Representatives from the pharmaceutical and biotechnology industries said last Friday (Dec. 12) they expect work on the bipartisan 21st Century Cures initiative this spring to be a staging ground for negotiations over the sixth iteration of the Prescription Drug User Fee Act (PDUFA) in 2016 and 2017. Industry leaders also said they expected the biotechnology industry to enter the negotiations with FDA as an equal partner with the pharmaceutical industry, speaking on a PDUFA VI panel at an IIR-sponsored FDA/CMS Summit in Washington.
Brian Harvey, vice president of Global Regulatory Affairs for Pfizer, echoed the sentiments of other panelists saying that 21st Century Cures will be a “dry run” for PDUFA.
“What better way to really sort of formulate your ideas and vet them to make sure they really hold water than to have to do it that much earlier,” Harvey said. “I’m very optimistic that something may come out of 21st Century Cures, but even for those things that don’t make it through the process we’re that much further along for PDUFA VI.”
The 21st Century Cures initiative is a bipartisan effort spurred by House Energy and Commerce Chair Fred Upton (R-MI) and Ranking Minority Member Diana DeGette (D-CO) that is expected to include a broad spectrum of provisions aimed at spurring development of new drugs and devices, and may bump up research funding at the National Institutes of Health. Upton said last month he expects to have a draft of the 21st Century Cures bill ready in January, with hopes of getting a full bill to the House floor by Memorial Day. Janet Woodcock, director of FDA’s Center for Drug Evaluation and Research (CDER), said at an earlier panel during the FDA/CMS Summit that the 21st Century Cures initiative is a “front burner” priority for the agency.
PDUFA was first passed in 1992 and updated in 1997, 2002, 2007 and 2012. The next version — PDUFA VI — needs to be passed by Sept. 30, 2017, when the latest version is set to expire.
Harvey said PDUFA VI needs to include a focus on modernizing the way clinical trials are done, adding that clinical trials are becoming unsustainable as the cost for a single trial is rising above $1 billion.
“We really need to use new technology,” Harvey said. “We need to use 21st century regulations and not 18th or 19th century perspectives to run clinical trials and do it in a more efficient way.”
Kay Holcombe, senior vice president for Science Policy at the Biotechnology Industry Organization (BIO), said the 21st Century Cures legislation will also help determine what resources FDA needs going forward that can be built into PDUFA. She said FDA gets “saddled” with a multitude of requirements in PDUFA updates, but finds that user fees don’t cover the costs of meeting those requirements. The 21st Century Cures bill is expected to impose new requirements on the agency, so FDA will know what it needs to fulfill those requirements as PDUFA VI is negotiated, Holcombe said.
“It gives us a good base of information as we’re going into PDUFA VI in terms of the resources the agency is going to have to come up with in order to do things out of 21st Century Cures,” Holcombe said.
When asked whether industry should be paying more or all of the costs for the drug approval process — user fees account for roughly 70 percent of the costs currently — Holcombe said it is highly dependent on what the benefits for drug makers are.
“I don’t want to sound crass, but when you put money on the table you’re kind of wondering what it is that you’re getting back,” Holcombe said.
She said industry may be supportive of paying a higher share if that leads to reducing the time and cost associated with developing and producing new drugs.
Holcombe also said that the “industry will speak with one voice” during PDUFA negotiations with BIO on equal footing with the pharmaceutical industry — noting that the two groups have already been talking and working with each other in this regard.
“BIO very wants to be, obviously, not taking a back seat to PhRMA, but working with PhRMA as an equal partner,” she said.
Robert Metcalf, the vice president of Global Regulatory Affairs for Eli Lilly who will be heading the Pharmaceutical Research and Manufacturers of America’s (PhRMA) regulatory affairs arm starting next year, said PhRMA views its relationship with BIO going forward as “very much of a joint partnership across BIO and PhRMA as equals.”
Metcalf and patients’ advocates on the panel also said PDUFA VI will build on PDUFA V’s efforts to open up the drug development and regulatory process to patient’s concerns.
Jeff Allen, executive director of patient advocacy group Friends of Cancer Research, said PDUFA V built the framework for including patients in the conversation and has created an opportunity for drug makers to build those concerns into their trials.
“It has to start earlier than it historically has in terms of understanding what patient outcomes are desired and incorporate those into the experimental process,” Allen said.
Metcalf said PDUFA VI will be an opportunity to began incorporating patient input into the development process in a scientific and systematic way.
“Patient input can and should be not only a motive, but also scientific as well,” Metcalf said. “That’s the opportunity I see going forward. We’ve got to bring in skills and capabilities that we haven’t necessarily done, like social science capabilities.”