How the FDA Approves Drugs: My Role on the Arthritis Advisory Committee

May 14, 2012 at 1:12 pm 21 comments

Behind the headlines “FDA Panel Unanimously Rejects…” and “Arthritis Pill From Pfizer Wins Support…” was, for me, over 600 pages of reading and two days at the FDA as the Consumer Representative to the Arthritis Advisory Committee (AAC). These were my first meetings following an almost yearlong process that started with a nomination by Diane Aronson, the Consumer Representative whose term was ending, and culminated with my four year committee appointment. The FDA has many advisory committees consisting of outside experts who provide the FDA with independent opinions and recommendations on applications to market new drugs.  As Consumer Representative, my role is to represent the consumer perspective on issues and actions before the FDA AAC.

The first meeting was to review Regeneron Pharmaceuticals’ Arcalyst, also known by its generic name rilonacept, for short-term use to treat gout, a form of arthritis. It was already on the market to treat a rare disease, Cryopyrin-Associated Periodic Syndromes. The second meeting was for Pfizer’s tofacitinib, which was under review for treatment of rheumatoid arthritis. Tofacitinib had quite a bit of press preceding this meeting, mostly focusing on it being a “potential blockbuster, with peak sales of $2 billion to $3 billion a year, if its benefits are deemed to outweigh the risks”.

The most grueling part of preparing for the meetings was reading the materials including clinical trial results from the pharmaceutical companies, who are called the sponsor, and from the FDA, who is called the agency. As I read, I focused on understanding the efficacy and safety of the drugs, and their impact on quality of life. I had many questions, some of which were answered in the documents, and some that I made note of to ask during the meetings.

Regenron’s rilonacept for gout

The May 8 meeting took place at the FDA White Oak Campus, where we had to go through security before going to the meeting room, which was largely empty. The front was roped off for the committee, who sat at a U-shaped table where name tents and folders were waiting for us. The sponsor, Regenron, had rows of chairs on one side, and the agency did on the other side. On the agency side there were people in military uniform, but I never found out who they were. When the meeting started, the process was very formal, with parallels to court although, carrying that analogy further, it wasn’t clear whether we were the judges, the jury, or both.

The meeting was presided over by Lenore M. Buckley, MD, MPH, the committee chair, and Philip Bautista, PharmD, the FDA’s Designated Federal Officer to the AAC. They started by summarizing the meeting agenda and instructing everyone on the protocols for speaking. Each committee member had a microphone with a talk button and yes-no-abstain voting buttons. It was an open meeting, with the public and press sparsely represented, and a live webcast.

The morning consisted of the sponsor’s presentation, followed by the agency’s presentation, each with time at the end for our questions. I was struck by how the sponsor had prepared slides for every anticipated question, all pre-loaded so readily available. I wondered about all the slides we weren’t seeing, and the questions we should be asking but hadn’t. The sponsor also brought experts, who all started by stating their financial involvement with the sponsor and if they benefit from the outcome of the meeting. I was struck by some of the differences between the language used and points made by the sponsor and the agency, including the sponsor’s use of “benefit risk profile” and the agency’s use of “risk benefit profile”.

Following lunch, any member of the public who registered in advance could make a statement. We had some letters in our packets from patients, patient advocates, and physicians that related to rilonacept or, in one case, to men’s health issues, since gout primarily affects men.

Once all sides had been heard, the agency posed their questions to the committee, some for discussion and some for vote. We had further opportunities to ask questions and many of them pertained to the risks. Once votes were captured for each of the three votes, the tally of the votes was displayed and then a table of how individuals voted. The chair asked every panelist to state their name, how they voted, and the reason. The stated reasons seemed to be the primary sound bites captured by the press, especially for the final vote, if the efficacy and safety data supported approval of rilonacept. The committee voted unanimously against approval; I later learned that unanimous votes are rare.

Pfizer’s tofacitinib for rheumatoid arthritis

The second meeting, on May 9, was very different from the first, starting with longer security lines when we arrived in the morning. It was an open meeting with a live webcast again, but this time the auditorium was packed, with a row of press and more patients and patient advocates, including one who I knew from Twitter, Kelly Young. There was a large contingent from Pfizer. One my fellow panelists had told me to expect far more people than the prior day because our vote has much greater financial impact, since this was potentially a blockbuster drug with projected sales of $2 billion to $3 billion a year.

The agenda was similar to the previous meeting. Pfizer presented and, like Regeneron, brought outside experts. Pfizer had many slides in addition to those used in their presentation to answer our questions. Clearly a lot of effort went into anticipating our questions, preparing slides, and indexing them to be able to locate them quickly in response to a question.

I was more confident asking questions than I had been at the first meeting because I better understood the process. I asked Pfizer to elaborate on the safety profile of the longer term clinical trial data regarding the two dosing options, 5 mg and 10 mg, evaluated in the clinical trials. I also asked them if they had plans for patient education and they showed a slide about Dear Doctor and Pharmacist letters which did not answer my question. There was some additional discussion about the efficacy and safety of smaller doses.

The final vote was 8-2 in favor.  Many who voted yes urged further research about some of its side effects, concerns that largely didn’t make the headlines. The New York Times accurately reported that we “urged the Food and Drug Administration to require rigorous follow-up studies.” Like the previous meeting, the final vote was only a recommendation to the FDA; as stated in the New York Times, “the agency, which is scheduled to decide on approval by August, usually — but not always — follows the advice of advisory committees.”

A public process for patient safety

The meetings were fascinating and I appreciated the public nature of the process, including making all materials available to the public before the meeting, opening it to the public in person and through streaming, and providing time for people like Jan Wyatt, RN, PhD to speak. I saw the meetings as a milestone in a long chain of events that started with years of research and clinical trials. The meetings were not the end since the FDA needs to make decisions based on the AAC recommendations and then work with the sponsors on their next steps to further ensure efficacy and safety.

I am honored to be part of this committee. Most of all, I have the deepest respect for the professionalism and knowledge of my fellow committee members, the agency, the sponsors, and the public who participated. Together I believe we have the shared goals of bringing new treatments to patients without compromising safety.

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21 Comments Add your own

  • 1. timimi  |  May 18, 2012 at 7:08 pm

    Apt and timely-I find the implied uneven access to primary data to be problematic, and emblematic of the process opportunity for more uniform transparency. Thank you so much for sharing!

  • 2. Peter Shea  |  May 16, 2012 at 8:10 am

    ” It was an open meeting with a live webcast again…” I wonder if anyone has done research about how the ubiquity of web-based communication tools has impacted the process of drug approval.

    • 3. Lisa Gualtieri  |  May 16, 2012 at 8:31 am

      I have no idea how many people watched – and the archived session is available from the FDA. But it is a great way of increasing access, don’t you think?

      • 4. Peter Shea  |  May 16, 2012 at 10:11 am

        Yes, I think it contributes to the transparency of the process and makes it feel more inclusive.

  • 5. Anneliz Hannan (@annelizhannan)  |  May 16, 2012 at 2:32 am

    Thanks for sharing Lisa, you really clarified the process. Happy to share.

    • 6. Lisa Gualtieri  |  May 16, 2012 at 5:32 am

      Thank you, Anneliz, I appreciate your feedback.

  • 7. Kathleen Hoffman, PhD  |  May 16, 2012 at 1:38 am

    This is a wonderful explanation of the process. I am thrilled that you are representing consumer interests. I’m especially glad that you asked about the educational piece and have let us know that your questions were not adequately answered. Thank you for this post.

    • 8. Lisa Gualtieri  |  May 16, 2012 at 5:31 am

      Thank you for commenting, Kathleen, and I appreciate your appreciation!

  • 9. westrwjr  |  May 15, 2012 at 7:10 pm

    Bravo Lisa! Thanks for taking the time to educate your fellow health activists!!
    – Bob

  • 11. lkafle  |  May 15, 2012 at 5:20 pm

    Reblogged this on lava kafle kathmandu nepal.

  • 12. dandunlop  |  May 15, 2012 at 4:50 pm

    Thanks for this rare look inside the FDA drug approval process. I was thoroughly engaged by your story and the insights you shared.

    • 13. Lisa Gualtieri  |  May 16, 2012 at 5:30 am

      Thank you, Dan, and thank you for helping me get more readers!!!!

  • […] you don’t know it already, I am a fan of Lisa Gualtieri and her blog: Lisa Gualtieri’s Blog on Health. Lisa is an Assistant Professor at Tufts University School of Medicine in the Health Communication […]

  • 15. Gary Schwitzer (@garyschwitzer)  |  May 15, 2012 at 6:56 am


    Interesting story about your involvement on that FDA committee.

    I serve on the FDA Risk Communication Advisory Committee, and contributed a chapter to the book, “Communicating Risks and Benefits: An Evidence-Based User’s Guide.”

    Gary Schwitzer

    • 16. Lisa Gualtieri  |  May 16, 2012 at 5:29 am

      Gary – you had mentioned this when you guest lectured and I need to read it, thank you for the reminder and for your great work.

      Please come back to the summer institute – if not this summer, the following one.

  • 17. Kathy Kastner  |  May 15, 2012 at 4:23 am

    So glad you found your courage:)
    Thanks for all the deets. Having read of the discouraging experiences of some patients brought into healthcare conferences as ‘token’, I am glad to hear the FDA shows due respect and appreciation.

  • 18. Kathy Kastner  |  May 15, 2012 at 3:02 am

    Lisa, this is doubly interesting for me: I was nominated but was disqualified 😦 because FDA’d hired my firm [ should you be interested, the task was to assess whether or not safety officers in hospitals knew about /used FDA’s Patient Safety News – which gives direct-from-the source-medication alerts and updates. Only 10% knew of it}

    Where I’d been overjoyed to get the FDA commission, I was totally bummed that it excluded me – so thanks for bird’s eye view.

    Whack of reading! I wondered how they’d get you up to speed on what I imagine must be pretty dense stuff.

    Did you have access to support if needed? (e.g. needed clarification)
    Were you given sufficient opportunity to give input?
    Did you feel you were being listened to?
    What weight does your input have, do you know?

    Thanks again for sharing. What an experience!

    • 19. Lisa Gualtieri  |  May 15, 2012 at 3:26 am

      Thank you for the feedback and insights, Kathy. To answer your questions:

      The person I was replacing, Diane Aronson, gave me a number of suggestions based on her experiences that helped me prepare for and participate in the meetings, including that there would be voting buttons. I think the people at the FDA, especially Phil Bautista, would have provided support if I had requested it in advance. And the other members of the committee, who I met that morning on the shuttle bus to the FDA, were very helpful too.

      I felt uncomfortable talking at the first meeting but less so at the second. I jokingly reminded myself that there is no such thing as a stupid question, but initially I was concerned that my questions were obvious to everyone else and not to me. The schedule had time built into it for discussion and questions, so there were no time constraints.

      I very much felt like I was being listened to and respected for my role. The FDA people in particular expressed appreciation formally and informally (during breaks and at the end).

      I am a voting member so my vote has the same weight as the MDs and other committee members. What else can I tell you? I appreciate your interest?

  • 20. Paulo Machado - @pjmachado  |  May 15, 2012 at 12:12 am

    Thanks for sharing & being part of the process Lisa!
    Glad that you were able to share a small piece of the BioPharma R&D process with us. Should provide a sense of why pharma pricing is higher than hoped for by the public. Very costly & time consuming process w/low odds of commercial success…

    • 21. Lisa Gualtieri  |  May 16, 2012 at 5:26 am

      Thanks, Paulo, and, yes, I agree that the expense of developing and testing drugs is huge and certainly must be reflected in the cost of the drugs that are approved.


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Lisa Gualtieri, PhD, ScM

Lisa GualtieriLisa Gualtieri is Assistant Professor at Tufts University School of Medicine in the Department of Public Health and Community Medicine. She is Director of the Certificate Program in Digital Health Communication. Lisa teaches Designing Health Campaigns using Social Media, Social Media and Health, Mobile Health Design, and Digital Strategies for Health Communication. Contact Lisa:

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