Fast Company praised Kids v Cancer for accelerating the discovery of pediatric cancer treatments. more→
COMPASSIONATE USE (EXPANDED ACCESS) NEWS
Right to Try does not make compassionate use access easier
The new law doesn’t change a fundamental reality about compassionate use: the biggest hurdle is persuading a drug company to agree to provide access. more→
Our abstract at 2018 ASCO annual meeting: Pediatric oncologists’ experiences with compassionate use
The lack of knowledge and paucity of support in the application process are immediate actionable barriers to wider utilization of compassionate use in pediatric oncology. more→
How to try an experimental drug when you don’t qualify for a clinical trial (Forbes)
How to handle compassionate use requests is not taught in medical school. In essence, the process has four steps. more→
To speed access to compassionate use, look beyond the FDA (Health Affairs Blog)
Contrary to widespread industry concerns that serious adverse events in a compassionate use program will lead to problems with the FDA, analysis of years of data does not show this to be the case. more→
Senate passes ‘right to try’ bill to help terminally ill patients get experimental drugs (Washington Post)
The Senate on August 3 passed a measure designed to make it easier for terminally ill patients to get access to experimental treatments without oversight from the FDA. more→
Senate passes F.D.A. funding and ‘Right to Try’ drug bills (New York Times)
The FDA bill includes new requirements that could increase the number of cancer drugs for children. more→
Nature supports RACE for Children Act
Kids v Cancer welcomes NATURE’s editorial in support of the RACE for Children Act to champion the development of promising cancer drugs for children. more→
Fairer way to distribute last-ditch drugs gets real-world trial (Nature Magazine)
Drug companies’ compassionate policies hard to decipher, and experts are testing what they hope is a fairer system to distribute drugs. more→
FDA simplifies compassionate use application and drug costs (Medscape)
As promised, the FDA has simplified the form physicians use to request investigational compassionate use drugs when all other options have been exhausted. more→
Compassionate Use Navigator addresses challenges and facilitates access to investigational drugs for pediatric oncologists (Proceedings: AACR 107th Annual Meeting 2016)
Proceedings: AACR 107th Annual Meeting 2016; April 16-20, 2016; New Orleans, LA Elena Gerasimov, Lindy Zhang, Scott Moerdler, Michael Roth, Nancy Goodman more→
Saved Josh: The gears of a successful patient advocacy campaign (Case study, by Elena Gerasimov)
Read a detailed account of a case of a 7-year-old boy whose request to be treated with an experimental drug started the nationwide conversation about compassionate use. more→
Phoenix teen brings story to panel weighing access to life-saving drugs (Cronkite News)
A report from the Senate committee considering whether potentially life-saving drugs should be made available to U.S. children with terminal diseases. more→
The CEO who saved a life and lost his job (Bloomberg Business)
BIO is working with its member companies, particularly small ones, to prepare for campaigns like the one the Hardy family waged against Chimerix. more→
Practical, legal, and ethical issues in expanded access to investigational drugs (New England Journal of Medicine)
An outline of potential problems with expanded access. more→