Expanded Access Programs

An EAP provides access to pre-approval, investigational drugs that are still being studied/tested in clinical trials and are not approved anywhere in the world.

There is no current regulation that requires companies to provide an EAP for their investigational drugs. However, as patient and physician demand for access to investigational drugs outside of clinical trials grows, many companies are now actively building pre-approval access strategies, like EAPs, into their drug development programs.

Although companies are not required to provide EAPs, those companies with drugs in phase 2 or 3 clinical trials in the US are required to have an EAP policy posted on their website.

• Further assessing a drug prior to the full launch
• Generating additional safety and real-world data that helps in understanding how the drug will be used in clinical practice
• Managing risk in the pre-launch setting through appropriate adverse event capture and patient screening to ensure the correct cohorts are selected to take the medicine
• Providing physicians with experience using the new medicine, creating advocates for the drug prior to its launch
• Building relationships with both doctors and patients which facilitates brand loyalty

Yes. Although less stringent than a clinical trial, an EAP will still have inclusion and exclusion criteria, set by the sponsor company.

The sponsor will reserve the right to deny access to patients who do not meet this criteria.

Yes. Sponsors sometimes chose to restrict the number of patients enrolled into an EAP, due to limited resources or product availability. However, companies need to be forthcoming with this information, by including it in their EAP Policy.

No. In order to qualify for participation in an EAP, a patient must not have access to, or qualify for, any active clinical trials.

Companies are required to report certain adverse event data to regulators. To clarify how adverse event data is viewed under expanded access circumstances, the FDA has updated the guidance document for industry. In the document, ‘Expanded Access to Investigational Drugs for Treatment Use: Questions and Answers’ (questions 25 and 26), it is stated that suspected adverse reactions must only be reported if there is “evidence to suggest a causal relationship between the drug and the adverse event.”

One of the benefits of providing an EAP is that companies can collect Real World Data (RWD) from patients enrolled in the program. When compiled and analyzed, this data becomes Real World Evidence (RWE) which can be used to supplement clinical trial data when seeking marketing approval for an investigational drug.

To find out more, check out WEP Clinical’s RWE offering.

Although companies are not required to provide EAPs, those companies with drugs in phase 2 or 3 clinical trials in the US are required to have an EAP policy posted on their website. Sponsors are permitted to include in their policy that they do not have an ongoing EAP or they are still working internally to devise a program and do not offer one at this time.

If a company does allow expanded access use, the policy must outline the process physicians should follow to request access and provide up to date contact details for physicians to use to make a request.

WEP has designed EAP Policy templates for companies to use when developing their policy. These can be accessed here.