“Adaptive pathways” to drug authorisation: adapting to industry?

After the thalidomide tragedy governments in Europe and North America established new requirements for drug testing intended to protect the public from ineffective and unsafe drugs. As no drugs are entirely safe, regulators must determine whether manufacturers have provided enough evidence to show that drugs’ benefits outweigh their harms. Precisely how high the evidence bar should be set for marketing authorisation has been hotly disputed ever since the enactment of modern drug regulation. The industry has long contended that the process is too complex and expensive, delaying patients’ access to lifesaving drugs.

Despite three decades of streamlining of regulatory processes and drug testing,1 the industry, some patients’ advocates, lobbyists, and investors still argue that the current model of drug development and regulation is unsustainable. They have called for a “paradigm shift” to allow greater numbers of new drugs to be approved on the basis of preliminary data, with key information about benefits and harms to be collected only after drugs have come on the market and are being used by patients.2 There may be limited circumstances that justify rapid access to new drugs on the basis of minimal data, but evidence indicates that regulators have been too permissive in their interpretation of existing criteria for expedited approval,3 that current regulatory standards are already too lax, and that low standards have failed to incentivise genuine therapeutic innovation.1 4 5 6

The European Medicines Agency (EMA) has embraced a new model of drug testing and marketing called “adaptive pathways”—the brainchild of an industry funded think tank, NEWDIGS (New Drug Development Paradigms), at the Massachusetts Institute of Technology. The adaptive pathways model would skip several steps designed to protect …