House Energy & Commerce chair Fred Upton (R-MI) and Rep. Diana DeGette (D-CO) on Tuesday laid out five broad goals of the 21st Century Cures draft bill they plan to introduce later this month: targeting clinical trials to the “right group” of patients; better integrating the patient perspective into the regulatory process through public-private partnerships; promoting better access to and sharing of data among researchers; increasing and targeting funding for scientific research; and incentivizing new drugs and devices for unmet medical needs by relying more on post-market studies and possibly offering exclusivity or reimbursement incentives.
In a Jan. 13 CNN op-ed on the upcoming bill, Upton and DeGette write that after a year of hearings on the initiative they are ready to put forward bipartisan legislation that will promote finding cures for 7,000 diseases and ensure the United States is a leader in the field of medical innovation.
“Our legislation will modernize all of these categories, removing outdated hurdles that can prevent innovation from flourishing or from fleeing the United States.,” Upton and DeGette write.
The bipartisan duo do not detail specific provisions of the forthcoming draft, but lay out brief justifications of the four goals laid out in the piece that offer a framework for the legislation.
Streamlining clinical trials. The lawmakers say clinical trails should use technology like genetic screening to create smaller, more-targeted clinical trials that ensure the “right treatments” are developed for “the right group of patients.” They say the legislation will also streamline the paperwork process for clinical trials to make it easier to recruit patients and adapt the operation of trials as new information becomes available.
“[T]he old approach of automatically applying a therapy to a broad group of patients is no longer the best path forward, particularly for those diseases for which we do not have a treatment or cure,” Upton and DeGette write.
Integrating the patient perspective into the regulatory process. The lawmakers say they want to build off the FDA’s Patient-Focused Drug Development Program to incorporate patients perspectives into the regulatory process by using public-private partnerships to help “build the science” around biomarkers and patient-reported outcomes.
“We also will create a predictable process for the qualification of biomarkers and patient-reported outcomes so we can utilize these tools in the development process,” they write.
Promoting better access to and sharing of data. Upton and DeGette say the bill will promote better access and sharing of genomic and other clinical data among researchers and foster interoperability. The bill will also “assist developers and entrepreneurs” develop patient-focused technologies by reducing regulatory uncertainty as they design new apps and products.
“With the explosion of technology, we can collect, monitor and analyze information like never before, helping patients stay healthy and adopt healthy habits while also making treatments safer and more effective,” they write.
Increasing and targeting funding for scientific research. The pair says the legislation help young scientists have more access to resources to fund their research and encourage them to conduct their research in the U.S.
“Many of our most promising investigators — many with great ideas and cutting edge training — are hamstrung by the challenges of securing much-needed support,” they write.
Incentivizing new drugs and devices for unmet medical needs. Upton and DeGette say the legislation will streamline the premarket process and create mechanisms to “better capture” evidence post-market in the development of drugs and devices designed to target unmet medical needs. They say the bill will also “examine incentives” like exclusivity and simplifying the reimbursement process.
“America has a strong history of finding solutions to diseases through medical innovation. Today, partnerships with patients and advocates, health care workers and researchers, and government and the private sector can speed the cycle of finding faster cures,” Upton and DeGette write.
Upton has said he expects to have final language for the bill in March, and hopes to have a vote on the legislation by Memorial Day with the goal of having the measure on the president’s desk by the end of the year.
Stakeholders at a recent IIR-sponsored FDA/CMS Summit said they view work on the 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, and Janet Woodcock, director of FDA’s Center for Drug Evaluation and Research (CDER), said the 21st Century Cures initiative is a “front burner” priority for the agency. — Todd Allen Wilson(firstname.lastname@example.org)