The text below is a shared statement about the Collaboration for a Cure event for use by all the associations in attendance. The International Alliance of ALS/MND Associations is honored to be part of this community! We look forward to all that we can achieve together.
Thanks in part to last summer’s ALS Ice Bucket Challenge, the first Collaboration for a Cure took place on April 17, 2015, in Arlington, Virginia. Representatives from 18 organizations who are fighting amyotrophic lateral sclerosis (ALS) gathered to identify key areas where collaboration among their organizations could help drive forward collective efforts to help people living with this disease. One such attendee, Dr. Kevin Horton, Chief of Surveillance and Registries within the Division of Health Studies, presented information about the National ALS Registry.
The invitation to this first meeting was to individuals at many of the leading ALS organizations. Patients who lead some of those organizations were invited. Although, none were able to attend, they were well represented by their chosen delegates. The group is committed to having people living with ALS and caregivers of people with ALS participate in future meetings and task groups. The following organizations were represented: ALS Canada, ALS ETF, ALS Hope Foundation, ALS Therapy Alliance, ALS Therapy Development Institute (ALS TDI), ALS Worldwide, Answer ALS, International Alliance of ALS/MND Associations, Iron Horse Foundation, Kevin Turner Foundation, Les Turner ALS Foundation, Muscular Dystrophy Association (MDA), Prize4Life, Project A.L.S., Target ALS, Team Gleason, The ALS Association, and The Robert Packard Center for ALS Research at Johns Hopkins.
The day began with a welcome from Barbara Newhouse, President and CEO of The ALS Association. While The ALS Association brought together this first meeting, it was acknowledged that future meetings will be convened and driven forward by the community of ALS organizations. Newhouse introduced the moderator for the day, Paul De Morgan, who has 16 years of experience in facilitating and mediating multi-party, consensus-oriented policy dialogues and site-specific dispute resolution processes.
After a full day of discussion (described below), there was near unanimous commitment to continue to support the collaborative into the future. While it was agreed to continue to discuss key issues pertaining to ALS as a group, it was acknowledged that the power of the meeting was in the diverse opinions and levels of experience. It was noted throughout the meeting that the focus must remain on the patient and finding solutions for their urgent needs. It was also unanimously agreed that despite the many diverse opinions, the participants were eager to work together if it meant immediate action.
Many areas need to be addressed, but for this meeting, three areas of focus were identified in advance to be topics of discussion. The first topic involved gaining consensus on the prevalence and incidence of ALS. The goal was to share detailed information of the known and unknown regarding current data and gain alignment in messaging regarding incidence vs prevalence. Individuals and organizations have used a range of estimates when describing the number of people living with ALS in the United States, from as few as 12,000 to as many as 40,000 or more. Representatives from the Agency for Toxic Substances and Disease Registry (ASTDR), including Kevin Horton, DrPH, MSPH, CPH, Chief of Surveillance and Registries within the Division of Health Studies, and Paul P. Mehta, M.D., Medical Epidemiologist, presented information on the National ALS Registry and its efforts to identify people living with the disease to better understand incidences and prevalence.
After a robust discussion among members of the group, it was determined that the data is not sufficient to support currently reported numbers of people living or diagnosed with ALS. The registry’s first report is based on one year’s worth of data. Although it is consistent with published literature, the organizations agreed that the first year data from the registry may have under-reported prevalence. Therefore, the organizations agreed that it was critical to work together to urgently explore options to enhance the registry. A task force made up of designees from the ALS organizations in attendance will join together to help accomplish that goal. Creating a strategy to foster enrollment through clinical experiences and also in patient and caregiver outreach are two areas agreed upon that need immediate improvement. It was ultimately agreed that all people living with ALS need to be counted and their medical voices heard through their critical data.
The second discussion of the day focused on the formidable challenges that exist to bring a new therapy to market and the development of a guidance document to help companies and researchers involved in ALS drug development navigate the regulatory process and speed drug development. Given that the U.S. Food and Drug Administration (FDA) does not have the resources to develop guidance documents for each disease, this group is unified in a commitment to assure the guidance document is not only produced but also reflects the recent community driven efforts. Resources from the ALS Ice Bucket Challenge are dedicated to that assurance. The initiative will involve participation from the entire ALS community: people living with ALS, ALS organizations, clinicians, researchers and partners in the industry. It will be governed by a steering committee comprised of content area experts who will chair individual working groups focused on specific chapters of the guidance document including, biomarkers, benefit-risk, clinical trials and outcome measures, natural history and diagnosis. The steering committee will also be advised by a patient and caregiver advisory committee to ensure patient input throughout the guidance development process. This will be the first patient-focused guidance for ALS product development that would be submitted to the FDA. And, it will be a consensus-based work product that incorporates stakeholder views in areas such as trial design, biomarkers, surrogate endpoints, patient-reported outcomes, benefit-risk and others.
The group fervently agreed to coordinate efforts to create a needed momentum for this important guidance. As part of this initiative, a communications plan will be included in order to provide the ALS community with timely input, updates and milestones.
Finally, expanded access was a topic of discussion and how ALS organizations can better communicate information about treatments in development. Because there is currently only one approved drug to treat ALS and given the devastating nature of the disease, there are often calls to support widespread access to therapies that have shown some promise in early stages of development. The FDA has a number of existing authorities that are designed to expedite the development and approval of new treatments and also provide access to those treatments, including outside of a clinical trial and prior to FDA approval. Those authorities include: expanded access; accelerated approval; fast track; priority review; breakthrough therapy designation; and orphan drug designation.
The conversation centered on educating attendees about these authorities as well as other related issues, such as “Right to Try” laws that have been introduced and enacted in states across the country. “Right to Try” laws seek to provide access to investigational therapies outside of FDA governed expanded access programs. The session also focused on how ALS organizations can communicate information about promising investigational treatments to people with ALS and how organizations can respond to calls for widespread access to those potential treatments.
The group recognized that ALS organizations can do more to explain the differences between the various FDA authorities mentioned above. Furthermore, the group acknowledged that people living with ALS have unique perspectives as it relates to the above options and should help guide organizations when they choose how to communicate opinions of a new therapy or treatment concept. It was recommended that a task force convene to discuss expanded access issues.
Those in attendance agreed that there will be future meetings for the Collaboration for a Cure and will immediately begin to work together to follow up on the various action items that were identified by the group.
One final note: the group was shown a video and letter from Pete Frates and Pat Quinn, co-founders of the ALS Ice Bucket Challenge, asking all the organizations to work together and follow their lead in order to make the event a success again in 2015 and the years to follow. ALS Association Chief of Staff Brian Frederick outlined for the group a collaborative Ice Bucket strategy that Pete and Pat are spearheading, which will be for the benefit of the entire ALS community. The message was well received and all agreed we are a greater force when working together.