Kaiser Permanente is proud to partner with the Program on Regulation, Therapeutics, and Law (PORTAL), out of the Division of Pharmacoepidemiology and Pharmacoeconomics at Brigham and Women’s Hospital/Harvard Medical School, to uncover trends in pharmaceutical evidence generation and highlight the clinical benefits associated with newly approved drugs. Through this partnership, the PORTAL team has published five prominent peer-reviewed studies so far.
For example, Analysis of Supportive Evidence for US Food and Drug Administration Approvals of Novel Drugs in 2020, published in JAMA Network Open, highlights the quality of evidence supporting new drug approvals, finding in particular that pivotal trials for cancer drugs were overwhelmingly non-randomized and likely to use a surrogate measure as the main endpoint. A review of new drug approvals in 2018-2019 also published in JAMA Network Open found that only about one-third of them were rated as providing moderate or greater improvements over existing treatments by international health technology assessment organizations (Therapeutic Value Assessments of Novel Medicines in the US and Europe, 2018-2019). One response to diminishing pre-approval evidence is to require manufacturers to conduct more post-approval confirmatory studies, but in New Drug Postmarketing Requirements and Commitments in the US: A Systematic Review of the Evidence, published in Drug Safety, the team found that manufacturers’ completion of any post-approval requirements attached to new drugs has been of uneven quality and timeliness over the last 2 decades.