ISPOR Europe 2024 – inside the first plenary: Navigating evidence-price challenges for patient access

ISPOR Europe 2024, the European conference for ISPOR—The Professional Society for Health Economics and Outcomes Research, is taking place November 17–20, 2024 in Barcelona, Spain. Featuring a comprehensive program centered around the theme, ‘Generating Evidence Toward Health and Well-Being’, the first plenary of the conference on Day 1 tackles this head on by addressing, "The Evidence-Price Conundrum: What Is the Way Forward for Patient Access?" Here, I share an overview and key takeaways from the first plenary session at the conference.

The ISPOR Europe 2024 opening plenary session, moderated by Yannis Natsis (European Social Insurance Platform [ESIP], Brussels, Belgium, and co-chair of the ISPOR Europe 2024 conference program), addressed the complex interplay between evidence generation, pricing and patient access. The session featured an esteemed panel of experts, including Valérie Paris (OECD, France), Pedro Facon (National Institute for Health and Disability Insurance, Belgium), Birgitte Klindt Poulsen (Danish Medicines Council, Denmark) and Richard Bergstrom (IQVIA, Switzerland). Discussions spanned regulatory evidence trends, affordability challenges and actionable solutions to navigate these pressing issues.

Evidentiary requirements: trends and challenges

The panel began by examining the shifting standards of evidence for regulatory approvals. Paris highlighted the increasing pressure on regulatory agencies like the FDA and EMA to accelerate drug approvals, particularly for treatments addressing unmet medical needs. This shift has led to acceptance of new trial designs and surrogate markers, reducing average approval times at the FDA from over 2 years to under 1 year. However, over half of EMA-approved drugs via accelerated pathways lack Phase 3 trial data, creating challenges for HTA agencies reliant on high-quality evidence for cost-effectiveness assessments.

“Uncertainty is just inflated during all these different assessments.”
Valérie Paris, ISPOR Europe 2024

Poulsen spoke next, emphasizing the risks posed by sparse datasets, which complicate evaluations of efficacy, safety and cost-effectiveness. These uncertainties, she argued, increase the risk of harm – both to patients and to healthcare systems – by potentially approving ineffective or unsafe treatments.

“Sparse data increases our risk of doing harm.”
Birgitte Klindt Poulsen, ISPOR Europe 2024

Similarly, Facon underlined that payers, constrained by budgets and opportunity costs, face significant difficulties in calculating the added value of therapies when evidence at market authorization is weak. Facon opposed incentives like extended market protection for gold-standard trials, arguing that robust evidence should be a baseline requirement.

“As a payer, we want to reimburse good medicines that have value for patients.”
Pedro Facon, ISPOR Europe 2024

Bergstrom, however, argued that regulatory standards have not weakened but have evolved alongside scientific advances. He called for greater ambition in post-launch evidence generation, suggesting that gaps in real-world comparative effectiveness studies reflect a failure of follow-through rather than a decline in pre-approval evidence standards.

“Evidence generation is a continuum.”
Richard Bergstrom, ISPOR Europe 2024

Post-launch evidence generation: a crucial need

Post-market studies emerged as a pivotal topic in the discussion. Poulsen partially disagreed with Bergstrom, advocating for stronger requirements for confirmatory trials. She noted that 15% of FDA approvals via accelerated pathways are later withdrawn due to failed or uncompleted trials, and proposed that EMA align with FDA practices by mandating, at a minimum, detailed protocols for confirmatory studies at the time of conditional approval. She suggested that other stakeholders, including HTAs, could also play a role in generating post-launch data.

Facon described Belgium’s experience with managed entry agreements (MEAs), which now account for 40% of pharmaceutical expenditure. While MEAs often include evidence-generation provisions, weak research designs and insufficient follow-up frequently perpetuate uncertainty. Facon revealed plans to cap contract renewals and collaborate with stakeholders on better research questions and follow-up mechanisms. He acknowledged that uncertainty has, at times, been leveraged as a negotiating tool to reduce prices – a practice he deemed unacceptable.

Pricing and affordability: a global challenge

With the discussion turning to affordability, Paris observed that while many countries have mechanisms to contain pharmaceutical costs, these often rely on broad rebates, which lead to their own consequences. She pointed to growing drug shortages, particularly in generics, as evidence that some price-containment measures may have unintended effects. Paris also critiqued the opacity of pricing systems, where rebates and discounts obscure true costs, complicating HTA evaluations and policy decisions.

Bergstrom elaborated on global pricing dynamics, noting parallels with US practices under the Inflation Reduction Act. He warned that over 50% of biologics in Europe lack biosimilars in development, raising concerns about sustained competition post-exclusivity. Bergstrom advocated for proactive negotiations, such as agreements to emulate loss-of-exclusivity price reductions even without biosimilar entry.

Natsis introduced findings from a recent report, “Trends in Pharmaceutical Expenditure”, which confirmed rising prices as a systemic issue. Facon echoed this, highlighting Belgium’s investments in forecasting and collaborations like Beneluxa to address budgetary impacts. He advocated for deeper European collaboration, envisioning a future with joint EU-level negotiations to address pricing disparities and inefficiencies.

“Prices are soaring across the board, and that … is a reminder that the high prices of medicines remain a systemic problem.”
Yannis Natsis, ISPOR Europe 2024

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Solutions and the role of transparency

The session concluded with actionable solutions. Facon defended the necessity of secrecy in contract negotiations under the current reference pricing system, though he acknowledged its drawbacks. Poulsen called for greater transparency in decision-making processes, emphasizing that stakeholders should focus on the bottom-line cost-effectiveness of treatments rather than being swayed by headline rebates.

Bergstrom expressed optimism about joint advice initiatives among regulators, HTAs and payers, calling for a broader understanding within the payer community that evidence generation is a continuum. He highlighted the potential of regulatory sandboxing – collaborative, multi-stakeholder frameworks – to align evidence generation with evolving healthcare needs.

Paris stressed the importance of horizon scanning to anticipate challenges and ensure timely decision-making. Collaboration emerged as a unifying theme, with Poulsen advocating for shared data efforts to accelerate evidence generation and strengthen HTA recommendations.

Conclusion

Overall, the first plenary session of ISPOR Europe 2024 underscored the complexity of balancing evidence, price and patient access in a rapidly evolving healthcare landscape. While progress has been made in areas like MEAs and regulatory alignment, significant challenges remain in addressing uncertainty, ensuring affordability and fostering transparency. The panelists emphasized that collaborative, multi-stakeholder approaches will be key to achieving sustainable and equitable healthcare systems.