European Federation of Statisticians in the Pharmaceutical Industry
EFSPI
EFSPI, the European Federation of Statisticians in the Pharmaceutical Industry, is open to constituted groups of statisticians.
ID: 420759225344-39
Lobbying Activity
Response to Health technology assessment - Joint clinical assessments of medicinal products
2 Apr 2024
EFSPIs HTA European Special Interest Group (HTA ESIG) is a group within the European Federation of Statisticians in the Pharmaceutical Industry (EFSPI), a learned society representing over 2000 pharmaceutical statisticians in Europe. The EFSPI HTA ESIG provides a direct link to scientific and operational HTA statistical expertise in the pharmaceutical industry. Our constructive suggestions on the EU HTA Regulation Implementing Acts focus on two aspects: leveraging the information of the HTD to ensure a workable and scientifically sound assessment scoping process and, through doing so, enabling a revision of the timelines to ensure sufficient time to deliver the high-quality statistical analyses needed for the JCA dossier. (1) Whether evidence is available to address a specific PICO (statistical feasibility) should be taken into consideration when developing the assessment scope. Failure to account for this could result in PICO questions that can be answered neither from analysis of registrational pivotal trials nor indirect treatment comparison analyses. The HTD's extensive literature reviews, data gathering, and evaluation of disease incidence and clinical practices should be utilized. The HTD should be allowed to submit an assessment scope proposal with appropriate references alongside the information required by Article (Art.) 2(1) and 2(2) and for any such proposal to be considered by the assessor under Art. 9. More broadly, the assessor should seek input from the HTD during any phase of the scoping process described in Art. 9 and 10, including seeking input on whether or not PICOs are likely to be statistically feasible. We specifically recommend that in section 16 of the introduction to the IA, the text be modified to include input from the HTD in the assessment scope proposal, and that the assessment scope proposal is reviewed with the HTD to evaluate statistical feasibility. Art. 2(2) should be amended to include the requirement to submit a proposed PICOs scope, with accompanying explanatory references. (2) Prior to finalizing the assessment scope, the opportunity to attend an assessment scope explanation meeting is welcomed (Art. 11), but the HTD should have the right to request attendance, and not attend only if deemed necessary by the JCA Subgroup. This is critical in order to align on PICO prioritizations in case of inadvertent conflicts, such as mutually exclusive patient-level data availability (this may occur when evidence for different PICOs is derived from very small patient subgroups were presenting the evidence for both could inadvertently disclose patient-level information). In specific, the introduction Section 20 of the IA should be modified to say that the JCA subgroup will not may meet with the HTD - with the aim of determining the evaluability and prioritization of PICOs where necessary. (3) As also noted by EFPIA, the HTD should have a minimum of 4.5 months from scope finalization in order to deliver the high-quality statistical analyses needed for JCA. Reducing this time risks compromising statistical quality and evidence integrity. By leveraging the insights of the HTD as suggested above, the scoping period can be shortened. We support shortening the scoping period from 140 to 60 days and allowing 110 days (AA and line extensions) or 150 days (initial MAA) for dossier production. The cut-off date for systematic literature reviews (as defined in Annex 1, 4.2.1) should align with the final assessment scope communication, rather than being fixed at 3 months before dossier submission. This allows for proper planning, updating, review, and execution of pre-planned statistical analyses for direct and indirect comparisons (as required in Annex 1, 4.3.3 and 4.3.4), and would mitigate any requirements to update all reviews and analyses in the event of regulatory clock stops that push out the date of the JCA submission. Specific proposals for wording changes are attached.
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