From ALS Bites

A Letter to Washington


Click here to read letterThe Food and Drug Administration (FDA) has announced a public meeting and invited public comment on a proposal related to its patient-focused drug development initiative under the Prescription Drug User Fee Act (PDUFA).

The FDA has developed a preliminary list of 39 potential “disease areas” from which it proposes to choose 20 to be the focus of disease-specific meetings over the next five years.[1] The purpose of these meetings will be to “address the significant interest in patient-focused drug development within the patient community.”

Some serious and life-threatening diseases and conditions are on this list, including heart failure, depression, HIV, lung cancer and others. Also listed are non-life-threatening diseases and conditions of varying seriousness such as fibromyalgia, chronic fatigue syndrome, alopecia, irritable bowel syndrome, female sexual dysfunction, “fracture healing” and nocturia (the need to get up at night to urinate).

Amotrophic lateral sclerosis (ALS), or “Lou Gehrig’s disease,” does not make the list.

See the FDA’s notice and request for comments.
Please consider submitting your comments.
Read what others have said.

One or more of six criteria identified by the FDA must apply to a “disease area” for it to be considered for this program. ALS meets all six criteria. For a variety of reasons beyond its serious, incurable, and fatal nature, ALS belongs at the top of this list.

The ALS community is growing increasingly frustrated with FDA rules and regulations that can stifle drug development and that deny “compassionate use” of promising drugs deemed safe, but not yet proven to be effective, to patients who have no other options for treatment. One measure of this frustration is that a very robust do-it-yourself movement is gaining traction. In April of this year, the Wall Street Journal published “Frustrated ALS Patients Concoct Their Own Drug”. Other media outlets, including the New York Times and ABC News reported on this story. This is not the only group of patients going this route. Others travel around the world to undergo unproven and expensive treatments, thereby making themselves ineligible for FDA-approved drug trials.

A nonprofit group, ALS Treat Us Now, has collected almost 20,000 signatures in an effort to push the FDA to allow increased access to experimental drugs to ALS patients who have no other hope for treatment, and very little to lose by trying unproven but promising therapies.

It is already difficult to recruit sufficient numbers of patients to participate in FDA-approved studies related to ALS because of narrow eligibility requirements and the short life expectancies of people diagnosed with the disease. The dissatisfaction with and distrust of the FDA by the ALS community threatens to further reduce this pool of potential study subjects. This is a serious problem with the potential to undermine and slow important research.

So it has never been more important for the FDA, pharmaceutical companies, and the ALS community to communicate and cooperate. This patient-focused drug development initiative under PDUFA is an important opportunity to make this happen. With all due respect to anyone who stumbles out of bed at two AM to urinate, amyotrophic lateral sclerosis should be at the top of this list.

Read a PDF copy of my letter to the FDA. I will mail this today, and I will send copies—with cover letters—to my two U. S. Senators and to my Congressperson.

Recent ALS Bites:
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05/12/2017 The Eyes Have It
08/04/2016 YFALS National Corntoss Challenge
07/18/2016 Scopolamine Blues
03/06/2016 Dragon Me Down
02/12/2016 Five Years In

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Notes

  1. The complete list of 39 “disease areas” is available in the FDA’s document. For those who can’t wait to see the list, here it is: pulmonary arterial hypertension, heart failure, primary glomerular diseases, narcolepsy, Huntington’s disease, depression, autism, peripheral neuropathy, fibromyalgia, obesity, nocturia, chronic fatigue syndrome, irritable bowel syndrome, inflammatory bowel disease, alopecia areata, diabetic ulcers, female sexual dysfunction, interstitial cystitis/painful bladder syndrome, fracture healing, diabetic foot infections, hepatitis C, HIV, patients who have experienced an organ transplant, sickle cell disease, chronic graft versus host disease, amyloidosis, aplastic anemia, melanoma, lung cancer, cancer and young patients, cancer treatment in pregnancy, cancer and sexual dysfunction, cancer and depression, clotting disorders, thrombotic disorders, primary humoral immune deficiencies, Neurologic disorders treated with immune globulins, hereditary angioedema, and alpha-1 antitrypsin deficiency. [^]