FDA RWD Guidance & CMS Star Ratings

MarcusWelcome to the Access Brief — your daily briefing on HEOR, HTA, and market access. I'm Marcus, and it's great to have you with us today.

SaraAnd I'm Sara. Always good to be here — and I'll say, today's topics are particularly timely given the shifts we're seeing in both US and EU policy.

MarcusSame here. We're looking at the FDA's new draft guidance on real-world data for accelerated approvals — what this means for evidence generation in the near term. Then CMS's proposed changes to Medicare Advantage Star Ratings — and how that might reshape provider incentives. And finally, NICE's threshold consultation for highly specialized tech.

SaraThat second one on Medicare Advantage is interesting to me — the impact on provider behavior could be significant, and I'm curious how that will interact with the IRA's drug pricing provisions.

MarcusExactly. Let's get into it.

MarcusThe FDA just released a draft guidance document outlining how real-world data can be used to support accelerated approvals. This is a significant shift because historically, accelerated approvals were based on surrogate endpoints from clinical trials. Now, the FDA is opening the door to using RWD from post-marketing studies to confirm clinical benefit.

SaraThat's one read — but I'd frame it slightly differently. The part that gives me pause is the quality and consistency of RWD. How do we ensure that the data is robust enough to support such a high-stakes decision as an accelerated approval?

MarcusThat's a fair point. The guidance does emphasize the need for fit-for-purpose data, but it doesn't specify exactly what that means in practice. We've seen in the past that RWD can be noisy and confounded by factors like selection bias.

SaraExactly, and from the payer side, we're concerned about the implications for cost-effectiveness. If a drug gets an accelerated approval based on RWD, but then the confirmatory trial fails, we've spent money on a drug that might not provide the expected value.

MarcusThat's true. But the FDA is also requiring post-marketing confirmatory trials, so there's a safety net. However, the timing of those trials is critical. If the confirmatory trial takes years, we have a drug on the market without full evidence.

SaraRight, and that raises questions about the budget impact. Payers might have to cover a drug without knowing its long-term value, which is a tough position.

MarcusI wonder if that's the full picture though. The guidance also encourages the use of RWD earlier in the development process, which could actually reduce the need for confirmatory trials if the evidence is strong from the start.

SaraThat's an interesting angle. But I still think the onus is on industry to generate high-quality RWD, and that's not trivial. It requires significant investment in data infrastructure.

MarcusAgreed. And it's not just about the data, but also about the analytical methods. The FDA is expecting sophisticated statistical approaches to handle the complexities of RWD.

SaraWhich brings us back to the payer perspective: how do we assess the quality of these analyses when we're reviewing submissions? We need clear standards.

MarcusThe guidance does mention that the FDA will be looking at the study design and analysis plan, but it doesn't provide a checklist. That leaves room for interpretation.

SaraAnd that's where HTA bodies will have to develop their own criteria. It's a bit of a wild west right now.

MarcusI think that's the reality. The field is evolving, and we're all learning as we go. But the potential is there to accelerate access to innovative treatments.

SaraAs long as we don't compromise on the evidence. Let's move to the next topic.

MarcusCMS has proposed changes to the Medicare Advantage Star Ratings system, which is used to assess the quality of plans. The changes include adding new measures related to social determinants of health and chronic care management. This could have a big impact on how plans operate and how they cover drugs.

SaraThat's right, and what strikes me about this is the potential for unintended consequences. If plans are incentivized to focus on these new measures, they might deprioritize other important areas, like timely access to high-cost drugs.

MarcusThat's a valid concern. The Star Ratings are a powerful tool because they drive plan participation and member enrollment. But adding new measures without adjusting the existing ones could create imbalance.

SaraExactly, and from a market access perspective, it's unclear how these changes will affect coverage decisions. Plans might be more reluctant to cover drugs that require intensive management if they're not getting credit for it.

MarcusI'd push back slightly on that. The new measures are meant to capture the whole patient, so actually, plans might be more incentivized to cover drugs that help manage chronic conditions effectively, because that could improve their Star Ratings.

SaraThat's one read — but I think payers would see it differently. They might view it as an additional burden without sufficient reward. We've seen in the past that when new quality measures are added, plans often struggle to meet them.

MarcusThat's fair. And the timing is tight. The changes are proposed for 2027, but plans need time to adjust. There's also the question of data collection. How will they capture social determinants of health in a standardized way?

SaraThat's a huge challenge. The data infrastructure in Medicare Advantage is already stretched, and this would require significant investment. It could lead to disparities in how plans are rated based on their ability to collect data.

MarcusThe CMS proposal does include a transition period, but it's still a lot to ask. And for industry, this means that HEOR teams will need to demonstrate how their drugs fit into these new quality measures.

SaraAbsolutely. We'll need to show that our drugs not only improve clinical outcomes but also contribute to social determinants and chronic care management. It's a broader value proposition.

MarcusThat's a good point. It could actually be an opportunity for innovative therapies that address unmet needs in these areas. But it also means more evidence generation.

SaraAnd that's where the budget impact comes in. If plans are focusing on these new measures, they might have less money for drugs that don't directly contribute. It's a zero-sum game in many ways.

MarcusI think that's the tension. The goal is better care, but the system is still constrained by budgets. We'll have to see how it plays out.

MarcusNICE has launched a consultation on updating the cost-effectiveness threshold for highly specialized technologies. Currently, the threshold is £100,000 per QALY, but they're considering raising it to £150,000 for these technologies. This could be a game-changer for ultra-orphan drugs and gene therapies.

SaraThat's one read — but I'd frame it slightly differently. The part that gives me pause is the budget impact. If NICE raises the threshold, it could open the door to more high-cost drugs, but where does that money come from?

MarcusThat's a fair point. The NHS is already under significant budget pressure. Raising the threshold could mean more drugs being approved, but also more strain on the system.

SaraExactly, and from a payer perspective, we have to consider opportunity cost. Every pound spent on one high-cost drug is a pound not spent on others. It's a tough balance.

MarcusThe consultation does mention that the threshold would only apply to technologies that meet specific criteria, like being for very small populations. So it's not a blanket increase.

SaraBut even with criteria, the cumulative effect could be significant. We've seen in the past that when thresholds are adjusted, it leads to a surge in submissions for high-cost drugs.

MarcusThat's true. And it raises questions about the sustainability of the system. How many drugs can the NHS afford at £150,000 per QALY?

SaraThat's the million-dollar question. The consultation is asking for feedback on the criteria, so there's an opportunity to shape the policy. But I'm still concerned about the long-term budget impact.

MarcusI wonder if that's the full picture though. The threshold increase might actually lead to more innovation in rare diseases, which could have broader benefits. And if it's limited to highly specialized tech, it might not affect the mainstream budget.

SaraThat's an interesting angle. But I think the devil is in the details. How they define "highly specialized" will be critical. If it's too broad, we could see budget creep.

MarcusThe consultation suggests that it would be for technologies that treat ultra-rare conditions, with a prevalence of less than 1 in 50,000. That's a narrow definition.

SaraBut even then, the number of eligible drugs could be substantial. And each one at £150,000 per QALY adds up. It's a significant investment.

MarcusI agree. But it might be necessary to encourage investment in these areas. Without a higher threshold, companies might not develop drugs for very small populations.

SaraThat's the patient perspective. We want these drugs to be available, but we have to balance it with system sustainability. It's a tough call.

MarcusIt is. And the consultation is open until September, so we'll see what feedback they get. But it's a clear signal that NICE is trying to adapt to the changing landscape of innovative therapies.

MarcusThe EMA has announced a new initiative for adaptive licensing in rare diseases. Adaptive licensing allows for earlier access to drugs based on interim data, with the requirement for more data to be collected post-approval. This is particularly relevant for rare diseases where traditional trials are difficult to conduct.

SaraThat's right, and what's striking here is the potential to accelerate access for patients with unmet needs. But I'm concerned about the evidence requirements for post-marketing studies. How do we ensure that these studies are actually completed?

MarcusThat's a valid point. The EMA is requiring companies to have a robust plan for post-marketing data collection, but enforcement can be challenging. We've seen cases where companies delay or fail to complete these studies.

SaraExactly, and from a payer perspective, we're taking a risk by covering a drug without full evidence. It's a trade-off between access and certainty.

MarcusThe EMA's initiative does include measures to ensure that the post-marketing studies are done, like linking approval to the completion of the studies. But it's still a work in progress.

SaraAnd that's where HTA bodies will have to play a role. We need to coordinate with regulators to make sure that the evidence generated meets our needs for value assessment.

MarcusI think that's key. The EMA is working with HTA bodies to align on the evidence requirements. This initiative is part of a broader effort to improve collaboration.

SaraThat's encouraging. But I still worry about the practicalities. How do we manage the data collection for very small populations? It's logistically challenging.

MarcusThe EMA is proposing the use of innovative trial designs, like basket trials and platform trials, to address this. It's about being flexible and creative.

SaraThat's one read — but I think payers would see it differently. We need to ensure that the data is robust enough to make informed decisions. Innovative designs are good, but they must still meet methodological standards.

MarcusThat's fair. And the EMA is emphasizing the importance of quality over speed. It's not about rushing drugs to market, but about getting evidence in a more efficient way.

SaraI think that's the right approach. But we'll have to see how it plays out in practice. The first wave of adaptive licensing applications will tell us a lot.

MarcusAbsolutely. It's an exciting development, but it requires careful implementation. The patient community is watching closely.

SaraA lot to think about today. I'll be watching how the CMS Star Ratings changes play out in the coming months, especially how plans respond to the new measures.

MarcusSame — and for me the thread running through today is the tension between accelerating access and ensuring robust evidence. It's a constant balancing act.

SaraThanks so much for listening — really glad you're here with us.

MarcusWe'll be back tomorrow. Show notes and transcripts at outcomes-analytica.no. See you then.

SaraThanks for listening — see you tomorrow.

MarcusBack tomorrow on Access Brief. Show notes at outcomes-analytica.no.