Related references
Note: Only part of the references are listed.
Article
Pharmacology & Pharmacy
Tobias B. Polak et al.
Summary: This article discusses the potential value of expanded access (or compassionate use) for patients with rare diseases and its supportive role in decision-making. The authors recommend harmonization of expanded access regulations across European countries to reduce burden on manufacturers, improve patient access, and obtain better data.
FRONTIERS IN PHARMACOLOGY
(2022)
Article
Medicine, General & Internal
Patrick Gould et al.
Summary: This qualitative study examines how oncologists at academic medical centers perceive expanded access (EA) to investigational medical products. The results show that oncologists view EA as an important tool to secure the best treatment options for their patients and are willing to pursue EA as part of their obligation. They also express confidence in evaluating investigational treatments and do not necessarily view EA as a last resort.
Article
Multidisciplinary Sciences
Jeremiah Stout et al.
Summary: The study interviewed 21 oncologists with previous expanded access experience at a major cancer center, revealing the challenges and various factors they consider when deciding whether patients are suitable for non-trial access to investigational drugs. The participants reported difficulties with administrative processes, drug company reluctance, and varying patient rationales for seeking experimental treatments. They also highlighted the importance of evaluating scientific rationale, risk-benefit ratio, patient functional status, and motivation when making decisions about compassionate use drugs.
Article
Humanities, Multidisciplinary
Stefan F. Vermeulen et al.
Summary: This study investigated physician experiences with expanded access to investigational drugs in different healthcare systems and identified factors such as the suitability of investigational treatments, the application process, hospital policies, support by pharmaceutical companies, and funding and reimbursement arrangements that determine the feasibility of expanded access in clinical practice. Despite being legally allowed and technically possible, expanded access is not always considered an option by treating physicians due to lack of familiarity, extensive application process, willingness or ability of pharmaceutical companies to supply the drugs, and funding issues.
HUMANITIES & SOCIAL SCIENCES COMMUNICATIONS
(2021)
