AN ANALYSIS OF REAL WORLD DATA USE BY GLOBAL MARKET ACCESS STAKEHOLDERS

• Published in: Value in health 2017-05, Vol.20 (5), p.A61
• Main Authors: Tarlecki, A, Horowicz-Mehler, N, Powers, E, Doyle, JJ
• Format: Article
• Language: English
• Subjects: Advisory committees; Decision making; Delayed; Electronic publishing; Health services; Market entry; Medical equipment; Medical research; Medical supplies; Public health; Reimbursement; Stakeholders
• ISSN: 1098-3015; 1524-4733
• DOI: 10.1016/j.jval.2017.05.005

OBJECTIVES: There are clinical and financial impacts of not having real-world data (RWD) upon market entry such as delayed approval, suboptimal reimbursement, and unfavorable re-evaluation. We aimed to assess the use of RWD for market access (MA) decisions globally and trends in the number of RWD studies to evaluate growing interest in RWD. METHODS: A global search was conducted in the HTA Accelerator©database from 2008 to 2016 for any recommendation of conditional reimbursement or approval requiring collection of RWD. An online search was conducted in the United States, United Kingdom, Australia, and Canada for uses of RWD by regulators and payers. Clinicaltrials.gov was searched for trends in the number of “observational” studies in the US and UK registered from 2010 to 2016. RESULTS: 17 HTAs recommended reimbursement or approval conditional on providing additional economic (7) or clinical (10) RWD. In the UK, the National Health Service is ramping up RWD capabilities by integrating clinical datasets. In Australia, the Pharmaceutical Benefits Advisory Committee is supporting risk-based approaches that condition reimbursement on RWD collection. The Canadian Agency for Drugs and Technologies in Health recently published a framework that suggests RWD may play a role in recommendations for orphan diseases. In 2016, the US issued a draft guidance on the use of RWD to support regulatory decision-making for medical devices. There was an increase in RWD studies in the UK, from 15.7% in 2010 to 21.5% in 2016 of all observational and interventional studies. RWD use declined in the US over the same period, from 18.2% to 15.1%. CONCLUSIONS: With the recognized value of RWD in MA decisions, there is a need to monitor stakeholders’ use of RWD to optimize treatment access. There is also a need to approach evidence generation systematically to differentiate assets beyond approval and initial P&R.