The US House of Representatives recently passed a bill to speed the discovery and development of new medical treatments. This legislation, known as the 21st Century Cures Act, authorizes an additional $1.75 billion per year for the next 5 years to fund the National Institutes of Health, and an additional $550 million to the FDA.
A key provision of this legislation would change the current “Breakthrough Therapy Designation” for drugs. This designation allows the FDA to expedite the review and approval of drugs for serious and life-threatening diseases for which early clinical data are available showing that they are better than current treatments. The bill would allow this designation to make the process of bringing drugs to market even faster.
The 21st Century Cures initiative will remove barriers that are inhibiting progress in the development of drugs and not providing added benefit to patients. The policies in the bill will help to make the development of drugs and medical devices less time-consuming and less costly, helping to ensure that the United States remains on the cutting edge of drug and device development.
The Advanced Medical Technology Association commends the passage of the bill because it contains proposals that strengthen innovation and support the development of life-saving, life-enhancing medical technology. The Pharmaceutical Research and Manufacturers of America also strongly supports the legislation.
Critics of the legislation say that it could potentially raise safety concerns for patients. However, the director of the Center for Drug Evaluation and Research at the FDA, Janet Woodcock, MD, has stated that the bill does nothing to lower the FDA’s gold standard of safety.
The overwhelming approval by the House does not mean that the 21st Century Cures Act will necessarily become law. The next stop for the bill is the US Senate.
About the Connexion Healthcare Rare Disease Center of Excellence
Connexion Healthcare is a full-service global provider of strategic medical and scientific communications with decades of experience providing exceptional service to the pharmaceutical industry. We provide services through 2 Centers of Excellence—Oncology and Rare Disease—and possess core expertise in these therapeutic areas and their marketplaces.
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