AIXIAL Review #5

For a better assessment of the health-related quality of life in the development of new drugs


Patient Reported Outcomes (PROs) and more specifically auto-questionnaires of health-related quality of life (HrQoL) have long been neglected within the Evidence Based Medicine (EBM) or considered more as medico-economic evaluation tools to be used for Value Based Medicine (VBM) in the context of utility theroy than in that of health evaluation[1]. 

PRO and HrQoL have become crucial elements in the development of new drugs and in the criteria for registration and access to reimbursement. This is particularly the case for conditions that severely alter this quality of life and for which its measurement, as appreciated by patients themselves, is one of the important judgement criteria expected by the Health authorities (see the HAS summary document on its expectationsin terms of quality of life documentation[2]).

However, it is clear that there is a lack of data available at the drug registration stage, for example, in areas such as oncology where quality of life is a particularly important criterium[3].

Moreover, it should be noted that the HrQoL data provided to the registration or Health Technology Assessment (HTA) agencies by pharmaceutical industries only rarely makes it possible to value health products (drugs or medical devices) because they are considered by the accessors as not acceptable for a methodological point of view. This is even true for HTA agencies or authorities such as, in France, la Haute Autorité de Santé (HAS), which regularly consider these data as exploratory ones.

Faced with this, AIXIAL, in the context of its R&D laboratory, in partnership with a renowned academic research team focused on quality of life (the team of Prof. O Chassany of the Hôtel-Dieu de Paris), is carrying out a methodological research program aimed at establishing recommendations so that manufacturers can build and conduct quality of life studies that can help them better understand the expected impact of their products and to promote them.

At the same time, in order to benefit the industries that develop and bring to the market these products, AIXIAL, with the help of its external experts (in HTA and PRO) as well as with patient representatives, opened a consultation to help manufacturers build their clinical and epidemiological studies including an assessment of quality of life.


— Written by Jacques Massol, MD, PhD, AIXIAL consultant

[1] Brown MM, Brown GC, Sharma S, Garrett S. Evidence-based medicine, utilities, and quality of life. Curr Opin Ophthalmol. 1999 Jun;10(3):221-6. doi: 10.1097/00055735-199906000-00012. PMID: 10537783

[2] (dernière consultation le 25/03/2021)

[3] Grössmann N, Robausch M, Rothschedl E, Wild C, Simon J. Publicly accessible evidence of health-related quality of life benefits associated with cancer drugs approved by the European Medicines Agency between 2009 and 2015. Eur J Cancer. 2020 Apr;129:23-31. doi: 10.1016/j.ejca.2020.01.020. Epub 2020 Feb 28. PMID: 32120272



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