21st Century Cures and Real World Data

  • by: |
  • 04/30/2015

FDA could approve drugs for new uses on less data: draft law

 (Reuters) - Draft U.S. legislation released on Wednesday could make it easier for drug companies to win Food and Drug Administration approval of products for new uses.

Currently a company with a drug approved for lung cancer must conduct additional studies if it wants to market it for breast cancer.

A bill drafted by the House Energy & Commerce Committee's health panel would eliminate the need for randomized, controlled clinical trials, the gold standard for assessing whether a product is safe and effective.

Instead companies could submit data from observational studies, in which researchers have no control over the experiment, ongoing surveillance studies and other clinical experience.

"Calling for the FDA to use this data is pretty revolutionary," said Peter Pitts, a former FDA associate commissioner for external relations and co-founder of the industry-funded Center for Medicine in the Public Interest. "In the past this kind of data was not considered gold standard."

If included in the final version of the bill, known as 21st Century Cures, "it really would allow the FDA to have a broader view of how the drugs work in the real world," he added.

In addition, the FDA would be allowed to approve new indications based on a review of clinical data summaries, rather than full packages, potentially speeding up the approval time.

The bill would also require the agency to consider using real world experience as opposed to randomized trials to support or satisfy requirements for post-market studies.

The FDA frequently approves drugs based on "surrogate" endpoints that are expected to reflect clinical benefits. If a drug causes a tumor to shrink there is an expectation it could also delay progression of the disease or prolong life.

But companies are required to conduct additional trials to confirm that the expected benefit actually materializes. The bill would reduce the need for such trials.

It would also make it easier for companies to provide economic analyses to insurance companies and others involved in reimbursement. A company with a high-priced drug might want to show why it is more economical than others in the long run.

A prior version of the bill was circulated for discussion earlier this year. A parallel bill is being developed in the Senate.


Center for Medicine in the Public Interest is a nonprofit, non-partisan organization promoting innovative solutions that advance medical progress, reduce health disparities, extend life and make health care more affordable, preventive and patient-centered. CMPI also provides the public, policymakers and the media a reliable source of independent scientific analysis on issues ranging from personalized medicine, food and drug safety, health care reform and comparative effectiveness.

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