Table 2 Table of 10 proposals to industry and 10 proposals to HAS to improve HRQoL data adoption in France, framed around the four key areas of work: patient perception, testing hierarchy, trial design and data collection

Patient perception

 Proposal 1.1: Establish the minimal clinically important difference or meaningful change for each relevant patient population

Proposal 2.1: Improve the input PAGs are able to have in the assessment process by asking them to comment on industry submissions (including on the difference observed in clinical trials), possibly by providing detailed guidelines on patient engagement allowed in the current legal context

 Proposal 1.2: Seek further input from patient advocacy groups when designing trials (specifically in selection of outcomes measures)

 Proposal 1.3: Where legislation allows, share relevant literature and data with patient advocacy groups

Testing hierarchy

 Proposal 1.4: Remind clinical trial teams of the importance of collecting HRQoL data

Proposal 2.2: Provide more feedback on the quality and appropriateness of HRQoL data submitted, and any challenges to be addressed in future trials

Proposal 2.3: Where possible, consider HRQoL data that was not included in a testing hierarchy

 Proposal 1.5: Clarify the HRQoL score(s) of interest upfront in the stats analysis plan

Proposal 2.4: Consider the value of HRQoL data from RWE studies (and, if relevant, develop appropriate guidance on how to obtain, analyse and report RWE data, and clarify the circumstances in which these would be most useful)

Trial design

 Proposal 1.6: Minimize the risk of a bias when collecting HRQoL data in open-label trials

Proposal 2.5: Define specific circumstances where using HRQoL data from open-label trials would be acceptable

 Proposal 1.7: Submit detailed rationales for using HRQoL data from open-label trials

Proposal 2.6: Clarify the guidance for the type of instrument to use

Proposal 2.7: Consider the role of novel trial designs and their role in collecting HRQoL data in future studies

Data collection

 Proposal 1.8: Use digital technologies where this could improve the quality of HRQoL data or the patient experience

Proposal 2.8: Clarify the relative importance of HRQoL versus other clinical parameters such as efficacy

 Proposal 1.9: Simplify HRQoL instruments used by reducing their length, and improve relevance to patients

Proposal 2.9: Consider differential recommendations for products where they have a similar efficacy but different impact on patient HRQoL

 Proposal 1.10: Thoughtfully choose appropriately validated instruments used in trials, and clearly report on these

Proposal 2.10: Support the definition of a Europe-wide threshold for missing data as part of EUnetHTA, or discuss methods that will enable the decrease of the impact of missing data in industry submissions