Expanding Patient Access to Investigational New Drugs: Overview of Intermediate and Widespread Treatment Investigational New Drugs, and Emergency Authorization in Public Health Emergencies

Van Norman, Gail A.

Releasing investigational new drugs to individual patients who are facing certain death or disability seems to be a relatively uncomplicated decision, but allowing EA to entire groups of patients for treatment with an investigational new drug presents more complex regulatory, logistical, and ethical challenges for scientists, commercial entities, and the FDA. The current regulatory process from IND filing to drug approval has evolved and includes not only the FDA's historical primary mission of ensuring patient safety, but also, since the latter half of the 20th century, the newer mission of ensuring that marketed drugs are actually efficacious for their advertised/approved use. EA for a single patient may not present much of a challenge to the assertion that a drug's benefits outweigh the risks, because as presumably the patient requesting compassionate use faces an otherwise dismal clinical future, taking even significant risks with a new drug still presents potential benefits to a patient without other options.