The Muscular Dystrophy Association and The ALS Association (ALSA) submitted a joint comment to the U.S. Food and Drug Administration (FDA) today, commending and thanking the agency for convening its first-ever ALS public hearing on Feb. 25, 2013, and encouraging the agency to quickly enact a number of specific action items to help accelerate therapy development for amyotrophic lateral sclerosis.
The joint comment follows up on the standing-room-only FDA ALS hearing, in which people with ALS, their families, clinicians, advocacy organizations and others urged the FDA to consider the unique needs of patients and families — for whom time is of the essence — when considering trial design and review of therapies. Today marks the last day of the FDA’s public comment period on this topic. (A webinar of the Feb. 25 FDA ALS hearing can be accessed online.)
Below is the entire text of the joint ALSA-MDA comment to the FDA, which was signed by MDA President and CEO Steven M. Derks, and ALSA President and CEO Jane Gilbert.
March 25, 2013
Margaret A. Hamburg, M.D., Commissioner
Janet Woodcock, M.D., Director
Russell Katz, M.D., Director
Re: Docket No. FDA-2013-N-0035, Considerations Regarding Food and Drug Administration Review and Regulation of Drugs for the Treatment of Amyotrophic Lateral Sclerosis; Public Hearing
The ALS Association (ALSA) and Muscular Dystrophy Association (MDA) applaud the Food and Drug Administration’s Division of Neurology Products and Office of Special Health Issues for providing the national ALS community of patients, health care providers, researchers, caregivers, academia, federal agencies and industry the opportunity to share the ALS story, our concerns, perspectives and recommendations to the Agency during the ALS Public Hearing on February 25, 2013, as well as during the public comment period. We are especially grateful to the personnel of the Office of Special Health Issues led by Drs. David Banks and Steve Morrin for their leadership and extraordinary accommodations on the day of the Public Hearing. The needs of every member of our community were anticipated and the Agency proactively worked to meet them on the hearing day. This was evident by the fact that the Agency made every effort to accommodate the more than 200 people in attendance and the nearly 60 individuals who provided testimony, as well as the hundreds of comments submitted by community members who were physically unable to travel to White Oak on February 25. We believe that the Agency’s efforts — and the significant efforts of the ALS community to participate in the hearing — reflect a recognition that there is tremendous urgency within the ALS community to establish policies that not only help to expedite the development and review of new treatments, but also patient access to those treatments.
The ALS Association and MDA are the two largest organizations representing and serving people with ALS in the United States. Both organizations help lead the fight against the disease through programs that include funding and directing worldwide ALS research and therapy development, providing care and services to people with ALS and their families, raising awareness of the disease and working with members of Congress and government agencies to advocate legislative and regulatory policies that benefit the ALS community.
Despite the resources leveraged by our organizations, federal partners and other nonprofit groups, as well as the exemplary care offered by multidisciplinary clinical teams throughout the country, the unmet medical needs of this disease are staggering. Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease. It is a particularly cruel disease that destroys a person’s ability to control all muscle movement, typically striking adults in the prime of their lives. As the disease progresses, its victims become trapped inside a body they no longer can control, often unable to walk, talk, eat, breathe or even blink an eye. There is no effective treatment for ALS, typically no known cause and no cure. Military veterans are approximately twice as likely to die from ALS as those who have not served in the military, although the disease can affect anyone, regardless of their age, gender, race or ethnicity. ALS is 100 percent fatal — in an average of two to five years following diagnosis.
While we were extremely pleased with the momentum leading up to and throughout the February 25 FDA ALS Hearing, we must ensure that there are real outcomes from this Hearing and the open Docket ID: FDA-2013-N-0035. To that end, the following are the most immediate action items that The ALS Association and the Muscular Dystrophy Association feel would help accelerate ALS therapy development with leadership from the FDA.
ALS is 100 percent fatal. The risk of no treatment intervention bears inherent side effects that would otherwise be categorized as "intolerable", and — in clinical trial terms — "SAEs" (or "serious adverse events"). In other words, the symptoms of ALS rival most side effects from experimental drugs: paralysis, loss of speech, loss of ability to swallow, loss of ability to breathe. The risks of the disease itself almost always outweigh the risks of the side effects from legitimate investigational treatments. Therefore, the willingness of ALS patients to assume a significant amount of risk must be considered at every step of the development and review process, whether that be clinical trials, expanded access programs or marketing approval.
Careful studies have documented that respiratory dysfunction from diaphragm and intercostal muscle weakness in ALS patients responds to respiratory therapy, and more specifically to the use of bilevel positive air pressure (BIPAP) and average volume-assured pressure support (AVAPS) devices. With the use of these devices, there is documented improvement in quality of life and length of life. Our patients often have difficulty adjusting to the masks used with these devices, and by trial and error finally find the right one. Furthermore, cough-assist devices that help immobilize secretions provide meaningful therapy preventing aspiration and pneumonia.
The ALS Association and the Muscular Dystrophy Association recognize that the FDA is charged with regulatory oversight of over 25 percent of the products used by the country’s citizens, yet is significantly underfunded. We continue to support additional appropriations for the Agency to ensure that it has the resources necessary to fulfill its mission and, as part of that mission, to help ensure people with ALS have access to effective and safe treatments as soon as possible.
Again, The ALS Association and Muscular Dystrophy Association appreciate the opportunity to participate in this landmark public hearing and comment period and are grateful to the FDA for making the acceleration of ALS therapy development a priority. We look forward to continuing to work with the Agency to ensure that all available resources are leveraged as best as possible in this effort and that people with ALS have a voice in the drug development and approval processes. This is not just the goal of our two organizations. It is the goal of the entire ALS community. As you are aware, people with ALS and those who have been touched by the disease and who have submitted a considerable number of comments to the docket for this initiative do not have time to wait. Their message is clear: ALS patients, and those who represent them, want to be heard and are looking to the Agency to help them access treatments that may save their lives. The ALS Association and Muscular Dystrophy Association support them, and we look forward to continuing to work with you as we search for the treatment and cure for Lou Gehrig’s Disease.
Steven M. Derks