This guest post is from Shelly Hoover, EdD, a member of the Patient & Caregiver Advisory Committee, and does not necessarily reflect the opinions of the Association staff, its Board of Trustees, or its chapters.

By guest blogger Shelly Hoover

Patient voices matter, yet the collective ALS patient community has been too patient and too quiet for too long.

The ALS Association led an unprecedented effort to develop an FDA Guidance Document for Industry. Here’s a quote from the introduction:

“In February of 2015, The ALS Association launched this effort to develop the first-ever community-driven drug development guidance for ALS with funding from the ALS Ice Bucket Challenge, bringing together over 100 participants including people living with ALS, caregivers, researchers, clinicians, and industry experts from across the world to contribute their expertise and experience. In parallel with this effort, a committee was formed to update the ALS clinical trial guidelines that were developed more than 15 years ago. The guidance and guidelines have different audiences and different goals, yet are meant to be consistent. This guidance is intended to represent the Agency’s interpretation of, or policy on, evaluating new ALS therapies for approval in order to assist industry in navigating the regulatory process. The clinical trial guidelines incorporate stakeholder views across all phases of drug development, from preclinical to market approval, and serve as “best practices” for clinical trial design and are used by researchers and industry to provide structure and direction for the design and conduct of clinical trials in ALS. The goal of clinical trial guidelines is to lead to more effective and efficient trials, but they do not directly impact the FDA regulatory process.”

This guidance and the subsequent FDA document ALS: Developing Drugs for Treatment; Draft Guidance for Industry, are conservative and uphold the FDA’s gold standards for drug trial design and approval. That is great news for patients who will be diagnosed decades from now. Patients who are living with ALS today will not benefit in any significant way. As a person living with ALS, I find this conservative approach unacceptable.

I applaud The ALS Association ’s efforts. However, as a member of the patient advisory committee, I feel the patient voice was drowned out by louder voices in the development of the FDA Guidance. Read the last sentence in the introductory paragraph (bold emphasis mine). Are minor tweaks in trial design going to get potential treatments to patients now? And, why aren’t we pushing for changes to the archaic FDA regulatory process?

Here are two changes that the patient community can demand today:

1.  Use historical controls instead of placebos until we have a widely effective treatment.
2. Treatments in Phase 2 must be made available all patients. This will require changes to the FDA Expanded Access Program that currently does not incentivise nor protect drug manufacturers

You can make a difference by forming your own opinion and leaving a public comment here. Please do it today.

Let me be perfectly clear -- this is not an ALS Association problem. The Association has multiple stakeholders, and the patient community played nice and our quiet voice got buried in the last pages of the The ALS Association Guidance Document (see Appendix C, of ALSA’s document). This is not an FDA problem. The FDA is upholding their gold standards of drug safety and approval. Why would they change?

This problem lies squarely on the shoulders of the patient community. We have the responsibility to speak out, speak loudly, and speak now. We need 1,000 public comments to get the FDA’s attention (101 comments have been submitted at the time of this writing). Then,  we must demand changes and do whatever it takes to save our own lives. If we don’t act now, we will die waiting for someone else to act for us.

About the Author

Shelly Hoover, EdD was diagnosed with ALS five years ago at age 47. She is an ALS Advocate, retired public school administrator, and US Navy Veteran. Shelly lives with her husband, Steve, in Northern California.