Early Access Programs are for patients who are suffering from a serious or life-threatening disease or condition who have no viable treatment options available to them.
This means patients must have tried any approved medication in their country and must be unable to enroll into any active clinical trial.
Early access cannot be used as a substitute for a clinical trial. Any patient eligible for an active study will not be approved for early access use and will be asked instead to enroll in the trial in order to access the treatment. At this time, clinical trials are still considered the safest option for patients when using investigational drugs, as treatment is provided in a highly regulated and controlled environment.
Early Access Programs are usually only provided for drugs in the late stages of clinical development i.e. phase 2 or 3 clinical trials.
This is because companies will want to ensure they have a good understanding of the safety profile of a drug, before they consider providing early access use to a group of patients outside the clinical trial.
Pharmaceutical companies can also provide an Early Access Program once clinical trials have ended. This provides continued patient access to the treatment, up until the product receives market approval and becomes commercially available. Once it is commercially available, the Early Access Program will be closed out, and patients will be transitioned to commercial supply.
An individual patient cannot apply for an Early Access Program themselves.
Any patient looking to enroll into a program must have a licensed physician willing to act on their behalf. The treating physician will be responsible for:
- Applying for the program
- Informing any local institutional or ethics review board
- Educating the patient on the risks involved in using an investigational drug
- Administering the treatment
- Reporting any serious adverse effects
Companies are not required to provide an Early Access Program for their investigational drugs.
While regulatory bodies, like the US Food and Drug Administration (FDA), European Medicines Agency (EMA) and Australian Therapeutic Goods Administration (TGA), support these programs, they do not currently have any legislation in place to require companies to provide them.
Furthermore, companies with an Early Access Program in place do not have to provide access to every patient. Just like in a clinical trial, an Early Access Program will have enrollment criteria that must be met before a patient can be approved into the program. If a patient does not meet these criteria, the company will have the right to refuse access.
Companies running phase 2 or 3 clinical trials in the US are required to have an early access policy posted on their website.
The policy must clearly state a company’s decision to provide or not provide early access and must explain the reason(s) behind this decision. It must also include clear instructions and up to date contact information for patients to use if they wish to contact the company or apply for a program. Companies must make this policy readily available and easily accessible to patients visiting their website.
WEP Clinical provides template documents that companies can download and use to create their policy for our website:
Expanded Access Policy Templates
WEP Clinical has been working with pharmaceutical companies to provide Early Access Programs on a global basis, for over a decade. Through our portfolio of programs, we currently supply treatment to patients suffering from a variety of rare diseases and cancers.
We also work with a number of patient advocacy groups across the world, using our experience and expertise to help connect patients with the right companies and treatments. We recognize the outstanding work that these groups do and are committed to helping them provide the best standard of care and support for their patients.