Changing Tides: FDA and I AM ALS Meet to Discuss FDA GuidanceOctober 4, 2019
The big news on Monday, September 23, 2019 was that the FDA ALS Guidance document at last was finalized! With great anticipation, we read and re-read it and were happy to find that so much mapped to the recommendations we provided on August 28, 2019. The recommendation document was a compilation of so so many ALS advocate voices since 2013. We ensured that this document reflected the time and expertise of so many before us and so many who are no longer with us.
As we read the final Guidance document we finally felt heard. See the timeline to understand why this means so much to those who have been in this fight for a long, long time. And here is the exciting part! This is actually a usable document for ALS drug sponsors, Pharma and biotech firms in their upcoming trial design. Even more inspiring was the press statement encouraging the drug manufacturers to be humane, be efficient and…get this…be more innovative. As noted in the press statement, “The FDA is open to considering alternative approaches to meeting our requirements for approval.” Refreshing. Keep in mind that we already have the FDA’s supportive stance for Expanded Access.
With these documents in our back pocket, the onus is ours and we are ready to roll. It is now time to educate those companies who are starting clinical trials, as to what the FDA will allow, and equally important what people with ALS and future trial participants (aka test pilots) will allow. Truly groundbreaking times - we are already working with five companies who have approached us to partner with them in clinical trial design. This is the epitome of a win/win as drug companies will fill their trials quickly and people with ALS will reap the benefits of drugs/treatments which have arrived to market faster. Say it with us – WIN/WIN.
Okay on to the meeting…to be entirely honest, it was almost anti-climactic after years of build-up, as the hard work had been done. What we had planned on talking about became a moot point. We had received what we had asked for. The most exciting part was when FDA leaders entered the room and there were quite a few. We looked up to see Dr. Ned Sharpless sit right across from us. Reread that - Dr. Ned Sharpless, Commissioner of the FDA. He spoke compassionately about ALS and how his cousin was afflicted with the brutal disease. He said that he had been contacted by Congressional leaders who wanted to help move us forward, specifically mentioning Senator Johnson who was bolstering our efforts. Dr. Sharpless assured us that we had his team’s attention. He thanked us for our advocacy. The I AM ALS team was touched by his genuine show of support.
Our slide deck of our September FDA meeting is here. We began with a review of the asks from our February 11, 2019 meeting. We set forth our updated asks, before diving into the scientific details. We thanked them for finalizing the document that reflected the needs of people with ALS for efficient, logical and humane clinical trials. We pointed out the importance that this guidance is a living, breathing document, not an encyclopedia to be set on the shelves for the spiders to do their work. We must ensure that the document stays current with changing science and relevant to upcoming discoveries.
We then dove into three areas that we believed should be included in clinical trials to reduce biostatistical noise: 1.) Interim analysis 2.) Heterogeneity of ALS - although somewhat addressed in document 3.) Importance of collection of data in PRO-ACT. The importance of this conversation was to make sure that if we tell the biotech firms and Pharma that these are acceptable, the FDA must also believe it is. The good news was that Dr. Billy Dunn, the Director of the Division of Neurology Products in CDER's Office of New Drugs, engaged with a member of our clinical trials committee, Bob and said he was “right on track.” He also stated that while none of these were novel ideas, the complexity of ALS makes them harder, but they had been done before. This was the nod we needed to proceed well. Dr. Dunn also said that the heterogeneity of our disease is the bane of clinical trials, which we all agree with, but we must continue to find a better way.
Our newest ask was this from a member of our community advisory council, Deb - when the FDA leaders meet with the drug sponsors in the IND meetings, ask this simple question “which one of you is the patient?” In the unfortunate event that there is no patient at the table, slightly raise an eyebrow so it becomes known that this is the new norm. It was in fact, the FDA leaders who encouraged us to find a seat at the table in these meetings. It happens in other disease states. It absolutely should happen in ours. And puh-lease let’s not discuss any nonsense about confidentiality or privacy. We are well-versed in the importance of CDAs and NDAs –problem solved. The best part about this is that everyone wins (again) as the drug sponsors are coming to the meeting – well-armed – with a patient (a stamp of humanity approval if you will). The unspoken words are “as a patient of this disease, I would join this trial.” Off to a great start with the well-balanced triangle of drug sponsor – scientists/neurologists – and patients. The perfect mix of funding, good science and humanity. The FDA leaders cannot mandate this, but believe it to be a good formula as well. It’s certainly a step in the right direction.
Dr. Dunn commented that the actions of the guidance document being finalized was not based on any one person or group and we agree that it was the collection of all the groups and advocates before and those currently engaged who accomplished this important feat. A huge thank you to all of you who brought us to this important day.
We disbanded with a request for a follow-up meeting to hold everybody accountable because the bottom line is this – the finalized guidance is just a document if clinical trial design doesn’t change to reflect the new rules. No one would be excited about the new guidance unless the new actions accompany that. It is the implementation that is critical, and we have already had follow-up calls with FDA to make sure this meeting is just the beginning.
As you are aware, this disease requires incredible bravery from those afflicted by it and those who love them. In turn, significant changes for this brutal disease will require brave and compassionate leadership. It will require brave and logical legislation. It will require brave and innovative next steps. This deters us in no way. We have and will continue to march boldly towards our brave new world.
Note: At I AM ALS, we appreciate everyone’s views, references and perspectives and value that our diversity only helps us be stronger in our movement and faster at changing the ALS landscape. Truly we believe we are better together.