The push for new drug discoveries—especially when it comes to drugs for progressive and life-threatening diseases—is driven in large part by urgent need, often by patients who are in a race against time. But “finding” a cure is only the first step, and it takes more than unmet need to fuel the drug development process. Legislation is necessary that supports and fosters innovation, and that at the same time ensures all stakeholders’ voices are heard.
Recently, a bill intended to accelerate the drug development process, known as the 21st Century Cures Act, was unanimously passed by the US House Energy and Commerce Committee (ECC). Currently the legislation is divided into three parts—Discovery, Development, and Delivery—showcasing the comprehensive and highly ambitious nature of the initiative, which attempts to enhance all phases of medical innovation. Sponsored by the ECC and chaired by Republican Representative Fred Upton, the overarching goal of the bill is, according to the House Committee, “[to close] the gap between the science of cures and the way in which they are regulated.”
Patients First, Last, and Center
Patient-focused goals are the centerpiece of the Development section of the current version of the bill. Giving highest priority to patients’ experiences, the legislation requires that the FDA create a formal structure for incorporating patients’ input into regulatory decision-making. “No one understands a particular condition or disease better than the patients living with it,” legislators explained in a white paper that accompanied the bill. As the ultimate end-user of any therapy, patients are acknowledged by the 21st Century Cures Act to be integral to drug development itself, with the bill requiring the FDA to include “patient experience data” as a crucial component of a “structured risk-benefit assessment.” For persons with a life-threatening or progressively disabling disease, this means self-determination—having a say in what risks they would or would not be willing to accept in order to improve or prolong their lives.
Drug discovery would be potentially heightened by the 21st Century Cures Act through increased funding for the National Institutes of Health, including an NIH Innovation fund for young emerging scientists as well as for precision medicine. To help speed the drug approval process, provisions call for the FDA to provide guidance for expediting clinical trials. Such guidance would be designed to encourage the use of streamlined data review processes, adaptive trial designs, and the inclusion of “real-world” data (that is, clinical data not confined to and collected strictly from randomized clinical trials).
Will These Incentives Have Staying-Power?
Exclusivity incentives, considered by many to be among the more controversial aspects of the legislation, had been conspicuously dropped from the prior version of the draft bill—but some of these incentives have now been restored to the latest markup. A provision called the Orphan Product Extensions Now Act would grant an additional 6 months of market exclusivity to the innovators of pharmaceutical agents, provided that the new drug would be used to treat a rare disease (ie, affecting fewer than 200,000 persons in the United States). Another newly reinstated provision is extension of the Priority Review Voucher for Rare Pediatric Diseases, which had been scheduled to expire in March 2016 but would now be continued indefinitely. Struck from the bill, however, was the noteworthy provision allowing pharmaceutical companies 15 years of market exclusivity—a considerably longer period than the 7 years currently permitted for orphan drugs.
The bill is due to be taken up by the Senate by autumn 2015.
About the Connexion Healthcare Rare Disease Center of Excellence
The Connexion Healthcare Rare Disease Center of Excellence specializes in communicating the science behind rare diseases and orphan drugs. We strategically partner with industry leaders in the rare disease community to proactively connect all stakeholders in launching products to underserved patient populations. Our team comprises skilled communications experts who plan, develop, and disseminate scientific communications based on their advanced knowledge of the science, the patient journey, healthcare providers, and the orphan drug approval process.
At Connexion, our cross-functional teams navigate complicated treatment and market landscapes by elucidating the appropriate pathways to brand success. We understand how brand strategy drives market share. Since launching its dedicated Rare Disease Center of Excellence, Connexion has developed prelaunch, launch, and postlaunch communications in numerous rare diseases and disorders: congenital and genetic, movement, metabolic, and lysosomal storage disorders; lung, blood, skin, and endocrine diseases; and rare cancers.
For further information regarding the Rare Disease Center of Excellence at Connexion Healthcare and how we can develop rare disease communications to differentiate therapies by their unique attributes, contact:
Susan Stein, MPH
Rare Disease Center of Excellence