Richard's Blog

Post date: February 2, 2011

Sometimes, things are just as easy as they seem; sometimes the book is exactly what the cover makes it out to be. Generally, when this is the case, you don’t really discover this until after you’ve read the book and given it some thought and maybe even read the study guide. This has definitely been the case with me and my early experiences with the FDA as a patient representative.

Before we dive into my new reality, let me make something clear: I was not actively looking for a place in the world of drugs, biologics and devices, but my search for advocacy led me to the amazing people at ICAN, and they nominated me. I was honored to be asked, thrilled to be accepted and, in hindsight, surprised where my road had led me. Being the willing participant in 3 clinical trials, I know the stakes are high for patients, physicians, and drug companies, (not necessarily in that order), so being asked to participate in the drug approval process was a natural step that I did not know a patient could even take. Like most cancer patients, I had done scads of undirected research, joined the requisite groups online, and progressed to being a very active patient in my own care and feeding, but still had a thirst I could not quite quench. I began looking at ways I could actively advocate for the community at large and found this was easier said than done. Then, I met Marcia at ICAN and the rest is unfolding before me.

I am not a sycophant, and I am not easily impressed with people or positions; in my professional life I have dealt with many extremely famous people and while I do enjoy it when they turn out to be good people (rarely), I am not star struck or feel the need to curry their favor. The observations I make here are sincere, and they serve no purpose to elevate me in any way with anyone. If I lost this position tomorrow, my opinions would be the same.

The public perception of the FDA through the popular media can be confusing; there is often a slant that the FDA is trying to keep potentially beneficial drugs, vitamins, supplements, and medical devices from the public for some reason that is never adequately explained. If you were to pose the simple question, “why would they do this?” there isn’t really a good or plausible explanation for it. The FDA is extremely careful not to be perceived as being influenced by any outside parties, particularly pharmaceutical companies, physicians, or even a patient representative.
Part of the FDA mission statement is:

"FDA is responsible for protecting the public health by assuring the safety, efficacy and security of human and veterinary drugs, biological products, medical devices, our nation’s food supply, cosmetics, and products that emit radiation."

Another relevant explanation of the FDA’s mission is:

FDA is responsible for: 

Seems simple enough, right? Not so much, as I found out rather quickly. The tasks associated with these statements are huge, varied, multi-layered, complex, and simply massive in number.

After going through the very meticulous and painstakingly slow, (it is the government after all!) process to apply, be vetted, educated and accepted, I got my first look into the world I had entered at a seminar held in Maryland in September. This was a fly in, go to the hotel, meet for 2 days, and fly out meeting that was all business, and I mean that in a good way. They do NOT mess around with wasted time, social functions, or ‘let’s pat ourselves on the back’ parties. Truth be told, I found that the FDA is a grossly under-funded organization, which cannot afford to waste its own time or that of its members. No wonder ICAN quickly became a member of the Alliance for a Stronger FDA when that group was formed (Please see The FDA has a very limited budget for these patient rep gatherings, and some years they don’t have the funds to have them at all. Think about it; a critical arm of the approval process can be patient representation, and the FDA does not have enough funds to keep it up-to-date, educated, and actively involved. From what I saw, this was not a ‘misappropriation of funds’ issue, this was simply a “lack of funds” issue.

The scene is not hard to imagine for anyone who has ever been to any hotel for a ‘function’ of any kind; a medium sized room with 7 round tables each having places for 6 or 7 people. A podium in front with a projection screen behind it, a longer table off to the right side of the podium with 4 seats and 4 microphones. On each table, water pitcher, glasses, note pads and pens. A very basic set up for listening, questioning, learning, and exchanging ideas. On the morning of the first day, we checked in, picked up our bags full of the necessary paperwork, presentation materials and general workshop needs.  A good spread of breakfast items was available, along with copious amounts of coffee, tea, water, juices, and sodas to get us through the morning sessions until lunch, which would also be served in the same room.


Since I began writing this, a few things of interest have occurred that I feel must be noted. First, I am back in treatment; while on the surface this might seem like a bad thing, it is actually a very good thing. As previously stated, I am in a Phase I clinical trial for a drug that appears to be doing miraculous things, not just for me, but for most of the trial participants. The one thing the drug is not doing is clearing my marrow, which is a critical part of any real ‘remission’ or the measure of success that needs to be seen for the ultimate, final yardstick, 'approval'. So, I am adding a second, already approved, drug to the mix in hopes that it will clear the marrow and I will attain a very complete remission and then the clinical trial drug will just keep me there, (fingers crossed). Next to a full-blown cure, this is about as good as it can get, which is amazing! I am 7 weeks into an 8-week initial cycle, followed by 4 more treatments once-a-month. No side effects, full of energy, and feeling fantastic overall.

Second, the ASH (American Society of Hematology) Conference has just come and gone, and I wanted to point out a couple of things, as they relate to our subject at hand and me. There is a tremendous amount of optimism in the leukemia and lymphoma worlds for new, more targeted treatments (Yea!). One of the star drugs being lauded is the drug I am fortunate enough to be taking, so I am extremely lucky. However, the thing I really want to point out is the content of a few video interviews I watched with doctors of some fame and notoriety. In short, they were particularly vocal about the slow approval process for drugs and the strenuous processes that physicians (be they hematologists, oncologists, or other specialists), pharmaceutical and biotech companies have to go through to get the drugs to approval status. While not naming the FDA by name, it was clear to whom they were referring. I actually found this somewhat disturbing.

Yes, the clinical trials process is cumbersome and must be very frustrating to doctors and drug companies, but the dangers inherent in prematurely releasing a drug into the public domain must trump that frustration. I completely understand that there are patients dying every single day who would do anything to get the latest and greatest hope of a drug in their system, but as we all know, sometimes the cure can be worse than the disease. I would also say that very few people in the public sector know about the wonders of Gleevec for CML (chronic myelogenous leukemia), but everyone knows about Vioxx! The failures of the system are always the ones that we see the most media coverage on, and if the process is sped up, short-cut or condensed, the truth is that we will undoubtedly hear about many more Vioxxes, and in a shorter amount of time. The conundrum for the FDA in this is a difficult one; the agency is already demonized in so many ways, whether they approve, disapprove, or pull a drug from the market, yet if they began approving drugs with a less arduous process, the resulting outcry would be deafening. So it’s a no-win situation for the FDA, and they are thus siding with slower and more laser-lens scrutiny. Lastly, I found it interesting that physicians would be shortsighted enough to demonize where they could actually do a little bridge-building and possibly work on helping to get the FDA properly funded so that more could be achieved with a more robust FDA budget.

The outcry against the FDA is almost ear-shattering right now, with a few voices of reason and experience trying to provide balance, but its akin to yelling into the wind. The system is not perfect, nor would anyone at the FDA tell you it was, but it is continuing to evolve and I feel they are listening.


So, the conference begins with the customary opening remarks, glad you are all here, nice to see old faces and thrilled to see the new ones. Very quickly I found out there were less than 40 actual patient representatives here and that there are only 120 of us in this FDA patient representative training program. Period. One hundred and twenty patient reps to represent the needs of millions and millions of patients dealing with all types of disease.  I felt very privileged to be a part of this program but also felt it was inadequate to address the myriad of issues associated with patients and their needs. Patient reps at this meeting were experienced in the following diseases and device problems: prostate cancer, LASIK complication, HIV, cardiac implant, lung cancer, leukemia, Rett syndrome, depression, joint replacement, renal cell carcinoma, Parkinson’s disease, pancreatic cancer, liver disease, COPD, scoliosis, Pompe disease, neuroblastoma, hypertension, diabetes, and associated issues including insulin pump problems, arthritis, Crohn’s disease, gastroparesis, and migraines.

I found all of my fellow reps to be proud to serve and extremely committed; many of them expressed that they felt being a part of the FDA patient representative program was one of the most rewarding things they had ever done. The ones who had served on committee approval meetings were full of stories and anecdotes about the meetings, the processes, the stresses, and the complexities of the preparation and experience of meeting with the other participants on such pivotal topics that affect the entire nation.

Put yourself in these shoes:

About a month prior to the meeting, you get a fax of a Nondisclosure Agreement, which you must sign and return immediately. Shortly thereafter you receive a package of confidential materials pertaining to the meeting’s purpose, the drug/device/biologic you will be speaking about, (in all cases, this will be a drug pertaining to the disease/device/biologic you have experience with). This packet, by all accounts, can be HUGE and in some cases as much as 1200 pages; you must digest this information as thoroughly as possible, but above all, you must be prepared to speak to the merits or faults of the drug or its application to you and your disease. When the time comes to attend the meeting, you may be phoned in your hotel, followed to and from your hotel or car, questioned, pressured, and pilloried by various parties who have a high level of interest in the outcome of the meeting, sometimes financial, sometimes personal.

In the meeting room, you may find television cameras, bright lights, patients hanging on to life, slick haired Wall Street types, physicians, lawyers, and a variety of vultures circling. There may be 20 people, or there may be a fully “packed to the rafters” gallery of spectators and constituents from every possible interest group. There are long tables of scientists, doctors, drug company representatives and other ‘experts’. And then, there is your chair, from which you are supposed to expound on the pros and/or cons of the drug being considered for approval and how it relates to you, the patient. The intimidation must be palpable.

The full schedule in Maryland was as follows:
(I have omitted the names of all but 2 participants for what I think are obvious reasons. Many of them are patients, like me, who may wish to stay anonymous outside these proceedings)

2:00–2:20 p.m.  Introduction of Patient Representatives and Office of Special Health Issues Staff
2:20–3:35 p.m.    Session I:  Stats/Epi 101
Center for Drug Evaluation and Research Division of Biometrics
    • Questions/Answers
3:35–3:45 p.m.   Break
3:45–5:00 p.m. Session II:  Patient Panel
5:00–6:00 p.m.   Social Gathering/Hotel Lounge Area (I must point out, NO FREE COCKTAILS!)
6:10–6:30 p.m.   Dinner Presentation Center for Devices and Radiologic Health
  • Devices-Related Topic
6:30–8:00 p.m. Dinner (NO ALCOHOL)
7:00–8:30 a.m.  Registration and Continental Breakfast
8:30–9:00 a.m. Opening Remarks Margaret Hamburg, M.D Commissioner, U.S. Food and Drug Administration
9:00–9:15 a.m. Overview of Day: Andrea C. Furia-Helms, M.P.H.
Office of Special Health Issues Patient Representative Program
9:15–10:30 a.m.  Session I:  Drugs and Biologics Development Life Cycle
Office of Special Health Issues
    • Questions/Answers
10:30–10:45 a.m.  Break
10:45–noon Session II:  Device Development Lifecycle
Center for Devices and Radiologic Health Surgical, Orthopedic, and Restorative Devices
  • Questions/Answers
12:00–1:30 p.m Lunch
1:30–1:45 p.m. Viewing of Clips from "When a Patient Speaks"
1:45–2:45 p.m.

Breakout Activity Part I
Center for Drug Evaluation and Research Division of Anti-Viral Products Office of Special Health Issues
• Introduction
• Work in Groups

2:45–3:00 p.m. Break
3:00–3:45 p.m. Breakout Activity Part II
• Group Presentations
3:45–4:00 p.m. Closing Remarks - Off to the Airport

As you can see, there was not a moment wasted, and everything proceeded on schedule. Also, as pointed out, this was not 'wining and dining', and that’s not a complaint; I merely point it out as this was not a bunch of government employees blowing taxpayers dollars on lavish meals and frivolity. To the contrary, everything seemed prudently managed.

Each section was accompanied by a slide show and discussion, and each was extremely informative and packed with information I found compelling, interesting and important. In addition to the subjects above, we were given the following handouts:

While some of the titles were almost as long as the documents themselves, the information contained in each was essential to understanding the duties and complexities of the process as a whole and the pitfalls of potential conflicts of interest. I digested them all on the first night and the plane trip home and have reread them again since my return. I have also looked through the reproductions of the slide shows we received and tried to prepare myself as much as I can for the date when my phone will ring, a fax will come through, and my journey to my first Oncologic Drug Advisory Committee (ODAC) meeting begins.

The most enjoyable and educational part of the training program was a mini-workshop exercise where we were split into groups for the purpose of setting up a fictitious clinical trial to evaluate a drug’s interactions with a list of other drugs. The exercise split us into four groups and gave us all the same project; create a clinical trial paradigm where we test the interactions of 20-odd drugs with a new drug using treated and untreated populations for the most effective and dynamic results. We had a very short amount of time, and the task was daunting and, as you would expect, the clinical trial structures were all over the map. It was a very small illustration of how one aspect of the clinical trials process could be very challenging, and getting it wrong could have devastating results to the drug’s chances for FDA approval.

My thoughts and observations of the meetings, and my first experience in person with the FDA are as follows:

This is an organization with a lot of very good and dedicated people working hard against all odds to keep the public safe from accidental death by moving something too quickly through a very complex process. There are many, many layers to what the FDA is doing and trying to achieve, and yet the public at large thinks it should be simple. It is not. On top of all that, the fact that science went from moving at the speed of sound to the speed of light, and the complexities multiply exponentially while the drug pipeline doesn’t get any bigger, and the body of FDA personnel grows very slowly because of inadequate funding.

I believe that the FDA as a whole would be thrilled if they could change the system to be more accommodating, but to take the focus off of doing what they do to make those changes would effectively halt all of the processes they are engaged in. It is a situation where it seems impossible to make anybody happy while trying to keep everyone safe. As I have read many case studies of drug approval processes, it is apparent that the FDA needs to have more tools available to it for proper evaluation. In a situation where there are new types of treatments for conditions and diseases with histories of being treated in one way, there need to be tools to look at them in a whole new way. In other words, the FDA needs to be able to compare apples to oranges if they end up with similar results, or a new result that is as good or better. The science is going to demand it if the results look promising.

I have been fortunate to be able to see the FDA from the inside. Does this make me an expert? Certainly not. I am trying to continue my education by reading the information I can get from the FDA’s website communications, the latest of which is, which was launched to help companies and others save time and resources in their interactions with the agency. I get many updates from the various FDA websites and can stay informed on all of the various activities and drugs that are under review as well as the meetings pertaining to each. You can easily see that there is a myriad of activities and mountains of information by going on the FDA site and browsing.

Finally, I was fortunate enough to be asked to participate in a call for the purpose of giving input into the creation of the FDA OSHI Patient Network Website. This a much needed site where transparency will be the goal, and information will be  extremely thorough so that any patient that has any question about drugs, clinical trials, and the clinical trial process, critical path, and regulatory science, patient guides, and many, many other tools for everyone from the newly-diagnosed to the caregiver can go to get valuable information about what is available to them and how they can access it as well as how all the processes of the FDA work.

So, back to my "book versus cover" analogy: the FDA really is what it says it is: a government agency devoted to the safety of the public. They are not perfect, they are aware change is needed, and they are trying hard with a very limited amount of funding. Science is moving so quickly that there are more and more potential treatments literally every day. With every new possibility comes potential liabilities and the FDA tries to mitigate them as best they can. There are a large number of lawyers hiding in the shadows just waiting for them to make a mistake, which has such deep and far reaching effects on doctors, insurance companies, and all health- related industries that a mistake is just not worth the rush to judgment. I, too, wish there was a quicker path to drug approval, as I want to stay well! I am having the best possible result from my current trial, and I know the drug could fail during the final part of the approval process, but I am also confident that change is in the wind and am hopeful that the approval process will evolve.

My very simple advice is: Get involved. Advocate. Write to your congressmen and senators. Get informed. There are a number of emails I get that inform me about drug trials from the drug company side, which I recommend. You might want to check out: and sign up for alerts if you want to see the types of transactions that go on in the pharmaceutical world. Go to if you want to see clinical research news. Definitely go to and do a number of searches to see the sheer volume of information available. Search for the latest clinical trials for your disease or condition, join a list of others with your diagnosis, and digest the amount of information you need to help yourself or those you are caring for.

I will be back from time to time to update you with any news I feel warrants your time. When the new patient site debuts, you will read it here first. When I get the call for my first meeting, you will know as much as I can tell you without disclosing what I am not allowed to. After the next meeting, I will update my blog at ICAN to give you the gist of what happened.

I hope all readers of this blog find their way to good health and wish you all good fortune in your various searches.

Be well.

Richard MacIntyre
February 2, 2011