Webinar: Harnessing Patient Reported Outcomes
Biotechnology is one of the most attractive of fact growing industries with revenues amounted to USD 137 billion in 2017 and an estimated annual growth rate of more than 10% p.a. In terms of clinical development, the focuses of the Biotech companies are to demonstrate clinical proof of concept at controlled costs, build enough data to gain regulatory approval worldwide, maximize the economic value of their NCE (New Chemical Entity), guarantee broad market access. So, NPV (Net product value) is high enough to facilitate licensing out or company acquisition in the best economic conditions.
WHO defined health as “A state of complete physical, mental and social well-being …”. Any health goods that demonstrate an impact on one of the 3 above dimensions become a candidate for health care market access worldwide. Patient reported outcome (PRO) is the measure of physical, mental and social functioning, and associated to life expectancy, is a key driver of NCE economic value.
The principle of “value-based pricing” is fixed by Weinstein since 1977: “Limits on health-care resources mandate that resource-allocation decisions be guided by considerations of cost in relation to expected benefits”. Today, evidencing scientifically the benefits of NCEs over current standard of care is requested by almost all Western developed countries to be priced and reimbursed. PRO plays a major role in estimating the benefits.
Incorporating PRO in a phase II trial impacts marginally its management and cost. Time to access the market will remain unchanged with no impact on patient recruitment. While PRO-related recommendations (FDA, EMA, other agencies) must be followed, on-site patient management, data flow, statistical analysis will be moderately impacted, since PRO activities are performed in parallel to the usual clinical activities.
By using the PRO results and applying the principles of health economics, the biotech company can justify the breakeven price of its NCE, based on scientific grounds collected at the end of the phase II, mimicking the post-MAA pricing negotiation. This information becomes one of the value drivers of the NPV of the NCE and is therefore a key item for Mergers & Acquisitions or licensing-out businesses.
Chief Medical Officers/ Medical Directors
About the Speaker
Gilles Berdeaux is experienced in market access, health economics, patient reported outcomes and real-world evidence. Medical doctor by training, with a PhD (DEA) in epidemiology and biostatistics, Gilles gained expertise in biostatistics, epidemiology and health economics, with a focus on quantitative evaluation of public health goods. After 8 years as Head of Biometrics in a CRO (Clinica and Statisica) and at Bayer Pharma, Gilles worked in the field of health economics and outcome research, patient reported outcomes, real-world evidence and market access in various organizations (Pierre Fabre, Alcon-Novartis, Quintiles and IMS health) for 25 years.
Patient reported outcomes are an important part of Gilles' projects, including questionnaire development, psychometric validation, both quality of life and satisfaction questionnaire, and use of PRO in various contexts.
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