The Bureau of National Affairs
Health Care Daily Report
FDA: Agency Should Speed Drug Approvals To Benefit Patients, According to Report
By Bronwyn Mixter
15 June 2010
Reproduced with permission from Daily Report for Executives, 113 DER A-2 (June 15, 2010).
Copyright 2010 by The Bureau of National Affairs, Inc. (800-372-1033)
The Food and Drug Administration should take steps to speed drug approvals to provide patients with quicker access to lifesaving medicines, according to a report released June 14 by the Manhattan Institute's Project FDA.
The report, Cost of Caution: The Impact on Patients of Delayed Drug Approvals, puts a price tag on the value of longer lives that would have resulted from getting new drugs to patients faster. Specifically, the report uses an economic model to determine the monetary value of decreased lifespan due to delays in drug development. The study examines three classes of approved and marketed drugs, including highly active antiretroviral treatment (HAART) for the treatment of HIV/AIDS, Herceptin (trastuzumab) for the treatment of breast cancer, and Rituxan (rituximab) for the treatment of non-Hodgkin's lymphoma.
To a single HIV-AIDS patient being treated with HAART, a year's earlier access would be worth $16,000, and $46,000 for three years' earlier access, the report said. For all HIV-AIDS patients being treated with HAART, a year's earlier access would be worth $19 billion and three year's earlier access would be worth $53 billion.
While the drug-approval process, once FDA receives a drug application, is faster today than in 1993, when Congress passed the Prescription Drug User Fee Act (PDUFA), the pre-application process has only grown slower and more costly, the report said. With FDA starting hearings and meetings regarding the reauthorization of PDUFA, Congress has an opportunity to expand on the legislation's original goals and streamline the pre-application process.
For all breast cancer patients being treated with Herceptin, the value of one year's earlier access would be $8 billion and three years' earlier access would be worth $22 billion, the study said. For patients being treated with Rituxan for non-hodgkin's lymphoma, a year's earlier access would be worth $310 million and three year's earlier access would be worth $850 million. By contrast, shortening phase III clinical trials by one year would save the typical drug company only about $40 million, the report said.
Specific reforms recommended in the report include the following:
Allowing the payment of stipends to clinical trial volunteers. Doing so, after obtaining informed consent, would address the shortage of volunteers, the report said.
Relying on biomarkers. Biomarkers are by-products of disease processes (such as elevated numbers of CD4 cells in AIDS cases) or indicators of disease risk (such as cholesterol levels). Their use would allow researchers to estimate the likelihood of a treatment's success before completion of full clinical trials, the study said.
Offering FDA regulators performance incentives. These could expedite the identification of promising new treatments or the evaluation of new guidelines for clinical trials.
Establishing an ombudsman. Such an official would review the impact of FDA regulations and practices on drug development times.
Tomas J. Philipson, chairman of Project FDA and managing director at Precision Health Economics, and Eric Sun, a resident in the department of anesthesiology at Standard University and a visiting fellow at the Bing Center for Health Economics at the RAND Corp., co-authored the report.