period is reached by all patients.  One of the requirements to ensure that a valid comparison is made between patients treated with the drug and those on placebo is to ensure that they adhere to the same treatment protocols.  Therefore we have rules that a patient must meet to be included in the trial.  If we make exceptions to these rules, and patients begin to receive different treatments, then the validity of the comparison between drug-treated and placebo-treated patients could be jeopardized.  If that comparison is jeopardized, then our ability to determine whether or not the drug actually works also could be jeopardized,  and therefore its availability to all patients with ALS could be substantially delayed.  Therefore, we are unable to grant these requests because to do so jeopardizes the validity of the trial, which could prolong the time until the drug is available to all patients.

I want to make sure that you understand that the drug has not been proven to work.  The drug had encouraging data in a phase 2 trial, but there are many examples of drugs that had such data and then failed to demonstrate a benefit in larger phase 3 trials.  The purpose of doing the phase 3 trial is to determine if the drug does indeed provide a benefit to patients with ALS.  After the last patient finishes their 1 year treatment period, it will take some time to unblind and analyze the data.  We should know whether or not the drug provided a benefit later this year.  It is possible that the drug will not work.  In that case, we will stop the trial and move on to our other projects designed to find a treatment for ALS.  If the drug does appear to provide a benefit, we will work with the FDA to make the drug available to some patients even before it is approved.  We understand the desperate need for treatment and the importance of time for patients with ALS, and are doing everything we can to bring a treatment forward as quickly as we can.  I am deeply sorry, but we simply can not, however, allow deviations that could jeopardize the validity of the trial.

You should know that we remain committed to find a cure for this disease over the long term.  We just last week initiated a collaboration with Duke University and the HudsonAlpha Institute to sequence the genomes of 500-1,000 patients with ALS to understand the genetic causes of the disease.  Our own researchers are working hard and working with top academicians around the country to find better treatments, and ultimately a cure for this horrible disease.

On a personal note, I am deeply sorry that we can not comply with your request.  Please know that my thoughts are with you and that we are doing everything that we can.

Sincerely,

George Scangos

 

 

George Scangos, Ph.D.

Chief Executive Officer

Biogen Idec

133 Boston Post Road

Weston, MA 02493

tel: 781-464-3288

fax: 866-899-3970

 

Posted in ALS, ALS Treatment, Health Policy | Tagged , , | 4 Comments