New initiative would speed FDA approvals

Bob GattyWashington-For urologists and other health care practitioners-and anybody who cares about having medicines to treat whatever disease might befall them-there is positive, bipartisan activity underway in Washington.

Despite the common perception of Congress being filled with a bunch of politically obsessed do-nothings, there actually is a determined and dedicated group of lawmakers and staff assistants who are working practically 24-7 to develop a sweeping new initiative that would help speed drug and device approvals and increase funding for new drug innovation.

Bill includes $10 billion for innovation fund

The so-called 21^st Century Cures initiative, released in an updated 200-page document last week by bipartisan leaders of the House Energy and Commerce Committee, would steer $10 billion in mandatory spending into a National Institutes of Health (NIH) innovation fund over 5 years and authorize another $1.5 billion in discretionary dollars in each of the next 3 years. The innovation fund would be used for precision medicine, including funding the work of emerging young scientists.

The draft bill was released by Committee Chairman Fred Upton (R-MI); ranking minority member Frank Pallone, Jr. (D-NJ); Rep. Diana DeGette (D-CO); ranking minority member of the House Oversight and Investigations Subcommittee; Health Subcommittee Chairman Joe Pitts (R-PA); and ranking Health Subcommittee member Gene Green (D-TX). Upton said he would have a completed bill on the House floor for consideration in June.

NEXT: Measure contains numerous provisions affecting the FDA

The measure contains numerous provisions affecting the FDA, including reforming clinical trials and establishing an expedited approval pathway for certain medical devices, vaccines, and antibiotics.

A committee statement accompanying the discussion draft said it includes provisions to:

• foster development of treatments for patients facing serious or life-threatening diseases

• repurpose drugs for serious or life-threatening diseases and conditions

• modernize clinical trials

• help the development of personalized and precision medicines so the right patient can receive the right treatment at the right time

• provide for continued work in telehealth

• advance a truly interoperable health system.

While work has been underway on the Cures initiative for more than a year, lawmakers have been unable to reach consensus on several key points, including how to encourage drug makers to take on the costly and time-consuming task of creating new drugs for rare diseases.

Initially, the plan was to grant longer exclusivity periods for manufacturers of seldom-used therapies for unmet medical needs, as well as giving U.S. generic drug manufacturers a longer protection time frame from foreign competitors. Both provisions have been dropped from the latest draft, as some advocacy groups raised concerns that a longer exclusivity period would increase costs to consumers while they waited for competitive versions to become available.

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Other unresolved issues include areas related to telemedicine, the interoperability of health records, and repurposing FDA-approved drugs for other uses. However, there were reports that agreement may have been reached on some of these issues, including repurposing drugs.

During an Energy and Commerce Health Subcommittee hearing April 30, Pitts said an Energy and Commerce Committee Working Group on Telemedicine is working toward a “bipartisan proposal that will encourage the use of telemedicine services to improve health care quality and outcomes, increase patient access, and control costs.”

On repurposing drugs, Upton said at the hearing, “As we move through the process to markup, we will continue to work on a policy to provide incentives to develop drugs that, while they may have failed in trials for one indication, show promise to treat patients facing other serious or life-threatening diseases.”

NEXT: Proposal raises concerns over safety

Proposal raises concerns over safety

The legislation includes a streamlined process for the FDA to approve breakthrough therapies. It would allow the FDA to approve drugs with “early state clinical safety and effectiveness data,” giving manufacturers the responsibility of conducting post-approval studies. That provision worries watchdog and consumer groups.

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“The bill emphasizes speed over safety,” said Vijay Das, health care policy advocate at Public Citizen.

There is agreement by many of the lawmakers involved in the development of the legislation that both the FDA and NIH need more money to handle their additional responsibilities, a difficult hurdle in today’s political environment.

“We are asking the FDA to make many changes to its current operation,” said Rep. DeGette, a lead sponsor of the bill with Upton. “We should make sure the agency has the resources to carry out these duties.”

Jeffrey Shuren, director of the FDA’s center for devices and radiological health, said the FDA’s purchasing power has declined over the past decade even as Congress expanded its responsibilities.

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Shuren cautioned that routine regulatory tasks could be more difficult to accomplish if the agency is given even more responsibilities without adequate funding to provide the necessary staff resources.

“We are excited that NIH gets more money,” said Shuren, “but all these great things don’t get out to the market unless we are in a position to help out. The more things that are piled up on people’s plates, the more they’re set up for failure.”

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