The FDA and your insurance ask different questions, and women’s health keeps falling into the gap between them.
I’m not a researcher but over the last few years I’ve learned so much from our women’s health research colleagues. One of the things we don’t talk enough about is why a lot of the innovative women’s health research stalls when coming to market. If you’re looking for a job in women’s health, it’s also important to understand why.
If I had to pick one word that explains why so many good products stall (and, honestly, why so many careers in this field stall too), it would be “endpoints.” Sounds like jargon, I know.
Let’s start from the beginning.
How a study gets built
Before a new drug is approved, the big pivotal Phase 3 trial usually enrolls several hundred to about 3,000 people (FDA). Before anyone is enrolled, the company decides what the trial will measure to say the treatment worked, and that measure is called the endpoint.
That choice affects the price. In one analysis of 59 drugs approved in 2015 and 2016, trials measuring a clinical outcome like a heart attack had a median cost of $65 million, versus $24 million for trials built on a stand-in (a “surrogate”) like a cholesterol level (coverage of the study). And it gets chosen long before anyone knows who will pay.
What the FDA is asking
The FDA needs “substantial evidence of effectiveness,” and the endpoint can be a direct measure of how a patient feels, functions, or survives, or a validated surrogate (FDA).
Take Veozah, approved in May 2023 for hot flashes and night sweats. The co-primary endpoints were the change in how often and how severe the symptoms were over 12 weeks compared with placebo. The women averaged 54, and no one 65 or older was included (FDA). That’s a real endpoint, and it answers the FDA’s question.
But your insurance asks a different question
But the endpoint that the FDA uses and the endpoint that your insurance company uses is often different. For example, Blue Cross Blue Shield plans have used five technology assessment criteria, and FDA approval is only the first one. The others include an improved net health outcome and a benefit that holds up outside the study (example policy), and Medicare wants to know whether the results apply to Medicare patients (CMS).
Back to Veozah. Before it was approved, the independent nonprofit ICER asked the coverage-style question, and its panel voted 11 to 1 that the evidence wasn’t adequate to show a net health benefit compared with no drug treatment (ICER). To be clear, I’m not saying anyone was denied (ICER isn’t an insurer). I’m saying those are two different questions.
“The FDA asks whether a treatment works. Payers ask whether it improves health enough to pay for. Very few trials are built to answer both.” |
Why I think the system is broken
You have two gatekeepers with two different tests, and nobody is responsible for the handoff between them, so a company builds the trial for the FDA and finds out what the payer wanted afterward.
Recently at a Milken workshop on menopause reimbursement, I saw a fascinating presentation. Hansilla Alaigh, WISE-R’s CEO, presented an analysis of menopause-related trials that found 2,588 of them, and only 28 were designed to meet both regulatory and reimbursement needs, with just three of those still recruiting. It’s one analysis, so I’d treat it as a signal rather than a census. And here’s the uncomfortable part: women were 51% of participants in FDA drug trials from 2015 to 2019 (FDA) so we aren’t missing from the studies. But being in the trial isn’t the same as the trial measuring what coverage will later ask for.
Why this matters if you want a job in this field
I get it, no job posting says “understands endpoints.” They say clinical operations, regulatory, market access, partnerships. But in every one of those jobs, someone eventually asks what the evidence has to show and for whom, and I think the person who can answer that ends up in the room.
I also think this is exactly where women’s health companies get stuck, which means it’s where they need people. The barriers WISE-R’s CEO named for investors were an unclear regulatory pathway and no guaranteed reimbursement, and one ask was to add payer-valued measures to trials that are already running. Somebody has to know to do that. (BTW, we recently hosted Tribunus Health, our Summit partner, on who they’re hiring for payer negotiation. Here’s the event.)
Whether you’re designing a study or interviewing for a role, ask yourself:
- Who will pay for this, and what do their written criteria say?
- Is the endpoint something a patient would call better, or just a proxy?
- Does the trial population look like the people who will actually be covered?
- Better than what? Payers compare against what they already cover.
To start small, read one FDA Drug Trials Snapshot and one payer medical policy for your area.
Final thought
I don’t think anyone set out to build it this way. The FDA and payers are each doing their job, and women’s health keeps falling into the space between them, which is also where the opportunity is. This is part of the gap we are closing with our work on reimbursement for 51&.
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