An Expanded Access Program (EAP) allows physicians and patients access to pre-approval, investigational drugs outside of the clinical trial setting. An EAP can also be called a Managed Access Program (MAP), Early Access Program, or Compassionate Use Program (CUP).

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
  • If there are approved treatments available, the patient has tried them, and they have been ineffective
  • It is not possible to enroll in an active clinical trial

Fundamentally, there must be an unmet clinical need. The provision of a drug through expanded access can only be granted by the drug manufacturer (sponsor) – no regulatory body or third-party provider can facilitate access without sponsor approval. Please refer to either our Patient and Physicians or Sponsors section below for further information related to your EAP needs.

Patients & Physicians

When patients with severe illnesses cannot access an investigational product through a clinical trial and have exhausted all available approved treatment options, EAPs may offer a means to obtain promising pre-approval drugs. Patients who wish to access an investigational drug through an EAP must have a physician willing to act on their behalf. Physician responsibilities include:

  • Making the clinical decision and justification to use an investigational drug
  • Contacting the drug manufacturer or WEP Clinical and requesting access to the drug
  • Completing the necessary documentation for the relevant regulatory bodies (e.g. the FDA, MHRA, etc.) to provide approvals to import/administer the investigational drug

  • Contacting the relevant local ethics committee (or institutional review boards in the US) to gain approval to use the product to treat the patient

  • Ethical handling of patient information and consent

Patients and physicians cannot receive drug through expanded access without drug manufacturer approval. For more information on the companies currently providing expanded access use of their products, check out following websites:

clinicaltrials.gov
The Reagan-Udall Expanded Access Navigator
EU Clinical Trials Register

Patients and physicians can also visit a company website and look for their expanded access policy. All companies with products in phase 2 or phase 3 clinical trial in the US are required to have an expanded access policy on their website.

WEP Clinical provides patient advocacy outreach to engage patient organizations and help bring needed treatment to patients around the world.  To learn more about our experience working alongside patient advocacy groups, please visit our patient advocacy page.

If you require further information or would like help researching treatment options or reaching out to drug manufacturers, contact us at:

SPONSORS

EAPs are regulatory approved programs that allow sponsors to make their pre-approval products available to patients outside the clinical trial. EAPs offer a variety of benefits to companies, including the opportunity to:

  • Further assess a drug prior to the full launch

  • Generate additional safety and real-world data that helps in understanding how the drug will be used in clinical practice

  • Manage 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

  • Provide physicians with experience using the new medicine, creating advocates for the drug prior to its launch

  • Build relationships with both doctors and patients which facilitates brand loyalty

Under the 21st Century Cures Act, all companies with investigational drugs in phase 2 or phase 3 clinical trial must now have an expanded access policy 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.

Our end to end EAP Delivery includes the following services:

  • Dedicated Project Management
  • Protocol Writing

  • Physician and Patient Request Management
  • Physician Training and Support
  • Medical Oversight

  • SAE Reporting

  • Regulatory Approval Management

  • Ethics and IRB Approval Management

  • Patient Advocacy Engagement

  • Real World Data Collection

  • EAP Site Monitoring

  • Product Warehousing & Distribution

  • Transition Plan to Approved Drug under an NPP

If you are a sponsor with a product in development and you require the deployment of an EAP, or if you would like to discuss distribution of your drugs to manage requests from patients/physicians, please contact us at:

FAQ’S

Both EAPs and NPPs 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 2 or 3 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 Clinical, 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.

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.”

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