New Research Underscores the Positive Impact of Accelerated Approval

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I’m always down for a good, wonky donnybrook, and we’re getting a good one now over accelerated approval. 

A quick review: first, a team from Harvard PORTAL published a piece in JAMA that suggested that “most” of the cancer drugs cleared under the FDA’s accelerated approval pathway never showed benefit. 

Then there were a bunch of overheated headlines (“Fast-tracked cancer drugs often fail to prove benefit,” etc.) based on that research.

THEN there was a spirited takedown of the JAMA research by Steve Usdin over at Biocentury. 

And now we have more research, from a team of researchers from Evidera and J&J, that offers a competing take on the impact of accelerated approval. The group, writing in Journal of the National Comprehensive Cancer Network, looked at survival data generated on accelerated approval drugs going back to 2006 and found that more than 260,000 life-years had been gained by earlier access to the medicines. 

That’s certainly a different narrative and a different way of looking at the public health impact of the program. It suggests that accelerated approval is doing what it is supposed to: giving early access to drugs that can change lives.

No, drugs green-lit under AA don’t always work. Yes, the FDA needs to continue to press for timely completion of confirmatory trials (and yes, ineffective drugs/indications need to be yanked). 

But there is a lot of baby in there with the bathwater, and the JNCCN paper does a great job of making that clear.

I’m not entirely sure what to do with this sprawling, thoughtful examination of the access, pricing, and financing issues around gene therapies from the Institute for Clinical and Economic Review and the Tufts NEWDIGS group. 

On the one hand, it is an excellent primer on many of the issues -- and many of the proposed solutions -- around gene therapy, and it probably ought to be required reading for anyone seeking to get up to speed on what’s going on. 

On the other hand, I fear that it’s too quick to suggest changes in pricing as the “fixes” to the problem. The very first “solution” that the paper discusses is “Determining a Fair Price,” and of the three strategies discussed in the context of “fair price,” two of them involve shifting thinking around gene therapies to drive lower prices. (In one case, to account for uncertainty, especially around durability, and, in the other, to better share savings across the health care system.)

I don’t want to suggest that there isn’t a place in the discussion for price. I’m just not sure that price is driving the issues around gene therapy right now.

On the other, other hand, the ICER/NEWDIGS project was done by smart people who have thought hard about this for a long time, and I certainly don’t have some well-developed counter-theory about how to fix gene therapy. (That’s Courtney Rice’s job!) 

And for all my reservations about some of the concepts here (e.g. “shared savings”), I have to give credit to ICER/NEWDIGS for trying to elevate and publicize this discussion. I only hope that this is the start of the conversation, not the end of it.

• STAT covered what Donald Trump might do around health care in a second term. It’s one of those stories that has to be leavened with a lot of caveats because Trump is, um, somewhat mercurial in his policy thinking. But there’s some consensus that he’s going to lean into international reference pricing if he gets the chance. My general feeling is that “most-favored nation” is a tactical tarpit of a policy, and the STAT piece added one more twist: any executive action taken under the IRA would be made more difficult by the IRA’s ban on QALY-like objects … which underpins a lot of the European pricing decisions that reference pricing would rely upon. 

• For those of you who are deep into the constitutional arguments around the IRA, I have good news: a Supreme Court decision earlier this month suggests that SCOTUS may have a more expansive view of the “takings” clause, which would be good for the pharma companies challenging the law. For its part, the government is arguing that pharma’s thinking is still way outside of what the law considers a taking and that the new decision is way too narrow to impact the IRA suits. Bloomberg Law has a good summary.

• This is my occasional reminder that we’re waiting on a LOT of IRA-case opinions. Briefs in the Merck case have been sitting with the judge for five months, and arguments are complete in the BMS, J&J, Novartis, and Novo cases (all of which are being heard by the same judge). So even though the cases feel out of sight, out of mind, we’ll likely get a series of opinions here in the not-too-distant future.

Cover image via Flickr user MIKI Yoshihito.