ISPOR Europe 2023, with the largest number of delegates on record (5,600) was held in wonderful, wonderful Copenhagen (cue the Danny Kaye waltz) last week. Neither congress nor venue disappointed, and despite leaden skies and gusty rain showers, attendees were able to sample scientific sessions by day and the Christmas Markets by night. The Panalgo team enjoyed sponsoring and exhibiting at another great ISPOR conference, and don’t get us started on the seafood, the hot spiced wine or ‘glögg’, or the Danish butter cookies.
ISPOR’s 2030 Strategic Plan
One of the biggest topics of conversation was the transformation of ISPOR itself. With HEOR at the Nexus of Policy and Science, ISPOR’s 2030 Strategic Plan – launching at ISPOR‘s North American meeting in Atlanta in May 2024 – includes three areas of focus: ‘Affordable Health Globally’, the ‘Science of Well-Being’, and ‘Better Health in the Digital World’. An emphasis on public health was threaded throughout the meeting and teed up in the opening plenary entitled, ‘European Health Data Space – RWE Put to Work for Public Health’.
EHDS, RWE, and Public Health
In the opening plenary, panelists talked about the use of real-world evidence (RWE) beyond pharmacovigilance and safety (the traditional focus of the EU), and the creation of the
European Health Data Space (EHDS). The EHDS will, among other things, establish a common EU framework and infrastructure through which scientists as well as public, private and not-for-profit entities can access health data for research and innovation, including in the oncology space. The European Union, with its 27 member states, has lagged behind the US (admittedly, a single entity) in healthcare data quality, consistency, and accessibility. As such, the European Commission proposed the EHDS to support or enable:
- individuals to take control of their own health data;
- use of health data for better: healthcare delivery, research, innovation and policy making, and;
- full use of the potential offered by a safe and secure exchange, use and reuse of health data.
The EHDS is not a pan-EU database made readily available to the public. It is rather a framework that lays out four phases of research: 1) execution of a meta-search to find the relevant data sources; 2) acquisition of permits for appropriate and allowable uses; 3) research conduct; and 4) data destruction when the research is done. The EU has long had strict privacy regulations and as such, data will not be made available on individual laptops and will not leave their respective jurisdictions. The EHDS is undoubtedly an ambitious project, but access to RWD and RWE is sorely needed in this disruptive era of small patient populations, single-arm trials, and fast-track approvals of novel, life-saving treatments.
HTA Regulation and JCA
Much of the ISPOR EU discourse centered on the new Health Technology Assessment (HTA) regulation. The regulation – adopted in 2022 and taking effect in just 15 months – aspires to facilitate the development of a ‘joint perspective’ on the ‘clinical’ aspects of drugs, in vitro diagnostics (IVDs) and high-risk medical devices. What is meant by ‘joint clinical assessment (JCA)’ as it relates to HTAs? The JCA aims to establish a single, transparent process to reduce redundant HTA practices and bring life sciences innovations faster to patients in Europe — no matter where they live. The JCA involves implementation of a standardized, pan-EU framework which all 27 member states will be required to use to assess the clinical evidence of newly submitted products or devices. Note that whereas the EHDS is not pan-EU, the JCA is just that.
The ultimate objective is to improve patient access by reducing HTA duplication and ensuring reproducibility while maintaining appraisal at the member state level.
The timeline for treatments to be assessed through this pan-EU lens is:
- 2025: cancer and advanced therapy medicinal products (ATMPs)
- 2028: orphan drugs
- 2030: all drugs, IVDs and high-risk medical devices
Collaboration on HTAs and harmonization of HTA processes across member states is invaluable, but the shift to a mandatory process will require the review and retooling of current practices and the creation of new workstreams to meet the new requirements. This assessment will start with a survey of the member states asking them for the patient/population, intervention, comparison and outcomes (PICOs) they need. This is such a critical topic for ISPOR EU delegates that a short course was offered at the conference – “Unveiling the EU-HTA Regulation: A Deep Dive into the Joint Clinical Assessment” – and Plenary Sessions #2 and #3 tackled the topic as well.
In the closing Plenary – which was well-attended despite being the last session of the ISPOR EU 2023 Congress – panelists talked at length about measuring the impact of the new HTA legislation. It will be a success, panelists debated, if it speeds access to medicines and brings consistency to the EU evidence paradigm. The HTA legislation must close the gap between high-cost treatment and weak real-world evidence. Review timelines – currently protracted at 400-500 days – must be reduced. One panelist summed up success as, “We aim for stronger, more comprehensive evidence with more timely, accurate, and transparent decision-making. And if the member states aren’t completely aligned, that’s okay. We can still move ahead.”
For a US citizen, the prospect of a single HTA framework across the 27 EU member states is both curious and daunting. The US currently has no HTA agency, except for ICER (which has no legal authority) and CMS (which appears to be moving in that direction, but challenges abound). Perhaps one day in the not-too-distant future, a global framework will take shape so that we bring life sciences innovations faster to patients on every continent — no matter where they live and no matter their circumstances.
 Neumann PJ, Tunis SR. Turning CMS into a Health Technology Assessment Organization. N Engl J Med 2023; 389:682-684. August 24, 2023. DOI: 10.1056/NEJMp2305280