Petition update

HopeNOWforALS.org next meeting with FDA -- we need your help

Jehad Majed
Dearborn Heights, MI

Jun 12, 2015 — Dear Petitioners,

The FDA has agreed to engage with us on behalf of the PALS community to discuss a path toward getting ALS treatments/drugs into the Accelerated Approval Program. This will require a series of discussions covering a number of discrete topics (surrogate endpoints and relevance of biomarker data, design of a validation process to determine accelerated approval efficacy where evidence of efficacy and safety coming out of a Phase 2 trial exists, etc.) with the goal of defining a method for validating efficacy for drugs/treatments where safety has already been demonstrated. We look forward to engaging with the FDA and taking concrete steps in getting this done. We recognize the urgency that this process has for the existing generation of PALS and will always make that our foremost consideration in moving forward in this process. Our next meeting with FDA is scheduled for June 17th and according to the list of FDA attendees provided by the agency in their invitation, the meeting will include most of the key agency personnel needed to make material progress toward establishing an expedited development and approval pathway for new ALS medicines. According to FDA, the following agency staff will be attending the meeting:

Dr. Janet Woodcock, Director of the Center for Drug Evaluation and Research (CDER)
Dr. Richard Moscicki, Deputy CDER Director for Science Operations
Dr. John Jenkins, Director of the Office of New Drugs, CDER
Dr. Billy Dunn, Director of the Division of Neurology Products, CDER
Dr. Ronald Farkas, Division of Neurology Drug Products, CDER
Dr. Nicholas Kozauer, Division of Neurology Drug Products, CDER

The objectives of HopeNOWforALS are described below:

• Clearly communicate our sense of urgency regarding the need for rapid development of the expedited pathway,
• Establish a framework for achieving Objective No. 1 with weekly meetings over the next 4 to 6 weeks,
• Reiterate the ALS patient community position on how FDA should evaluate the potential for drug-related risks versus the potential for benefit in the context of the risks posed by the disease,
• Hear FDA’s thoughts and proposals for an expedited pathway and express our own,
• Attempt to reach agreement on the major elements of an expedited development and approval pathway for new ALS medicines, including evidentiary requirements that would be considered adequate to support agency acceptance and review of an application for Accelerated Approval before the end of this year.
• Set action items for FDA and HopeNOWforALS,
• Schedule the next meeting.

Our objectives for this initial meeting are ambitious, and may exceed the FDA’s customary pace of discussions regarding drug development programs; consequently, we are communicating our expectations for the meeting to FDA in advance in hopes that they will come prepared to negotiate a set of initial agreements in good faith, and with a shared sense of urgency. We will know more about our goals for the next meeting after our upcoming meeting on the 17th.

Because the details of an expedited drug development and approval pathway must also be agreed to by the drug sponsor(s) who will navigate the pathway, it is our intent to bring sponsors who are interested in using an expedited pathway into the process quickly, potentially as early as the third meeting. We anticipate that this involvement will include Genervon in pursuit of an Accelerated Approval for GM604, and we are hopeful that others will elect to collaborate in this process as it moves forward.

We need your help leading up to our June 17th meeting. Please contact the FDA (see below) and your contacts on the Hill. Urge them to ensure that FDA enters this process with a shared sense of urgency and a willingness to fully consider and factor into its regulatory policy and decision-making, the perspectives of the ALS patient community regarding the dire need for a process that allows all PALS the opportunity to access promising new medicines for ALS at the earliest possible point in the learning process, and to participate in the completion of that learning process in a post approval environment, as intended by Congress in FDASIA, enacted in 2012. Please make the critically important point that FDA’s current, inflexible, years-long process for bringing new ALS drugs to the point of approval is failing to meet the dire unmet medical needs of PALS, and needs to be remade into a process where FDA views the patient community as full and equal partners in the making of medical progress against ALS.

In Food and Drug Administration Safety and Innovation Act (FDASIA) Congress expressed:

SENSE OF CONGRESS.—It is the sense of Congress that the Food and Drug Administration should apply the accelerated approval and fast track provisions set forth in section 506 of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 356), as amended by this section, to help expedite the development and availability to patients of treatments for serious or life threatening diseases or conditions while maintaining safety and effectiveness standards for such treatments.

Congress went on to describe the evidence that could support an Accelerated Approval:

‘‘(B) EVIDENCE.—The evidence to support that an endpoint is reasonably likely to predict clinical benefit under subparagraph (A) may include epidemiological, pathophysiological, therapeutic, pharmacologic, or other evidence developed using biomarkers, for example, or other scientific methods or tools.”

The intent of these provisions was to direct FDA to use its Accelerated Approval authority for disease like ALS, and to make exceedingly clear that the agency did not need to wait for evidence from multiple phases of long, expensive, randomized controlled clinical trials before granting Accelerated Approval.

We are standing on solid legislative ground with this effort, but to date, FDA has not shown a willingness to use its Accelerated Approval authority as intended by Congress for new drugs intended to treat neurological diseases like ALS. Please weigh in and strongly urge FDA to work with us in good faith, and in accordance with Congressional intent, to bring new medical options to ALS patients as rapidly and flexibly as possible.

We will provide an update after the meeting.

We're in this together,
Jehad Majed (jmajed@mac.com), Matthew Bellina, Nick Grillo (gm6nick@gmail.com), and the Torrino Family

Please email the Dr. Woodcock at the FDA:
janet.woodcock@fda.hhs.gov
and copy these people:
druginfo@fda.hhs.gov
richard.moscicki@fda.hhs.gov
william.dunn@fda.hhs.gov
stephen.ostroff@fda.hhs.gov
john.jenkins@fda.hhs.gov
eric.bastings@fda.hhs.gov
ronald.farkas@fda.hhs.gov
nicholas.kozauer@fda.hhs.gov
brad_grantz@toomey.senate.gov
Grace_Stuntz@help.senate.gov
josephine_eckert@murray.senate.gov

[WHAT TO SAY]

Start with any opening you choose but please include any of the following that you feel represents your voice:

It is my understanding that you will be meeting with members from HopeNOWforALS on June 17th to discuss a pathway forward for the evaluation of investigational treatments for ALS. I recognize that the FDA -- and particularly the Division of Neurology Products -- has been unwilling to implement the Accelerated Approval Program in the manner intended by Congress and so expressed in the FDASIA of 2012. I am also aware that the ALS patient community has not been given the chance to participate in discussions regarding risk vs. benefit and that those discussions have either been non-existent or differed to large "advocacy organizations" that I feel do not represent my best interests.
Over the past 6 months, the ALS patient community has been fighting for Accelerated Approval of Genervon's GM604 and I understand the FDA has denied Genervon's request to discuss this further. As a member of the grassroots movement working to get safe investigational treatments to market, I expect that you will work closely with HopeNOWforALS and make a change from the status quo -- and make that change very soon!

The FDA’s current, inflexible, years-long process for bringing new ALS drugs to market is failing to meet the needs of PALS and needs to be remade into a process where FDA views the patient community as full and equal partners.

Please do not patronize the patient community with information about the Expanded Access Program (Compassionate Use). This program is unable to meet the needs of and entire population of patients with no treatment options. Accelerated Approval was designed for this purpose.

By the FDA’s own admission, "It can take too long for an investigational treatment to definitively demonstrate a clinical benefit and then go through the process of securing FDA approval." On the website, the FDA claims that the 1992 Accelerated Approval Program is a way to speed new drugs to market that treat serious diseases and fill an unmet medical need. If this program isn’t meant to address ALS, then I want to know what are your definitions of “serious” and “unmet medical need"?

Know that I will continue fighting with my last ounce of energy and last breath to help the ALS Community see viable treatment options.