Regulatory Sclerosis

Upcoming medication for MS, Ocrelizumab

Regulatory Sclerosis, Guest post by Chris DeMuth Jr.

Thank you very much for the opportunity to write a guest blog post.  Earlier today, the Wall Street Journal published an article that I wrote on MS and a recent FDA rejection for an innovative therapy for MS.  If you’re at all interested in the article itself and don’t have access to the Wall Street Journal, you can read it here so I won’t paraphrase its points.

Instead, I want to offer a few observations about the MS community itself, how it is organized, and impediments to its effectiveness at getting path breaking therapies through the byzantine FDA bureaucracy in Washington, DC.  These observations are based upon my firsthand interactions with regulators, patients, and doctors over the past few months as I have worked – unsuccessfully, thus far – towards the goal of getting a new product for MS from the laboratory to the market in the US.


The Regulators

The FDA bureaucracy takes its job seriously.  Regulators examine new drug applications based on their safety and efficacy.  However, their incentives are quite different from that of patients.  First and foremost, they are in the business of protecting the FDA’s control over medicine and protecting their institutional reputation.  Neither patients nor bureaucrats want errors in deciding which drugs to approve.  However, there are both “type one errors” (false positives in which a drug is approved that should not have been) and “type two errors” (false negatives in which a drug that should have been approved is rejected).  For patients, they are both problematic.  For regulators, all of their downside lies in type one errors.  The FDA does not share the downside associated with waiting for a drug that could be saving or improving lives.

The Patients

Late last year, I had the opportunity to attend an FDA advisory committee meeting along with a friend with MS who wanted to go so that she could speak during the open forum.  She was courageous and eloquent in her advocacy for new therapies for MS.  She was also extremely knowledgeable about her disease.  Whether it is due to changing relationships between patients and doctors, information on the internet, or progress in medical innovation, today patients are sophisticated in managing their health.  However, the regulators have not kept up with this development.  Much of the FDA’s energies are dedicated to eliminating choices – choices that would be appropriate for patients and their doctors to consider.

The Doctors

I have been gratified by an outpouring of supportive e-mails from neurologists who treat MS since my article came out.  Doctor after doctor has written with variations on the same message:

I agree that doctors should have the choice to treat our patients with Lemtrada.  In fact, I am treating patients with Lemtrada today.  However, I want to avoid retribution from the FDA, so I am remaining silent.

Thus, many of the most knowledgeable and supportive experts have not been heard from.  The Lemtrada appeal is at a critical stage that could be influenced by the views of MS doctors and patients.  If you have a view on my article – favorable or unfavorable – or on whether or not Lemtrada should be one option for MS doctors treating their patients, then please consider writing a letter to the Wall Street Journal’s editor.
Editor, The Wall Street Journal
1211 Avenue of the Americas
New York, NY 10036

One important attribute of the FDA’s review was the total exclusion of MS experts and patients from their decision-making process.  They handpicked bureaucrats who lacked any sub-specialty background in MS.  So, now could be a useful time for the people whose lives are directly impacted to speak up.  For something to think about, here are some recent quotes from three experts who are risking retribution from the FDA by speaking up for new therapies.

My hope and expectation had been that this would be a normal process involving evidence-based science.  There was no reason for any controversy.  However, the FDA turned this into a battle of the FDA versus MS patients, MS doctors, and clinical research.  The FDA regulators have made clear that they can live with any amount of delay in the approval of new therapies.  If you would like to weigh in on this issue, it could greatly befit everyone for whom new therapies demand a greater urgency.  It is heartening to see MS patients and their doctors willing to step forward by signing and commenting on Lisa’s petition.  MS patients and their doctors are becoming harder and harder for the FDA to keep ignoring.

Chris DeMuth Jr and daughter, St. John, USVI

MS Blogger and Multiple Sclerosis Activist shares her journey living with MS, tips for others living MS and her husband, Steve, offers his insight as a caregiver for MS.