DECEMBER - JANUARYLIFE SCIENCES REVIEW9France recommends inclusion of a short generic questionnaire such as the EQ-5D-5L to support economic evaluations as well as well-validated disease specific PROs.4Unlike the UK and Germany, France prefers PROs that are included as primary or secondary endpoints to adjust for multiplicity. Although the effect of PROs on the benefit rating cannot usually be isolated,the absence of HRQoL data may negatively impact reimbursement decisions involving chronic conditions with a high HRQoL impact as well as end of life treatments. HRQoL assessments indirectly affect pricing through the benefit rating assigned.According to a recent review of HTA submissions in decentralized markets including Italy, Spain and the US, PROs have been influential in certain reimbursement submissions particularly for Oncology but there are no formal processes established for consideration of PROs in reimbursement decisions.5In the US, this is now slowly changing because the Institute for Clinical and Economic Review (ICER) has been conducting economic evaluations based on cost-effectiveness modeling to influence the reimbursement of new therapies by various payers in the US.In addition to influencing pricing and reimbursement directly as described above, PROs may also be useful in differentiating new therapies from existing treatments to gain market share. This is particularly useful when there is a highly competitive landscape where there are many therapies available to healthcare providers and patients for the treatment of a specific condition. Publication of PRO results from clinical trials to evaluate HRQoL is critical. New standards have been established for conducting comparisons of competing therapies based on HRQoL and utility measures in Europe.6In certain situations, for chronic conditions where the impact on HRQoL is substantial, the ability to promote a new therapy's impact on HRQoL relative to its competitors may be highly desirable. When determining which PROs to include in clinical trials, the needs of these various stakeholders must be considered. The purpose of including disease specific PROs is to determine the effect of the new therapy on HRQoL in relation to the comparator included in the clinical trial as well as the durability of the effect with the experimental treatment over time. By contrast, the primary reason to include the EQ-5D-5L in clinical studies is to determine the decrement in HRQoLfor certain clinical events or health states likely to be included in the cost-effectiveness analysis. If the decrements in HRQoL are well-documented in the published literature,it may not be necessary to include the EQ-5D-5L in the clinical program in certain situations. If the purpose of including the PROs is to include the HRQoL results in a regulatory label in order to actively promote the results in relation to its competitors, then the PROs generally need to be included in Phase III studies as primary or secondary endpoints with adjustment for multiplicity. This is also an important consideration for reimbursement decisions in France. The standards for PROs established by each targeted regulatory agency must be met. The standards differ for different regulatory agencies just as they do for different reimbursement agencies.Early clarity on the target markets, and on the purpose for which the PROs will be used is essential. The standards differ for different regulatory agencies just as they do for different reimbursement agencies
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