Field Trip: FDA Breast Cancer Patient Focused Drug Development

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I think it all the time.  I am so happy to live where I do.  Last week, after running a couple of errands, I headed up to spend the afternoon at the FDA White Oak campus in Silver Spring, MD. `I didn’t have to hop on a plane, get a hotel, and try to arrange my family’s schedule so that I could be gone for several days.  I just headed over after stopping at Tyson’s. It’s a brand new campus and everything is so fancy– much different than my visits to the FDA buildings that were on the NIH campus when Clay worked there.  The FDA is conducting meetings over a five year period on different disease sites, inviting patients to influence the drug development process.  I was there for the breast cancer meeting (obviously!) along with roughly fifteen to twenty other patients and patient advocates.  The rest of the audience was made up of academics and pharmaceutical representatives who were eager to hear our comments.  (They were not permitted to participate in the discussion, they were just there to listen.)
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I thought the meeting was well organized and well run, and the FDA staff and reviewers listened very attentively and asked thoughtful questions.  Even when the discussion started to get off course, they managed to respectfully and gently guide us back– and that’s no small feat!  I had three main takeaways from this meeting.  First, I continue to be in awe of the passion and energy of the metastatic breast cancer community.  I sat with several ladies from the Metastatic Breast Cancer Network and the Metastatic Breast Cancer Alliance.  It makes sense that the women who were chosen to be on the panels were mostly metastatic patients– they’ve had far more treatments, so of course they’ve had to deal with more side effects.  Many of these ladies talked about overwhelming fatigue, pain, and the stress of trying to make sure they’ve scheduled their “life” to fit within their two or three week chemo cycles.Their cancer cards are still shiny– though be it a bit worn– and no one would call them selfish for wanting to stay at home– either to feel sorry for themselves, or just to spend as much time as possible with their families.  And yet they traveled from Chicago, New York City, Texas, California– all to share their stories, hoping they will make a difference, if not for themselves, for those yet to be diagnosed with breast cancer.  They spoke with candor about things most people would rather hide– diarrhea, depression, sexual intimacy.  They shared the joys of being without evidence of disease after treatment for multiple metastatic lesions, and they shared the disappointment of recent disease progression. While I know one person’s story is always just one person’s story, I was reminded of the power of an individual’s personal narrative.

The other two things that struck me were two of the questions posed by the FDA panel.  They wanted to know from the patients– What would our dream drug look like? Um, one pill, no side effects, totally and instantly curative.  OK, I know that’s not what they were going for!  And that’s not what anyone said, either. The most poignant answer came from Shirley Mertz, from the Metastatic Breast Cancer Network.  I’m totally paraphrasing here, (apologies to Shirley if I botch it!) but she pointed out the need for more endpoints in new drug trials.  Most trials for metastatic disease look at tumor shrinkage as an endpoint.  (Makes sense, it seems like a shrinking tumor is a good thing.) But she encouraged the FDA to consider no new metastatic lesions– tumors– as an endpoint.  She said most metastatic patients feel like they can handle their current level of tumor burden, what they all fear is the spread to new organ sites.  Her dream drug would stop the tumors in place, prevent them from going anywhere new. She eloquently presented a perspective (and idea) that I found very interesting. I really love how excited she was to share her ideas with the FDA– she wants them to change the way they do the trials and she wasn’t afraid to tell them! (And she did it with the biggest smile, you could tell she reveled at the opportunity!)

The final question that impressed me: What’s’ your deal breaker? I didn’t love that the panelist started talking about hair loss, as if there were a lot of  women who A) didn’t know that chemo causes hair loss and B) wouldn’t take the needed chemo treatment if they’d known. ??? But once I got past that, I thought it was a brilliant question.  The FDA sees a list of potential side effects for every drug, but a patient’s perspectives on how to judge those side effects could make a difference in how they look at the applications. For the most part, the discussion was pretty clearly split.  First treatment to stages I-III: there are no deal breakers.  Theses women see the treatment as potentially curative and so are willing to endure most anything for a limited time to avoid feeling later like they didn’t do enough.  Once you move on to long term hormonal treatment (anti-estrogen, aromatase inhibitor, ovarian suppression), it gets tougher, this is five or ten years of a drug for a women with no evidence of metastatic disease. Young women in particular often struggle with the side effects– early menopause, sexual side effects, depression, weight gain. For some women, these side effects are so extreme that they become deal breakers and they stop treatment.  Metastatic disease is obviously a different ballgame entirely.  The Metastatic Breast Cancer Network describes the routine simply as “Scan, Treat, Repeat.” For the most part, this cycle is repeated every three months.  Women who are fortunate to live longer than the average three years after metastatic diagnosis have often undergone numerous treatment regimens. They stay on one until their disease progresses or they are unable to cope with the side effects, and then their doctors search for yet another option.  Their deal breakers are very different, and they seem to fall into two groups. The young mothers I’ve encountered with metastatic disease seem to be willing to do just about anything, to endure any side effect that is not actively threatening to claim their life before the cancer can.  They want one more day to see their babies grow up, to get to help their daughter buy a prom dress. They just want one more day, and they are willing to suffer any amount of pain to get it.  At some point, though, quality of life becomes a much larger argument. Many present, women who didn’t have little ones running around the house or children about to graduate from high school, spoke of their deal breakers– they were tired of all the side effects and would not choose a treatment that promised them little additional time at a great cost to their body.  One woman spoke about choosing a treatment that had a more convenient dosing schedule– an injection once a month over biweekly, three hour infusions.  Sadly, the financial cost of treatment was mentioned several times, one women adding that she wouldn’t want a drug that affected her so adversely that she couldn’t keep her job– and therefore her health insurance. They emphasized that quality of life could be more important than length of life in the decision making process, and while rough side effects shouldn’t keep a drug off the market, we shouldn’t settle for drugs without considering their toxicities.

I won’t say that I think this afternoon solved all the problems that exist in the treatment of breast cancer. I wasn’t sure what to expect, and frankly, didn’t expect much. I thought it would probably be the FDA’s way of checking off a box– yes, we talked to breast cancer patients. Done. I know that cancer drugs are still going to have some pretty rotten side effects, and my talking about them won’t make them disappear. Yet I feel like the FDA panelists were truly listening, and that our discussions might influence what level of side effects they find acceptable. Perhaps they will consider adding new trial endpoints, or they will require more data on dosing to stop the “more is better” philosophy that is common in cancer treatment. The meeting is over, but the public docket is open for comments until June 2, at which time they will be summarized for the permanent record. If you’d like to add your comments, please head to their page and do so. (There are some specific questions that they want patients to address, found about halfway down the page under the “Public Meeting Information” heading. You can add your comments by clicking the blue “comment now” button at the top right of the page.)

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3 Thoughts on “Field Trip: FDA Breast Cancer Patient Focused Drug Development

  1. Great Post, Jamie. We do live in an amazing area! And you were the perfect person for this panel.

  2. Nice summary. I wish I could have been there. I am submitting comments and observations via the feedback program they made available.

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