Patients and their families, physicians, drug companies, and regulatory agencies have common goals: to find effective therapies for life-threatening conditions. In oncology, the lines between clinical research and treatment are often blurred. more→
COMPASSIONATE USE (EXPANDED ACCESS) NEWS
(Blog) FDA’s Project Facilitate is a Step in the Right Direction
Six is Lydia’s age in years. Six is also a number of months she lived after being diagnosed with DIPG. FDA’s Project Facilitate should help kids who, like Lydia, are denied compassionate use drugs. more→
(Blog) Make compassionate use program easy to navigate
The study we co-authored identified knowledge gaps and misperceptions about the compassionate use application process among pediatric oncologists and echoes new FDA’s Expanded Access Program Report. more→
(Pediatric Blood & Cancer) Physician perspectives on compassionate use in pediatric oncology
Full text Scott Moerdler, Lindy Zhang, Elena Gerasimov, Chong Zhu, Tamar Wolinsky, Michael Roth, Nancy Goodman, Daniel A. Weiser First published: 08 November 2018 Pediatr Blood Cancer. 2018 Nov more→
(Blog) Expanded Access Summit 2.0: It’s time for solutions
Expanded Access Summit 2.0 will address some of the unanswered questions on how to make expanded access feasible. more→
(Blog) 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→
(KVC publication) 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→
(Nature Magazine) 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→
(AACR abstract) 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→
(KVC publication) Saved Josh: The gears of a successful patient advocacy campaign, 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→
Viral crossroads (BioCentury)
By Steve Usdin pdf