The 70-page report is online here. Trade press at BioPharmaDive here.
The report focuses on cancer drug prices and the rising number of therapies that are priced >$100,000 per year. From several word searches, although focused on current cancer policy, the report has virtually no discussion of genetics, genomics, precision medicine, or diagnostic testing. (The word "test" only occurs in contexts like "testing new payment models.")
Right to Try
In other news today, Right to Try legislation failed to reach a 2/3 majority vote in the House, falling 7 votes short. Right to Try is "access to experimental options for life threatening illnesses when patients have run out of options." It was supported by most Republicans and by the President per his State of the Union speech. US regulators were generally not in favor, pointing out there are already "compassionate use" frameworks that are similar to the right-to-try concept, but follow trackable channels. In October 2017, FDA commissioner Gottlieb "raised concerns" about the bill, as did Deputy Commissioner Rachel Sherman and CBER director Peter Marks. I've borrowed from a trade article here.
Stem Cell Therapies & FDA
Stem cell therapies have, in some cases, been relatively unregulated as "practice of medicine," hence the billboards and odd clinics and claims we see in California. FDA has vowed to crack down on that. In a new article in NEJM, FDA outlines its path for regulation and approval of stem cell therapy products. Trade press here. The article cites to four recent FDA guidance documents. The article states:
Such an approach is just one example of how the FDA is taking an original policy approach to the regulation of a highly innovative field, one in which our traditional approach to regulation may not be as efficient or effective as in more mature fields.
As part of its efforts in the area of regenerative medicine, the FDA is also encouraging investigators who are involved in innovative product development to engage in dialogue with the agency early on in the process, including through informational meetings, before more formal discussions are held about submitting an application for an investigational new drug.... Our aim is to refashion our traditional tools for regulation to meet the challenges and opportunities presented by such highly innovative products as cell-based regenerative medicine.
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Richard Rettig's 1977 book on the National Cancer Act of 1971: "Cancer Crusade," Princeton. Legislative history was a carnival and the history traced back a decade.
