RWE Guidance, Star Ratings, Digital Therapeutics, EU HTA

MarcusWelcome to the Access Brief — your daily briefing on what's moving in 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 policy shifts we're seeing across jurisdictions.

MarcusSame here. We're looking at FDA's new RWE guidance for rare disease indications — which could reshape evidence generation for these populations. Then CMS's proposed changes to Medicare Advantage Star Ratings for 2027 — a significant shift in quality metrics. And NICE's updated framework for digital therapeutics in chronic conditions — finally some clarity. Plus, the EU's first wave of Joint Clinical Assessments for oncology drugs — a major milestone.

SaraThat second one on Medicare Advantage Star Ratings is interesting to me — the quality metric changes there could have profound budget implications for plans and member access.

MarcusExactly. Let's get into it.

MarcusStarting with the FDA. They've just released new guidance on using real-world evidence for rare disease indications. This is significant because rare diseases often have limited patient populations, making traditional trials challenging. The guidance emphasizes the use of natural history studies and patient registries to support drug development and approval.

SaraThat's one read — I'd frame it slightly differently. The part that gives me pause is the methodological rigor. How do you ensure that these real-world data sources are robust enough to support regulatory decisions? There's a risk of confounding factors that traditional RCTs control for.

MarcusThat's fair. The guidance does stress the importance of prospective data collection and clear endpoints. But I wonder if that's the full picture though... The FDA is also encouraging the use of novel statistical methods to handle small sample sizes. Still, the onus is on sponsors to demonstrate data quality.

SaraRight, and from the payer side, we're already seeing HTA bodies like NICE and others using RWE in their assessments. This FDA move might align with that trend, but the challenge remains in translating regulatory evidence to value assessments. The budget impact question there is one the field hasn't fully worked out yet.

MarcusAgreed. The guidance also mentions that RWE can be used to support accelerated approvals. That could be a game-changer for ultra-rare diseases where placebo-controlled trials are unethical. But the post-marketing requirements become critical then.

SaraExactly. If a drug gets approved based on RWE, but then the real-world effectiveness doesn't hold up, who bears the cost? That's a concern for sustainability.

MarcusTrue. The FDA does require confirmatory trials, but the timeline can be long. This guidance might speed up access, but it also increases uncertainty for payers. It's a delicate balance between innovation and evidence certainty.

SaraI keep coming back to the patient perspective here. For rare disease patients, faster access is crucial. But we also need to ensure that the evidence is solid. This guidance is a step in the right direction, but we need more clarity on the validation processes.

MarcusThat's a key point. The FDA is inviting comments on the guidance, so the industry and patient groups will have a chance to weigh in. We'll see how it evolves.

SaraNext, CMS has proposed changes to the Medicare Advantage Star Ratings system for 2027. The key updates include new measures for care coordination and social determinants of health, and adjustments to existing measures like chronic care management. The goal is to better reflect the quality of care provided.

MarcusThat's one read — I'd frame it slightly differently. The part that gives me pause is the potential for increased administrative burden. Plans will need to collect and report on new data, which could divert resources from patient care. Also, the weight given to these new measures isn't clear yet.

SaraThat's fair. CMS is proposing a phased approach, but the timeline is tight. Plans will have to adapt quickly. What strikes me about that is the shift towards more holistic quality metrics. It's a recognition that health outcomes are influenced by factors beyond clinical care.

MarcusRight, and from the payer side, this could lead to more investments in social services and community partnerships. But the budget impact question here is significant. If plans are required to invest in these areas, how does that affect premiums and member costs?

SaraExactly. CMS is also proposing changes to the star rating calculation methodology, which could lead to volatility in scores. Plans that were previously high-performing might see their ratings drop if they don't meet the new standards. That could affect enrollment and revenue.

MarcusTrue. CMS is accepting comments on the proposal, so there's an opportunity for the industry to provide feedback. But the direction is clear: CMS is pushing for a more comprehensive view of quality. It's a challenge, but also an opportunity to innovate.

SaraI'll be watching how plans respond to this. It will be interesting to see if they invest in the required areas or try to game the system. The long-term impact on patient outcomes is what matters most.

MarcusNow to NICE. They've updated their framework for digital therapeutics, specifically for chronic conditions. The new guidance includes a more streamlined assessment process and clearer criteria for evidence generation. This is a response to the growing number of digital health products in the market.

SaraThat's one read — I'd frame it slightly differently. The part that gives me pause is the evidence requirements. NICE is still demanding robust clinical evidence, which can be difficult for digital therapeutics that are often iterative and update frequently. How do you handle that?

MarcusThat's fair. The framework acknowledges that digital therapeutics are different and allows for a more flexible approach to evidence, such as using real-world data and adaptive trial designs. But the core requirement for demonstrating clinical effectiveness remains.

SaraRight, and from the payer side, we're concerned about the long-term value. A digital therapeutic might show short-term benefits, but what about adherence and sustained impact? The framework doesn't fully address that.

MarcusExactly. NICE is also emphasizing the importance of user experience and engagement, which is crucial for digital therapeutics. They're requiring sponsors to provide data on how patients interact with the product. That's a step forward.

SaraThat's good, but I keep coming back to the budget impact. These products often come with ongoing costs, such as subscription fees and device support. How do we ensure that they offer value for money compared to traditional treatments?

MarcusTrue. The framework includes a cost-effectiveness assessment, but it's not clear how they will handle the dynamic nature of digital products. NICE is open to iterative evidence, which might help. But it's still a work in progress.

SaraI'll be watching the first few appraisals under this new framework. It will be telling how NICE balances innovation with the need for solid evidence.

MarcusFinally, the EU has launched the first wave of Joint Clinical Assessments under the HTA Regulation for oncology drugs. This is a major milestone, as it's the first time that multiple countries are assessing the same products simultaneously. The goal is to increase efficiency and consistency.

SaraThat's one read — I'd frame it slightly differently. The part that gives me pause is the practical challenges. Coordinating assessments across multiple HTA bodies with different methodologies and timelines is complex. How do they ensure consistency?

MarcusThat's fair. The EU has established a common framework, but each country still has the final say on pricing and reimbursement. The JCA is meant to provide a shared clinical assessment, but the value judgments remain national. It's a compromise.

SaraRight, and from the payer side, we're concerned about the workload. Each country will still need to adapt the JCA to their local context. That might not save as much time as hoped. Also, the first wave includes only oncology drugs. What about other areas?

MarcusExactly. The EU is starting with oncology because it's a high-priority area, but the plan is to expand to other therapeutic classes. The JCA process is designed to be iterative, so they'll learn from this first wave. But the timeline is tight — the first assessments are due by the end of the year.

SaraThat's ambitious. The industry will need to provide comprehensive data, and HTA bodies will need to collaborate effectively. The patient perspective is important here — faster assessments could mean earlier access, but only if the process is robust.

MarcusTrue. The EU is also working on a common HTA work plan to align priorities. This is a significant step towards more harmonized assessment, but there are still hurdles to overcome. We'll be watching how this first wave plays out.

SaraA lot to think about today. I'll be watching how the FDA's RWE guidance evolves and how CMS's Star Ratings changes impact plan behavior.

MarcusSame — and for me the thread running through today is the increasing emphasis on real-world data and holistic quality metrics. It's a shift towards more patient-centered evidence.

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.