FDA in Debate over Developing, Pricing New Cancer Therapies

Amidst hundreds of presentations and seminars on new developments in cancer research and treatment discovery at the annual meeting of the American Association for Cancer Research (AACR) in Washington, D.C. this week, FDA policies for speeding promising new therapies to patients garnered a good deal of attention. Richard Pazdur, director of FDA’s new Oncology Center of Excellence (OCE), was center stage, literally, commenting on broad R&D policy issues raised in a keynote speech on the promises and challenges of the Cancer Moonshot, particularly as it has lost its home in the White House.

Pazdur explained that OCE is looking for ways to improve how clinical trials are conducted, possibly by merging phases 1, 2 or 3, and by examining whether clinical trial eligibility criteria can better reflect real world situations. Greater disclosure of key information before drugs are approved is under examination as FDA strives not just to approve more drugs, but to ensure that effective therapies get to patients.

A unique session billed unofficially as “Pazdur’s revenge” gave the FDA official an opportunity to question leading science and business reporters on how they write about cancer research and drug development. Twenty years ago, he noted, the pharmaceutical industry had little interest in research involving cancer therapies, which were associated with high toxicity and limited gain in extended life. Now 40% of new breakthrough therapies are cancer treatments, Pazdur noted, a development that the media panel attributed to important scientific discoveries, plus a favorable regulatory environment at FDA.

Pazdur raised the issue of whether FDA is approving too many or too few breakthrough drugs, and the media commenters said that the designation may be handed out too often. But they agreed with Pazdur that the breakthrough program is most helpful in streamlining the regulatory process. The FDA official observed that the real value of the breakthrough program is not speeding a specific drug to market, but in bringing about a “cultural change” at FDA. The program encourages agency reviewers and industry sponsors to communicate more closely, particularly in planning timelines for review, he said, adding that if reviewers have a question, now they will just go call the sponsor, and not wait for the next scheduled meeting.

Another factor spurring more industry investment in cancer research is a predictable economic situation, the panel agreed. Drug prices are the “big gorilla” in the room, commented Adam Feuerstein, who writes TheStreet.com blog on the biopharmaceutical industry. Five years ago or so, no one talked about drug pricing, but now every story addresses that issue. Matt Herper of Forbes magazine noted that the free market hasn’t worked to restrain drug prices, leaving patients bearing too much of the costs. Laurie McGinley of the Washington Post pointed out that more combination and triple therapies, with $150,000 price tags multiplied by three, would be unaffordable for everyone.

Noting that industry is focusing more on promoting drug value, Meg Tirrell, a reporter for CNBC, questioned whether “value” has any meaning these days, while Feuerstein agreed that it’s hard to put a number on drug value.