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

Abstract

Aim The 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. Methods Based 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. Results Our 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. Conclusion New clinical trial designs are crucial to improving productivity within the pharmaceutical industry and to fostering a sustainable health-care system.

Innovative Designs and Logistical Considerations for Expedited Clinical Development of Combination Disease-Modifying Treatments for Type 1 Diabetes

“…From a financial point of view, conservative assessments estimate that drug companies stand to save on the order of $100 million/year in drug development costs when early go-no decisions afforded by adaptive trials are in place for testing candidate therapies (85). Moreover, conservative estimates suggest that adaptive trial designs can increase success rates of clinical trials by 4%, which translates into lowering development costs for a new drug from $2.6 to $2.2 billion.…”

“…This cost reduction would free up an additional $4.2 billion globally for pharmaceutical research and development. This opportunity should serve as a catalyst for much-needed master protocols to find DMTs that quantitatively equate with the generation of up to 3.5 million life-years (85).…”

“…This versatility also allows a mechanism for investors to diversify and de-risk their investments, and increase their investments in response to promising milestones, based on real-time measurements of the net present value of a drug. The probability of attracting pharmaceutical licensees is expected to similarly increase when promising milestones occur and may even enable events like an early end to a phase 2 trial that accelerates phase 3 start (85,86). To this end, there may be a vital role for funding agencies to pioneer the establishment of adaptive clinical testing vehicles that incentivize and de-risk T1D opportunities through early clinical proof-of-concept testing.…”

Practical guidance for planning resources required to support publicly-funded adaptive clinical trials

“…The key factor that justifies an AD is the ratio of benefit it provides (both to the efficiency and patient benefit of the trial itself and the long-run impact of the evidence generated) to the additional cost it incurs [ 44 , 45 ]. Research that can inform and improve both parts of this ratio is needed.…”

Adapt to Translate

“…From another vantage point, the pharmaceutical industry is voicing hopes about the usage of adaptive trials as a means to end the productivity crisis (Mahlich, Bartol, and Dheban 2021). In this section, I place the emergence of adaptive trials in a wider context of biomedical movements initiated to improve the productivity and cost-benefit of biomedical research.…”