"THE MOUNTAIN LABOURED AND GAVE BIRTH TO A MOUSE": A SURVEY OF CURRENT AND FORMER EUROPEAN PAYERS ON THE FIRST EU JOINT CLINICAL ASSESSMENT REPORT
Author(s)
James Horscroft, MA, PhD1, Sylwia Lach, MPH2, Helena Emich, PhD3, Rachel Beckerman, PhD4.
1Maple Health Group, New York, NY, USA, 2Maple Health Group, LLC, Cracow, Poland, 3Maple Health Group, Cambridge, United Kingdom, 4600 Third Ave, New York, NY, USA.
1Maple Health Group, New York, NY, USA, 2Maple Health Group, LLC, Cracow, Poland, 3Maple Health Group, Cambridge, United Kingdom, 4600 Third Ave, New York, NY, USA.
OBJECTIVES: The first European Union (EU) Joint Clinical Assessment (JCA) was published in June 2026, covering tovorafenib for paediatric low-grade glioma, which scoped eight PICOs but assessed only one, citing insufficient comparative data. We surveyed experienced EU payers to characterise perceptions of the inaugural JCA report and its anticipated impact on local health technology assessment (HTA).
METHODS: A structured online survey was administered to current and former healthcare decision-makers/payers across EU Member States. Respondents rated six statements on a five-point Likert scale (strongly disagree-strongly agree) spanning PICO relevance, evidentiary thoroughness, local HTA efficiencies, attainability of JCA standards in rare diseases, and overall optimism, plus an open free-text field. Responses were summarised descriptively.
RESULTS: Eleven payers from Germany (n=4), Spain (n=3), France (n=2), and Italy (n=2) responded; three were currently active (including an AMNOG arbitration board member) and eight formerly held relevant positions. A majority (6/11) agreed that the eight scoped PICOs reflected locally relevant populations. Views on thoroughness were mixed (6/11 satisfied; 3 neutral). Most (6/11) disagreed that the report would save local HTA review time, and over half (6/11) were not optimistic about the JCA's overall benefit to local HTA. Six of 11 agreed the report signaled that JCA evidentiary standards may be unattainable in rare diseases; whether JCA would raise local standards was evenly split (4 agree/4 disagree/3 neutral). Free-text themes described a voluminous yet largely "non-assessment", concern over a single manufacturer-conducted matching adjusted indirect comparison (MAIC) and minimal patient/clinical input, and expectation that national bodies will set their own PICOs regardless.
CONCLUSIONS: Experienced EU payers regarded the first JCA report as largely methodologically rigorous but of limited practical utility for local HTA, tempering early expectations. Whether this reflects an atypical rare-disease index case or more fundamental underlying issues warrants monitoring as further JCA reports are published.
METHODS: A structured online survey was administered to current and former healthcare decision-makers/payers across EU Member States. Respondents rated six statements on a five-point Likert scale (strongly disagree-strongly agree) spanning PICO relevance, evidentiary thoroughness, local HTA efficiencies, attainability of JCA standards in rare diseases, and overall optimism, plus an open free-text field. Responses were summarised descriptively.
RESULTS: Eleven payers from Germany (n=4), Spain (n=3), France (n=2), and Italy (n=2) responded; three were currently active (including an AMNOG arbitration board member) and eight formerly held relevant positions. A majority (6/11) agreed that the eight scoped PICOs reflected locally relevant populations. Views on thoroughness were mixed (6/11 satisfied; 3 neutral). Most (6/11) disagreed that the report would save local HTA review time, and over half (6/11) were not optimistic about the JCA's overall benefit to local HTA. Six of 11 agreed the report signaled that JCA evidentiary standards may be unattainable in rare diseases; whether JCA would raise local standards was evenly split (4 agree/4 disagree/3 neutral). Free-text themes described a voluminous yet largely "non-assessment", concern over a single manufacturer-conducted matching adjusted indirect comparison (MAIC) and minimal patient/clinical input, and expectation that national bodies will set their own PICOs regardless.
CONCLUSIONS: Experienced EU payers regarded the first JCA report as largely methodologically rigorous but of limited practical utility for local HTA, tempering early expectations. Whether this reflects an atypical rare-disease index case or more fundamental underlying issues warrants monitoring as further JCA reports are published.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
HTA168
Topic
Health Policy & Regulatory, Health Technology Assessment, Organizational Practices
Topic Subcategory
Decision & Deliberative Processes, Systems & Structure