Proposals to curb high drug prices are hardly evidence-based

26-03-2020

Solutions proposed to curb the sky-high prices of new anti-cancer drugs, and thus ensure access to them, are insufficiently empirically and quantitatively substantiated, according to a study by researchers from the Netherlands Cancer Institute. The results of their study were published in the scientific journal JAMA on 26 March 2020. The researchers are currently investigating several promising policy proposals using game theory and computer simulations.

Health care system under pressure
More and more new anti-cancer drugs are being brought to market, but the prices of these drugs, such as immune therapies or targeted drugs, are often excessively high, even when they are first introduced. As a result of this, our health care system is under serious pressure, potentially limiting access to these drugs for people who need them. For this reason, the scientific literature proposes various solutions to curb sky-high drug prices. But how evidence-based are these solutions?

Combing through scientific literature
To investigate this, Wim van Harten of the Netherlands Cancer Institute – research leader of Health Technology Assessment in addition to his position as a Professor at the University of Twente – and his colleagues combed through almost 5,000 scientific literature articles in search of empirical and quantitative evidence for the proposed policy solutions.

Solutions insufficiently substantiated
Their conclusion: the justification is insufficient across the board. The vast majority of the proposed solutions are based on qualitative arguments, are hardly evidence-based, and often do not go beyond the level of an educated guess. In fact, the researchers conclude that this literature does not give enough ammunition to advise governments on this important problem.

IP, R&D, and pricing
The researchers distinguish three overarching domains in which prices can be controlled: intellectual property (e.g. through admitting more competitors), the way in which drug pricing is set (e.g. through profit regulation) and the domain of research & development (e.g. bringing drug studies into the public domain).

Gaming and computer simulations
Van Harten and his colleagues did, however, identify a number of promising approaches that deserve more thorough and innovative investigation, using game theory, computer simulations, and real-life pilot studies, for example. They have in the meantime taken up this gauntlet themselves, together with University of Twente researchers and a team of microeconomists led by Theo Offerman of the University of Amsterdam. This should lead to a number of pilot project proposals that can then be tested in practice.

These promising approaches are:

Separating R&D and production
Transferring drug research and clinical trials from the pharmaceutical industry to the public sector
Increasing transparency across the board: in R&D costs, in the results of drug studies, and in the way in which prices are set
Reforming the Orphan Drug Act
Tiered pricing model: high entry-level price but low user costs (the so-called Netflix model)
International or large-scale purchasing

Proposals to curb high drug prices are hardly evidence-based

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