Division of Documents Management
Food and Drug Administration
10903 New Hampshire Ave.
Bldg. 51, Rm. 1168
Silver Spring, MD 20993
Re: Docket No. FDA-2012-N-0967, Prescription Drug User Fee Act Patient-Focused Drug Development
The ALS Association and Muscular Dystrophy Association (MDA) appreciate the opportunity to comment on the Patient-Focused Drug Development Initiative and the Agency’s preliminary list of nominated disease areas for the initiative. We commend the FDA’s commitment to obtaining stakeholder community perspectives on disease severity and unmet medical needs. We believe that it is absolutely critical that people with ALS and the organizations representing them have opportunities to share with the FDA our unique perspectives on benefit-risk and other elements of the drug development and approval processes.
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.
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, 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% fatal — in an average of two to five years following diagnosis.
We believe that people with ALS have an important role in the drug development and approval process, a process where results can mean the difference between life and death. This is especially true when it comes to ALS. As we have stated, currently there are no effective treatment options that significantly slow, stop or reverse the progression of the disease. It is a disease with significant unmet medical needs and those diagnosed with ALS will die from the disease in the absence of an effective treatment.
Because of the certainty of rapid progression and the short timeline, the perspective and benefit-risk tolerance of people with ALS is much different than that of the general public or even a person with a chronic condition. It is different than that of an FDA reviewer
and those in industry. The views of people with ALS are instrumental and must be taken into account throughout the drug development and approval process — whether it is their views on benefit-risk decisions, expanded access, accelerated approval, clinical trial design and enrollment, the use of surrogate endpoints, discerning meaningful clinical benefit, and much more. We were heartened to learn that the definition of ‘disease expert’ was broadened to include individuals living with the named disease; in ALS you have an illustrative example of where such expert voices are mandatory in all pertinent discussions. The drug development and approval process must not be a one-size-fits-all proposition.
While it is critically important that the FDA listen to the views of people with ALS, it is particularly important now. We cannot recall a more promising time in the history of ALS drug development than the one in which we are in today. As the Agency knows, multiple promising treatments currently are in Phase 2 or 3 clinical trials. Until now, the ALS community has shared our perspective and concerns with FDA and with Congress largely based on a hypothetical – we have shared the policies we believe are important if an ALS treatment progresses through the approval process. Today, the FDA needs to hear from people with ALS because ALS treatments are progressing towards approval. Within the next few years, it is possible that multiple therapies could be available to people with ALS for the first time since the disease was identified more than 140 years ago. The time that FDA absolutely must hear from people with the disease is now.
The ALS Association and Muscular Dystrophy Association actively participated in the PDUFA stakeholder meetings with FDA and the PDUFA discussions on Capitol Hill that have led to this Patient-Focused Drug Development Initiative. We strongly supported the inclusion of provisions that required FDA to hold the October 10 and October 25 meetings with patients and patient organization and also provided patients with many other opportunities to voice their concerns to the Agency directly. We believe that these opportunities for engagement should not be limited to public meetings like the one held on October 25. Rather, FDA must provide for patient input in a range of different ways, whether through broad public meetings, smaller, more focused face-to- face meetings or through electronic communications and requests for comments and web-based patient surveys.
It is equally important that the FDA actively solicit and provide opportunities for patient input throughout the regulatory review process, not simply at one point in time. Patient input and that of the organizations that represent them is important not simply at the beginning of the review process or at the end. This input is important throughout the process and will help ensure FDA is fully aware of, and takes into account, the patient’s perspective at every stage of the drug development and approval process.
It is clear that ALS meets the criteria FDA is using to determine the list of the 20 disease areas that will be subjects of the Patient-Focused Drug Development meetings:
As the primary organizations representing the ALS community, we have sought every opportunity to be engaged in this process with you and feel very strongly that ALS is a disease that meets the criteria that you have outlined. The ALS Association and Muscular Dystrophy Association understand that the Agency is committed to providing multiple points of engagement for patients and patient organizations over the next five years and that this Patient-Focused Drug Development Initiative is just one of many ways FDA intends to seek patient input. However, we are concerned with the way in which this initiative was communicated, the way in which FDA communicated the preliminary list of nominated disease areas, and the fact that FDA did not provide the context for how this initiative fits into the broader goals of PDUFA and FDASIA and FDA’s other plans to seek and facilitate patient input.
Again, we applaud FDA’s efforts to promote more fluid communication between the Agency and patient communities as you work to enhance the benefit-risk context for decision making as it relates to the reauthorization of PDUFA, however we have some concerns based on the outpouring of feedback that we have received from members of our ALS community following the posting of the preliminary list of disease areas:
We understand these concerns and believe that, before finalizing the list of disease areas, the FDA should provide the patient community with a more clear picture of what it plans to accomplish from these disease specific meetings and how the meetings will benefit other diseases not included on the list. The Agency also should develop and communicate a process that ensures people with ALS have opportunities to actively engage the FDA and provide direct input into the drug development and approval process whether that input is provided through this specific initiative or another mechanism.
Regardless of whether or not ALS ultimately is included as one of the 20 disease areas, The ALS Association and Muscular Dystrophy Association respectfully request that the Agency convene a public meeting at which ALS stakeholders, including people with ALS, have an opportunity to provide their perspective on all aspects of the drug development and approval process. We urge the Agency to make this opportunity available to people with ALS as soon as possible, for people living with ALS today do not have time to wait.
The ALS Association and Muscular Dystrophy Association appreciate the opportunity to participate in this important initiative and we look forward to working with the Agency to ensure 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 our patient community. As you are aware, people with ALS and those who have been touched by the disease have submitted a considerable number of comments to the docket for this initiative. Their message is clear: ALS patients, and those who represent them, want to be heard. The ALS Association and Muscular Dystrophy Association support them.
The ALS Association
Public Policy Department
1275 K Street, NW, Suite 1050
Washington, DC 20005
Muscular Dystrophy Association