• PDF / 1,213,780 Bytes
• 7 Pages / 595.276 x 790.866 pts Page_size
• 97 Downloads / 241 Views

DOWNLOAD

REPORT

---

COMMENTARY

Bridging the Gap Between RCTs and RWE Through Endpoint Selection Robert J. LoCasale1 · Chris L. Pashos2 · Ben Gutierrez3 · Nancy A. Dreyer4 · Toby Collins5 · Alan Calleja5 · Michael J. Seewald6 · Jonathan M. Plumb7 · Johan Liwing8 · Maurille Feudjo Tepie9 · Sajan Khosla10  Received: 9 March 2020 / Accepted: 23 June 2020 © The Drug Information Association, Inc 2020

Abstract This commentary is authored by several industry real-world evidence (RWE) experts, with support from IQVIA, as part of the ’RWE Leadership Forum’: a group of Industry Leaders who have come together as non-competitive partners to understand and respond to RWD/E challenges and opportunities with a single expert voice. Here, the forum discusses the value in bridging the industry disconnect between RTCs and RWE, with a view to promoting the use of RWE in the RCT environment. RCT endpoints are explored along several axes including their clinical relevance and their measure of direct patient benefit, and then compared with their real-world counterparts to identify suitable paths, or gaps, for assimilating RWE endpoints into the RCT environment.

Introduction What underlies the choice of a study endpoint? In today’s healthcare industry, this decision is highly nuanced and influenced by a host of factors including the study’s intended audience (regulatory, health technology assessment (HTA)/ payer, HCP or patient) or the provenance of data utilized. Mounting evidence pressures on drug manufacturers to differentiate their products in congested therapeutic markets can necessitate demonstrating more than drug safety and efficacy data alone. Here, real-world evidence (RWE) with Sajan Khosla is the Head of RWE, Oncology R&D. * Sajan Khosla [email protected] 1



Real World Evidence, Sanofi, Bridgewater, NJ, USA

2



AbbVie, Chicago, IL, USA

3

GSK, Philadelphia, PA, USA

4

IQVIA, Real-World Solutions, Cambridge, MA, USA

5

IQVIA, Real-World Solutions, London, UK

6

Novartis Pharma AG/Pharma Medical Affairs, Basel, Switzerland

7

Ferring Pharmaceuticals SA, St Prex, Switzerland

8

CellProtect Nordic Pharmaceuticals, Stockholm, Sweden

9

Center for Observational Research, Amgen, Cambridge, UK



10

Real-World Evidence Center of Excellence, AstraZeneca, Cambridge, UK

appropriately selected endpoints has the potential to support key regulatory and market access decisions by broadening the value message of a medicine with evidence of real-world effectiveness, including direct assessment of patient benefit-risk. As the real-world data (RWD) landscape advances, RCTs that are complemented or supplemented with RWE have potential to generate evidence for an expanded audience of stakeholders, from regulatory bodies to patients [1]. Moreover, RWD that is “fit for purpose” (i.e. sufficiently comprehensive and complete to answer research questions and objectives) and readily accessible has potential to reduce the data collection and cost burdens of traditional RCTs that are time-consuming and expensive to conduct. Yet, as it stands, th