Vertex Pharmaceuticals' cystic fibrosis drug Kalydeco is a success story that is difficult to replicate for Alzheimer's disease, diabetes and other complex conditions, due in part to misplaced incentives and an inadequate drug development system, writes David H. Freedman, co-founder of Global HealthCare Insights. The scene is changing, though, as the government, academia and the drug industry refocus on basic research and collaboration, Freedman writes. And if a drug as successful as Kalydeco comes along to treat a more common condition, if similarly priced, there wouldn't be "enough money in the health care bank to give drugs at those prices to everyone who will need them," said Bernard Munos, a senior fellow at FasterCures.
Signed into law on Dec. 13, 2016, the 21st Century Cures Act is designed to speed up and improve the medical research system, enabling faster and more efficient discovery and development of medical products for patients who need them. This landmark, bipartisan legislation touches virtually all aspects of biomedical research and development, from medical product discovery through regulatory approval. FasterCures worked closely with Congress, federal agencies, and the advocacy community throughout the legislation's journey to the President's desk. We continue to assess its effect on the biomedical innovation system, and -- as recent House and Senate hearings have confirmed -- we are pleased to see progress on implementing Cures Act provisions. Get insights from tracking the implementation of this legislation one year later.
Are you interested in learning about the landscape of patient foundations? The new Investigator Portal is officially live on the Patients Count Network. Investigators will be able to use the network to develop stronger relationships and create opportunities for engagement with patient organizations. Explore and analyze organization profiles to learn about their research initiatives, disease focus, patient population, and more. Join today!
Janssen Pharmaceuticals and the partnership of Sanofi and GlaxoSmithKline are at work developing an HIV vaccine despite many other companies failing to create an effective vaccine. Janssen and Sanofi/GSK are testing their vaccine candidates in trials being conducted in southern Africa, while Aphios is developing a treatment for HIV that uses its critical fluid nanosome technology to deliver small interfering RNA.
A genetic mutation has been found in six members of the same Italian family that makes them feel very little, if any, physical pain, a finding that could one day lead to new pain relief medications, according to findings published in Brain. Researchers found the same mutation in the ZFHX2 gene in a 78-year-old matriarch, her two daughters and three grandchildren.
An enzyme dubbed MAD7 that could be used in CRISPR gene editing will be available to scientists free of charge from startup Inscripta. Users will be charged a small royalty for selling the enzyme without changing it, including it in medical therapies or using it for manufacturing, says CEO Kevin Ness, and the company will make money selling instruments and reagents.
The Miami Dolphins football team has joined with the V Foundation for Cancer Research to raise funds. The team, which hosts the annual Dolphins Cancer Challenge to benefit the Sylvester Comprehensive Cancer Center at the University of Miami Health System, will give $100,000 raised through the challenge, which will be matched by the V Foundation for a grant for an early-career cancer researcher at Sylvester.
The scientific and academic communities tried to educate their senators and representatives about the effects that proposals in tax legislation would have on students, research and drug development. Some proposals could make it harder for people to pursue advanced degrees, chill promising research and increase the likelihood of cuts in federal funding for science, the advocates said.
Patient-reported outcomes data was included this year in more than 75% of approved, pivotal original and new study investigational device exemptions submitted to FDA's Office of Device Evaluation, according to a report from the agency's Center for Devices and Radiological Health. Far fewer post-approval studies included PRO data, according to the report, which also includes examples of how PRO data can be used to measure patient priorities or as safety endpoints.
The 21st Century Cures Act included substantial changes to the Substance Abuse and Mental Health Services Administration to improve coordination among federal agencies and address funding concerns. Assistant Secretary for Mental Health and Substance Abuse Elinore McCance-Katz gave the Senate Health, Education, Labor, and Pensions Committee an update on progress since the law was enacted and answered questions regarding how funds are being spent.
Despite numerous initiatives to open access to publicly funded scientific data, academic researchers and their institutions still hoard data, driven by a system that rewards such secrecy, writes Authorea CEO Josh Nicholson. Incentives in the computer science industry that lead to data sharing could be applied to the biomedical science field, Nicholson writes.
Watch this video to explore the challenges and opportunities that exist in the data sharing space.
FasterCures is an action tank that works across sectors and diseases to improve the effectiveness and efficiency of the medical research enterprise. FasterCures, a center of the Milken Institute, is nonpartisan and independent of interest groups.
1101 New York Avenue, NW
Washington, DC 20005
(202) 336-8900 |