Editors' ChoicePolicy

System-level prescriptions for adaptive drug licensing

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Science Translational Medicine  04 Mar 2015:
Vol. 7, Issue 277, pp. 277ec36
DOI: 10.1126/scitranslmed.aaa9493

The next generation of therapeutics is increasing in complexity, while at the same time, being honed to smaller segments of the population. As statisticians churn over how to match the future of translational medicine to the “adequate and well powered” evidence standard required for regulatory approval, an open question remains as to how the accelerating pace of product innovation can be accommodated in the real world. In a highly ambitious publication, Eichler et al. take this subject head on. The group argues that the key to providing timely patient access to future therapeutics requires expanding beyond a one-dimensional view of regulatory policy, incorporating multiple stakeholders and an ongoing assessment of risks and benefits throughout the full life cycle of a drug. Following a worldwide trend of drug approvals being balanced with requirements for post-licensing data collection, Eichler et al. identify six real-world transitions required to take this concept to the next level. Particularly interesting is a recommended shift from a “single, gated licensing decision” to a “life span management” approach that would lower risks through “learning-confirming-(re)licensing.” The group goes so far as to describe a future de facto standard of “adaptive licensing,” where nearly all product licenses would be dynamically related to data sourced from clinical practice. Critical to this future, they argue, are improved tools to generate both randomized and observational assessments of benefit-risk after an initial product license has been granted.

One could say that a licensing pathway requiring the alignment of not one but multiple billion-dollar stakeholders is about as easy as a moonshot. Yet, the power and coherency of the life cycle approach is reflected in the remarkable list of contributors to the work of Eichler et al. With 41 coauthors sourced from the largest payers, pharmaceutical companies, regulators, and patient advocacy groups in the world, this mega-stakeholder opinion paper certainly gives an interesting consideration for a new trajectory in drug licensing.

H.-G. Eichler et al., From adaptive licensing to adaptive pathways: Delivering a flexible life-span approach to bring new drugs to patients. Clin. Pharmacol. Ther. 10.1002/cpt.59 (2015). [Abstract]

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