Expanded Access Programs
An Expanded Access Program (EAP) allows physicians and patients access to pre-approval, investigational drugs outside of the clinical trial setting.
EAPs allow sponsors to provide access to pre-approval products outside of clinical trials. These programs are unique in each country and require different levels of regulatory approval.
These programs are for patients suffering from a serious or life-threatening illness who have no viable treatment options available to them. This means that:
- There are no approved treatments available in the patient’s home country
- The patient has exhausted all approved treatment options
- It is not possible to enroll in an active clinical trial, or clinical trials have ended and patients are waiting for commercial supply
How Sponsors Benefit
- Do the right thing and provide treatment to patients with unmet needs
- Expose physicians to drug prior to commercial approval
- Give regulators an early look at a drug which may assist in commercial approval
- Collect Real World Data (RWD) which can generate Real World Evidence (RWE) — This evidence may support clinical trial data
- Gain knowledge and experience from EAP to design more effective clinical trials
Under The 21st Century Cures Act, all companies with drugs in clinical trials that meet the criteria for expanded access must now have an Expanded Access Policy posted on their website.
If you have a product in, or reaching, late stage clinical development for cancer or rare diseases, now might be a good time for you to start thinking about expanded access and your company’s ability to handle potential patient requests.
21st Century Cures ActAn Expanded Access Program may also be called
Compassionate Use Program (CUP)
Early Access Program
Managed Access Program (MAP)
Post-Trial Access Program
Check our blog to learn more
Our EAP Services
- Dedicated Project Management
- Protocol Writing and Review
- Physician and Patient Request Management
- Physician Training and Support
- Global Regulatory Management and Support
- IRB and Ethics Approval Management
- Ongoing Physician Training and Support
- Product Labeling, Warehousing, and Distribution
- Data Management / Real World Data Collection
- Data Monitoring
- Full-Service Pharmacovigilance
- Medical Monitoring
- Patient Advocacy Engagement
- Market Access Strategy and Support
- Transition of EAP Patients to Commercial Product or Paid-For Named Patient Program (PA-NPP)
Our EAP Case Studies
Sponsor FAQs
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.
HELPING A SPONSOR TRANSITION AN EXISTING EAP OVER FROM ANOTHER VENDOR
Condition: Debilitating neurological disorder Clinical Trial Phase: Completed Phase 3