First-in-Class HEOR Challenges and Regulatory Framework Shifts

Episode 20 — 03 June 2026 Multiple first-in-class therapies create new HEOR value demonstration challenges while industry increasingly funds academic research to support policy positions. EMA's annual report reveals optimization of medicines assessments and HTA regulation coordination progress. Hosted by Marcus and Sara. Access Brief — Daily briefings on HEOR, HTA strategy and the evidence access landscape. Full transcript and sources at outcomes-analytica.no/podcast

Transcript

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Welcome to Access Brief, the daily AI podcast on Hior, HTA, and market access. I'm Marcus with Sarah. Today, Backstrawstat's First in Class Hior Challenge, Revolution Medicine's CRAS Breakthrough, and Genentech's academic research strategy. Let's get into it. Starting with Backstrawstat, FDA approved it May 18th as the first aldosterone synthase inhibitor for hypertension. That's a novel mechanism in a crowded therapeutic space with established cheap generics. The her challenge here is demonstrating value when you're not just another ACE inhibitor. If this truly targets resistant hypertension effectively, the comparator set narrows significantly. Cost per controlled patient becomes the key metric, not cost per prescription. But that's assuming resistant hypertension is the real-world positioning. The approval doesn't restrict it there. If this gets prescribed broadly as another anti-hypertensive option, you're back to competing on acquisition cost against decades old generics. Fair point. The evidence strategy has to be laser focused on the resistant population where existing therapies fail. Otherwise, the HEOR story falls apart completely. Exactly. And payers won't differentiate mechanisms, they'll differentiate outcomes. If you can't show superior blood pressure control or reduced cardiovascular events in the population that matters, the novel mechanism becomes expensive novelty. Moving to Revolution Medicines, they presented what they're calling stunning results for Diraxinracib in pancreatic cancer at ASCO. KRAS has been the holy grail target, previously undruggable. Pancreatic cancer is where he or frameworks get stress tested. Median survival measured in months, not years. Any meaningful extension justifies significant cost under most value frameworks. True, but stunning is marketing language. We need to see the actual survival curves and response rates. Pancreatic cancer has seen promising early results before that didn't translate to practice changing outcomes. That's the perpetual optimism bias in oncology. But if this truly cracks KRS and they're already starting early access programs, the health economic implications extend far beyond pancreatic cancer. CRAS mutations drive multiple tumor types. Which raises the platform value question. Do you price for pancreatic cancer where patients are desperate, or price for the broader Karas mutant oncology market where you'll face more competition? The strategy has to be sequential. Establish value in the most desperate indication first, then expand. But the HEOR models need to anticipate that expansion from day one. Now this Genentech story is fascinating. They're offering up to $125,000 grants to academics for research on pricing policy consequences and RD risks. That's he or evidence generation with a very specific agenda. It's not subtle. They want academic credibility for positions that support their commercial interests. The question is whether the research questions are legitimate, regardless of who's funding them. But there's an inherent bias problem. When you're soliciting research to blunt pharma reforms, you're not funding neutral inquiry, you're funding advocacy with academic window dressing. I disagree. The research questions around pricing policy unintended consequences are valid. If academics can maintain a methodological rigor despite funding source, we get useful evidence. The funding source doesn't invalidate good research. That's naive, Sarah. The solicitation itself shapes what gets studied and how it gets framed. When you're explicitly seeking research to support policy positions, you're corrupting the evidence base. So who should fund research on pharmaceutical policy impacts? Government agencies with their own political biases? The questions need answering regardless of funding source politics. Finally, EMA's annual report from March shows their focus on optimizing medicines assessments and implementing the HTA regulation. The coordination between regulatory approval and health technology assessment is tightening. That integration is long overdue. The artificial separation between safety efficacy assessment and value assessment creates inefficiencies and delays patient access. But it also creates new risks. When regulatory and HTA processes become too aligned, you lose the independence that makes each valuable. Oh, HTA bodies need freedom to reach different conclusions than regulators. The goal isn't identical conclusions, it's coordinated processes. Better information sharing, aligned timelines, reduced duplication. That benefits everyone. As long as it doesn't become regulatory capture where HTA decisions get predetermined by EMA positions. The market access landscape keeps evolving. First-in-class approvals like BackstrawStat test our value frameworks. Industry funded advocacy research blurs evidence boundaries. And regulatory HTA integration changes how we think about evidence strategy from the beginning. The old sequential model is dead. Everything needs to be coordinated from phase one forward.no.