Patient Advocacy & HTA Evolution

Marcus"Welcome 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."

Sara"And 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 the field."

Marcus"Same here. We're looking at the evolving role of patient advocacy in evidence generation — why that's becoming a central pillar of modern market access. Then the intersection of ESG factors with drug pricing strategies. And the unique challenges of demonstrating value for first-in-class therapies. Plus, the future of HTA in emerging markets."

Sara"That first topic is interesting to me — the budget impact question around patient advocacy engagement is one the field hasn't fully worked out yet. Who bears the cost of that engagement?"

Marcus"Exactly. Let's get into it."

Marcus"We're seeing a clear trend: patient advocacy groups are no longer just stakeholders but active participants in shaping the evidence requirements for HTA. They're bringing forward patient-reported outcomes that matter most, and in some cases, even co-designing studies."

Sara"That's one read — I'd frame it slightly differently. What strikes me about that is the resource implications. These groups often lack the methodological expertise, so companies are stepping in to provide that support. But then you get into questions of independence and objectivity."

Marcus"That's fair, though I think payers would see it differently. They're concerned about the authenticity of the evidence when it's co-developed with industry. But the patient perspective is critical, especially for rare diseases where traditional endpoints might not capture the full burden."

Sara"And what's interesting is that some HTA bodies are starting to formalize this. We're seeing more requests for patient evidence in submissions, which is a shift from just clinical data to a more holistic view."

Marcus"Exactly, and that connects to something I keep coming back to: the value of lived experience. When patients articulate what matters to them, it can reframe the entire value proposition. But the challenge is ensuring that this evidence is rigorous and not just anecdotal."

Sara"The part that gives me pause is the sustainability. If every company is engaging deeply with advocacy groups, how do we ensure consistency and avoid a situation where the evidence is skewed by the most vocal groups?"

Marcus"That's a real concern. Some are calling for standardized frameworks for patient engagement in research. The FDA has issued guidance on this, but HTA bodies are slower to adapt. The risk is a patchwork of approaches that create uncertainty for developers."

Sara"And from a payer perspective, the question is: does this evidence lead to better decisions? If it improves the relevance of assessments, that's good. But if it adds complexity without clear value, it could become a burden."

Marcus"I wonder if that's the full picture though. The trend is irreversible. Patients are demanding a seat at the table, and ignoring that could lead to products that don't address real needs. The key is integration without compromise on methodology."

Sara"Agreed, but the burden of proof is on the industry to demonstrate that this engagement leads to better outcomes. Payers will need to see the data to be convinced."

Marcus"That's the tension. The potential is huge, but the execution must be flawless. We'll see how it plays out."

Marcus"Now, the integration of ESG factors into drug pricing strategies. Companies are increasingly highlighting sustainability efforts as part of the value proposition, especially in Europe."

Sara"That's one read — I'd push back slightly. What I'm seeing is that payers are skeptical. They're focused on clinical and economic value, and ESG factors are often seen as secondary. The budget impact question here is significant: how do you price in sustainability?"

Marcus"That's fair. But consider this: in some markets, particularly those with strong ESG mandates, there's a willingness to reward companies with better sustainability profiles. It's not about replacing traditional value, but augmenting it. The challenge is quantifying it."

Sara"And that's the crux. How do you measure the social impact of a drug in a way that's comparable? If a company reduces its carbon footprint, does that justify a higher price? The evidence isn't there yet."

Marcus"I'd frame it differently. It's not about justifying higher prices, but about avoiding discounting for poor ESG performance. Some payers are starting to incorporate ESG into their value frameworks, but it's still early. The risk is greenwashing without real impact."

Sara"What strikes me is the inconsistency. One country might reward ESG, another might ignore it. That creates complexity for companies and uncertainty in the market. And patients don't necessarily benefit from this; it's more about corporate image."

Marcus"That's a valid point. But the patient angle is important too. If a company's ESG efforts lead to more equitable access in low-income countries, that could be part of the value proposition. But it has to be transparent and verifiable."

Sara"The part that gives me pause is the potential for distraction. When companies focus on ESG, it might divert resources from core evidence generation. Payers need to see that the drug works and is cost-effective first. ESG can be a tie-breaker, not the main event."

Marcus"Exactly. The smart approach is to integrate ESG into the overall strategy, not as an afterthought. But until there's a standardized way to measure it, it will remain a niche consideration. We'll see if it gains traction."

Sara"Now, the challenge of first-in-class therapies. These are often breakthroughs, but they come with high uncertainty. How do you demonstrate value when there's no comparator? This is particularly acute in areas like oncology and neurology."

Marcus"That's one read — I'd frame it slightly differently. The issue isn't just the lack of comparators; it's the opportunity cost. When a first-in-class drug is priced high, it displaces other treatments that might offer more value for the same money. Payers are grappling with that."

Sara"That's fair. But consider the innovation premium. First-in-class therapies often represent a leap forward, and the initial price reflects that. The question is: can they sustain that price as more options emerge? The evidence needs to show durability of effect."

Marcus"I wonder if that's the full picture though. The challenge is that first-in-class drugs are often for small populations. The budget impact might be low, but the precedent for pricing could be high. That's a tension for both sides."

Sara"The part that gives me pause is the evidence gap. Without comparators, you rely on historical data or modeling, which can be speculative. Payers need to see that the drug delivers on its promise in real-world settings."

Marcus"Exactly. The key is generating evidence early and continuously. Some companies are using basket trials or platform designs to build evidence faster. But it's a resource-intensive approach. The risk is that without strong evidence, the drug fails to gain traction."

Sara"And from a patient perspective, the hope is that these therapies address unmet needs. But if the price is too high, access could be limited. The industry needs to balance innovation with affordability."

Marcus"That's the tightrope walk. The potential is huge, but the execution must be evidence-based. We'll see how the market evolves."

Sara"Finally, the future of HTA in emerging markets. Traditionally, these countries have relied on the assessments of wealthier nations. But now, there's a push for local HTA capacity building. Why does that matter now?"

Marcus"That's one read — I'd push back slightly. What I'm seeing is that these countries are overwhelmed. They lack the resources and expertise to conduct robust HTA, and the burden of disease is high. The question is: can they build capacity without compromising quality?"

Sara"That's fair. But consider this: as more innovative therapies become available, emerging markets need to make informed decisions. Relying on external assessments might not reflect local epidemiology or healthcare systems. The trend is towards more localized approaches."

Marcus"And what strikes me is the funding issue. Who pays for this capacity building? If companies or donors step in, there's a risk of bias. Independence is critical for credible HTA."

Sara"I'd frame it differently. It's not just about funding; it's about collaboration. Some initiatives, like the World Health Organization's efforts, are helping to build frameworks. The potential is more equitable access to innovative treatments."

Marcus"The part that gives me pause is the timeline. Building HTA capacity takes years, and in the meantime, countries are making decisions with limited evidence. That could lead to suboptimal use of resources."

Sara"Exactly. The key is incremental progress. Starting with simple assessments and building up. But the risk is that without international support, these countries might fall further behind. The industry has a role to play, but it must be done ethically."

Marcus"Agreed. The goal is to ensure that emerging markets can participate in the global conversation about value. That's essential for sustainable access."

Sara"A lot to think about today. I'll be watching how patient advocacy groups evolve in their role in evidence generation. It's a space that's moving fast."

Marcus"Same — and for me the thread running through today is the growing complexity in value demonstration. Whether it's ESG, first-in-class, or emerging markets, the evidence requirements are becoming more nuanced."

Sara"Thanks so much for listening — really glad you're here with us."

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

Sara"Thanks for listening — see you tomorrow."

Marcus"Back tomorrow on Access Brief. Show notes at outcomes-analytica.no."