Can adaptive clinical trials help to solve the productivity crisis of the pharmaceutical industry? - a scenario analysis

Jörg Mahlich,Srirangan Dheban,Arne Bartol

doi:10.1186/s13561-021-00302-6

Abstract

AimThe productivity of pharmaceutical research and development (R&D) investments is declining due to high failure rates in clinical research. Recently, the US Food and Drug Administration (FDA) acknowledged that adaptive designs can make drug development more efficient and less costly. Our objective is to simulate cost-saving effects and estimate the impact on global R&D expenditures as well as possible outcomes measured in life-years gained.MethodsBased on published drug-development cost data we calculate potential cost savings derived from variations in clinical success rates that result from employing adaptive trial designs. In a subsequent step we estimate how those cost changes affect global R&D expenditures and outcomes.ResultsOur calculations indicate that an adaptive trial design with the potential to increase success rates of clinical trials by 4 percentage points could lower development costs for a new drug from 2.6 to 2.2bn USD. On a global scale, this cost reduction would free up an additional 4.2bn USD for investment into pharmaceutical R&D to bring about drug innovations that in turn would be capable of generating up to 3.5 million life-years.ConclusionNew clinical trial designs are crucial to improving productivity within the pharmaceutical industry and to fostering a sustainable health-care system.

Highlights

The increasing health care expenditure as share of the Gross Domestic Product (GDP) has been subject to considerable political debate
(2021) 11:4 study found that the number of new drugs approved per billion US dollars spent on research and development (R&D) has halved roughly every 9 years since 1950, which means a drop by a factor of 80 in inflation-adjusted terms
Based on the assumptions stated above, the increase in clinical success rates would reduce the total costs of bringing a new drug to the market from 2.56 bn USD to 2.19 bn USD (− 14.4%) and 2.39 bn USD (− 6.6%), which is the result of a 25% and a 11.5% decrease, respectively, or 1.3bn USD) of the clinical phase (Fig. 4)

Summary

Introduction

The increasing health care expenditure as share of the Gross Domestic Product (GDP) has been subject to considerable political debate. The Organisation for Economic Co-Operation and Development (OECD) concluded in a report that health-care costs in advanced economies are rising so fast as to become unaffordable by mid-century if there are no reforms [1]. It was the economist William Baumol who coined the term “cost disease” when he offered the explanation that the increases in health-care spending can be attributed – at (2021) 11:4 study found that the number of new drugs approved per billion US dollars spent on R&D has halved roughly every 9 years since 1950, which means a drop by a factor of 80 in inflation-adjusted terms. As Paul et al [6] put it: “Without a substantial increase in R&D productivity, the pharmaceutical industry’s survival (let alone its continued growth prospects), at least in its current form, is in great jeopardy.” Paul et al believe that a cost reduction of 50% per new chemical entity (NCE) will be needed to sustain a viable business model

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