Expanded access (compassionate use)

United States, federal regulation, with counterparts elsewhere. Expanded access is set out in Title 21 of the Code of Federal Regulations, part 312, subpart I, first formalised as the treatment IND rule of 1987 during the AIDS crisis and rewritten in 2009 into three categories: individual patients (including emergencies), intermediate-size populations, and widespread treatment use. Primary text: the FDA's expanded access page links the regulations and the single-patient form (Form FDA 3926, introduced 2016). The 21st Century Cures Act of 2016 required companies to publish their expanded access policies, and the FDA Oncology Center of Excellence's Project Facilitate (2019) is a call centre that helps oncologists file requests.

What it means for patients: expanded access is how a patient with a cancer that has progressed on every approved therapy may receive a drug still in trials, provided the company will supply it, the treating physician takes responsibility and an ethics board agrees. The FDA authorises the large majority of requests, and emergency requests are handled within a day. The European Union has a compassionate use provision in Article 83 of Regulation 726/2004, implemented country by country (France's early access system, the UK's Early Access to Medicines Scheme, named-patient supply in many countries).

The arguments: companies fear that an adverse event in an uncontrolled setting will harm the development programme, though the FDA rarely holds such data against a drug; ethicists argue over fairness when supply is limited; and patient advocates argued that the whole process was too slow, which produced the Right to Try Act of 2018. Expanded access remains the main route in practice.