欧盟HTA在行动：健康技术开发人员如何为成功的JCA做好准备

EU HTA in Action How HTDs can prepare for a successful JCA ANKE VAN ENGEN,Vice President, Global Category Leader Health Economics, HTA, Value and Access, IQVIAMAX SCHLUETER,Senior Principal, EU HTA Solutions, IQVIAEDEL FALLA,Principal,EU HTA Solutions, IQVIASIAN TANNER,Principal, EU HTA Solutions, IQVIA Introduction The adoption of the Health Technology AssessmentRegulation (HTAR) marks a transformative milestone • How can EU HTA readiness be shaped in a waythat brings organisational efficiency and benefits in the European Union (EU) market access landscape,1and is one of many policy evolutions that health technology developers (HTDs) are currently navigating: • How do we align our regulatory, market access,and JCA strategies, given what we can learn from EU Critical Medicines and Biotech Acts,2,3EU General • How do we envisage JCA readiness evolving as theCoordination Group (CG) undertakes a review of the pricing dynamics, and rising use of health technologyassessment (HTA) frameworks globally, e.g., Association Drawing on IQVIA’s experience from 90+ engagementssince the inception of the EU HTAR, this white paperdelivers strategic guidance and actionable steps for • How do we balance EU Joint Clinical Assessment (JCA)against competing global evidentiary needs? EU HTAR 101 •EU HTAR entered into force on 12th January 2025,bringing two new processes:1. JCA is currently applicable for new oncologyand Advanced Therapeutic Medicinal Agents(ATMPs), for selected medical devices from 2026, • JCA runs in parallel to the EMA regulatorysubmission, with very short timelines to submitthe dossier once the scope is confirmed • The JCA report will evaluate the relative treatmenteffect but will not provide any value judgementor conclusions on the overall clinical added value; • MS should give due consideration to the JCA reportand not request the same information, data, • The scope of the JCA will encompass the clinicalevidence needs of all 27 EU member states (MS)in the form of PICOs (Population, Intervention, • MS remain responsible for drawing conclusions onthe value added for their health systems and for Where do you begin?Prepping for JCA success Working backwards from your first JCA-eligible asset’starget EMA filing date provides a clear roadmap toprioritise EU HTA readiness activities and identify critical A successful JCA submission begins long before thedossier is initiated. Irrespective of company size or assetspecifics, HTDs need to implement new ways of working ADAPT YOUR ORGANISATIONAL MODEL EU HTA requires an evolution of processes across theorganisation from market access, health economicsand outcomes research (HEOR) and biostatistics, toregulatory, medical, commercial, patient advocacyand legal functions. Strategic cross-functionalalignment across global, (regional) and local levels ofan organisation, beginning early in the asset lifecycle,is therefore critical. Establishing a clear above-asset Asset-level preparations must also begin early whilethere is still a window of opportunity to influencethe clinical development programme — a shift inorganisational thinking that can be challenging. Organisational models for EU HTA oversight and delivery have variable levels of complexity and centralization, Equally important to structural alignment is theengagement of internal stakeholders throughout theorganisation. To achieve JCA success, organisations mustprioritise education and regular, open communicationacross all internal stakeholders. Many HTDs have run PICO simulation is not enough in isolation: it musttranslate into a JCA strategy that is developed early andis clearly aligned to wider market access goals for theasset. Given the high volume of PICOs for JCA (based onIQVIA predictions, the first JCA scopes coming out of theJCA subgroup, and statements by the HTACG themselves), An asset-level EU HTA readiness plan ideally shouldcover three strategic domains: i. Evidence generationand JCA strategy; ii. Internal and external stakeholderengagement; and, iii. JCA dossier development. Itshould remain dynamic and extend all the way to assetlaunch and beyond. A cross-functional JCA/JSC team IQVIA, through collaboration with HTDs, has seenseveral of the first consolidated scoping documents, and 1. The number of PICOs remains high, driven byrequests for sub-populations and different 2. One can anticipate the PICO scope — our PICOsimulation methodology closely predicted those that 3. As anticipated, the proposed EMA label statementis key in determining the JCA scope and even small 4. MS-level PICO requests can best be predicted basedon previous HTA body (HTAb) assessments in related IMPLEMENT BEST PRACTICES FOR PICO PREDICTION Early anticipation of the JCA scope through PICOsimulation is the cornerstone of effective JCAplanning (Figure 2). This process involves generatingplausible PICO combinations based on the targetregulatory label, current and future EU-level and local 5. Subgrou