Frequently Asked Questions
Both EAP and NPP allow access to unapproved medicines. The difference between these two programs is that an EAP provides access to pre-approval, investigational drugs that are still being studied/tested in clinical trials. A NPP provides access to post-approval drugs that are no longer in clinical trial and are commercially available in one or more country.
Because an EAP is not a clinical trial, pharmaceutical companies can charge patients for drugs provided through these programs. However, it is not unusual for companies to provide these drugs at no cost to the patient.
There is currently no 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 the traditional clinical trial setting grows, many companies are now actively building pre-approval access strategies, like EAPs, into their drug development programs.
Patients cannot apply for expanded access themselves. They must have a licensed physician willing to act on their behalf. Physicians must first contact the pharmaceutical company or EAP partner and request access to the drug. Without company consent to use the drug, the expanded access process cannot proceed.
In certain countries, like the US, once the drug company has agreed to provide the drug, the patient’s physician must then apply to the appropriate local regulatory agency (e.g. the FDA) on the patient’s behalf.
Physicians will also have to submit an expanded access request and informed consent document to an Institutional Review Board (IRB) or Ethics Committee.
For patients looking for information on established EAPs being conducted in their therapeutic area, information can be found on websites such as clinicaltrials.gov. If a patient knows of a company developing a drug for their condition, they can contact the company directly. Companies with drugs in phase II or III clinical trials in the US should have an EAP policy posted on their website. This policy should give patients all the information they need to inquire about expanded access use of a drug. Alternatively, please feel free to contact the WEP patient advocacy group, and we can contact the manufacturer on your behalf.
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.
Under current legislation, drugs in late stage development, either in phase II or phase III clinical trials, are considered appropriate for expanded access use. However, for certain conditions, like cancer and rare diseases, companies may even consider providing expanded access use of drugs in phase I 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 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.”