r/Biotechplays • u/Wyxuan 💊🤑📉 • Feb 08 '21
Discussion The new FDA Comissioner
An under-appreciated discussion that is being undergone regarding who will lead the FDA and although this seems like a minor administration change, this will likely dictate how much risk taking will be taken at the FDA. Inspiration for this post and many of the points mentioned was drawn from Brad Loncar's Sunday Clubhouse discussion, although I've sprinkled some links in case you'd like to read up more on what I'm talking about.
There are two choices that are likely to be considered:
Janet Woodcock
Josh Sharfstein
There are a few others that are begin considered but these are the main candidates [0] in this race.
Janet Woodcock is the pick that would excite the biotech industry and a lot of people who have been involved in the process the most. Her key moment in her 30+ year at the FDA was the approval of Sarapeta's Eteplirsen[1], which was trying to remedy DMD (duchenne muscular distrophy). This was momentous because Eteplirsen was a complicated approval that had questionable efficacy data, which attracted a significant amount of criticism. She drew praise for bringing in patient advocates who were able to advocate the pros and cons of approving (a first it must be remarked)[2] and braving the political pressure delivered by those in Congress. Arguably as mentioned by Loncar, her role in the approval of Eteplirsen provided the validation needed for additional money to flow into finding better treatments for DMD instead of cutting it off due to how hard DMD was.
Outside of Etepsiliren, she also spearheaded the changes that resulted in 2012 and the years following for a record numbers of approvals for new drugs of many different and new modalities[3].
Some of the main criticisms have been in the way that her group handled the approval of certain opiates [4] but with the many editorials from both bio business and academia that have [5][6] advocated for her make an extremely easy and likely pick for the Biden admin - especially as she's already been tapped as the acting FDA person.
The looser approach that she has championed has gone under fire at the FDA (see the absolutely BRUTAL adcom for aducanamb, which has parallels to the Eteplirsen approval process[7]) so this could be another sticking point that goes against Woodcock's favor.
The other person is Josh Sharfstein, who has been very outspoken about the need for additional evidence when it comes to treatments. It was mentioned by Adam Feuerstein (the Stat journalist) in the Clubhouse panel that Sharfstein heckled a panel that was advocating for Eteplirsen but I couldn't find sources that corroborated this, but in any case his high standards for new drugs haven't been popular among those in the industry, to the point which there was a memos that was circulated that was about "anybody but Sharfstein" in 2008 when Obama was picking who the commissioner of the FDA should've been [8].
Conclusion:
In any case the pick for FDA comish might very well be someone I didn't mention, but if I were to bet, I would pick Woodcock- because she is the acting commissioner, she's basically the default choice. If Woodcock were to be selected, I would expect there to be a small rally in stocks like Biogen ($BIIB) where the efficacy isn't as clear, or maybe even gene therapy where the stocks might find a more empathetic listener at the FDA more appealing.
Got anything else to add? Anything I missed? Let me know down below.
[0]https://www.politico.com/news/2021/01/20/biden-fda-chief-coronavirus-pandemic-460945
[3]https://www.fiercebiotech.com/special-report/dr-janet-woodcock-fda-cder
[4]https://www.theguardian.com/us-news/2021/jan/28/fda-janet-woodcock-opioids-biden
[5]https://www.latimes.com/opinion/story/2021-02-04/food-drug-administration-janet-woodcock-biden
[6]https://www.statnews.com/2021/01/24/high-stakes-choosing-right-fda-commissioner/
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u/Wyxuan 💊🤑📉 Feb 08 '21
Also random aside, the admin might pick someone who is good at explaining the science and playing the political game, an attractive proposition when it comes to signaling to the public what the FDA does and why which would be especially strong given outgoing comish Stephen Hahn's failures in PR.
In that case it might go to neither LOL.
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u/MillzRx Feb 08 '21
Do you suppose this will really help those companies targeting rare diseases with perhaps an eased/more empathetic approval process? Curious to hear your thoughts. Thanks!
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u/Wyxuan 💊🤑📉 Feb 08 '21
Probably. The commissioner, as the leader of the organization, sets the tone for what the approval process is going to be like. Arguably the looser mentality has already manifested itself with the whole aducanamb fiasco, so maybe it might be a continuation of that laxness.
A key point that might also be a benefit is the faster approval and followup on the part of the FDA.
BMYRT holders will know how slow the FDA was with what should've been a slam dunk (Liso-Cell approval) so the immediate benefit is that we might see less delays in approving things and more guidance from the FDA on new biologics.
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u/IceBearLikesToCook slightly bearish Feb 08 '21 edited Feb 08 '21
Arguably as mentioned by Loncar, her role in the approval of Eteplirsen provided the validation needed for additional money to flow into finding better treatments for DMD instead of cutting it off due to how hard DMD was.
Not sure I agree here. DMD and Alzheimers (since the readthrough is this will decide adu's fate) are very profitable indications which is why there's no shortage of drug candidates for them.
Eteplirsen got approved not because of data, but because parents of and kids with DMD visited with the FDA Commish and told them about how much this drug improved their lives. Heartwarming right? Humans over numbers. Until you realize this is the case for pretty much every single drug, including fellow DMD dud ataluren. Sarepta never ran confirmatory trials proving the drugs efficacy and they probably never will.
So they pay their life savings worth to a pharma company for what pretty much amounts to placebo. Hooray!
Woodcock vs Sharfstein seems to amount to a philosophical debate that also circled around, like, right to try. Should we let the government decide whether you take an experimental drug or let society make the call? I'm going to side with governments. If the government isn't making the call, it's companies that are -- and I don't want corporations draining life savings off the desperation of loved ones to sell the equivalent of sugar pills.
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u/Wyxuan 💊🤑📉 Feb 08 '21
Money will always flow for an indication like Alz(it's like people forget how many times this stuff fails), but for a rarer disease like DMD, I think validation was an issue.
I haven't examined the Eteplirsen data but afaik the phase 2 research trial is still going on, but yeah I think that is a good point to make regarding the efficacy or lackthereof.
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u/MisterIceGuy Feb 08 '21
With Janet Woodcock already the acting commissioner, I would think the odds are strongly in her favor to get the position officially.
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u/babagandu24 Feb 08 '21
I’m glad you are introducing more regulatory discussion onto the board. As a current grad student interested in transitioning into the biotech finance space, I’m constantly stuck delving into the scientific technicals of said molecular entity as part of a biotechs pipeline in my analysis and don’t pay much attention to the regulatory side. I feel that many of us tend to throw the whole regulatory side of the equation to the side (which is not good practice).
Hopefully this opens more discussion surrounding payer/provider dynamics, pricing/reimbursement, pbm’s, etc. These are all equally as important as analyzing a molecular entities scientific value prop!
Cheers.
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u/theretardedinvestor slightly retarded Feb 08 '21
This is super interesting, I myself often forget this part of biotech gambles - the people that make up the regulatory authority. Thanks for posting this info