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Managing patient requests for Your investigational medicines
Are you receiving unsolicited requests for your phase 2 or phase 3 medicine from patients who cannot enroll in the active clinical trial? Unsure of how to manage these requests moving forward?
Expanded Access Programs (EAPs), also called Compassionate Use Programs (CUPs) or Managed Access Programs (MAPs), allow companies to provide their investigational (pre-approval) medicines to patients that do not qualify for the clinical trial. These programs are set up alongside a phase 2 or phase 3 trial to fulfil unmet clinical needs of patients around the world.
Now you know expanded access is a pathway that allows you to manage the unsolicited patient requests for your medicine, here is what to do next:
1. Decide if you are going to provide expanded access use of your medicine
Companies are not required to provide an EAP. Regulatory bodies do encourage expanded access supply, if and when the benefits to the patient outweigh the risks, and there is no potential for disruption to the active clinical trial. However, the decision on whether to run a program lies solely with the pharmaceutical company.
Visit the Expanded Access Page on our website for more information on the benefits companies have seen from running an EAP.
2. Create an Expanded Access Policy for your website
Once you have made your decision, you must create an Expanded Access Policy for your website. Under The 21st Century Cures Act, all companies with drugs in phase 2 trials in the US that meet the criteria for expanded access must have an Expanded Access Policy posted on their website. This policy must clearly state whether expanded access is being provided by the company. It must also include instructions and up to date contact information for physicians to use if they wish to contact the company or apply for a program on their patient’s behalf.
You can click here to learn more about the 21st Century Cures Act and its requirements.
3. Understand the internal roles and responsibilities
If you choose to provide an EAP, you will need to understand what internal teams to include in the set up and management of the program. Depending on the size of your company, the teams involved could include:
- Clinical Operations
- Medical Affairs
- Market Access
- Commercial Operations
- Supply Chain
- Regulatory
- Quality
- Pharmacovigilance
4. Define the scope of the program
Just like in a traditional clinical trial, an EAP will have defined territories in scope and specific inclusion criteria that patients must meet in order to be enrolled into the program. Companies can specify the exact number of patients and sites they will allow to enroll as well as the end date for the program. Determining these factors at the outset and building them into the project plan will help to control patient access and keep internal teams aligned. It will also help with the development of project documentation, forms, and timelines and will ensure streamlined management and drug supply processes.
5. Understand the regulatory requirements in each country in scope
The regulations governing expanded access differ from country to country. In order to get product to patients in an expedited, efficient, and compliant manner, companies should take time to understand the complex regulatory processes and requirements in each of the countries in scope. This will help your team effectively support sites and physicians when obtaining the appropriate regulatory approvals and the needed importation permits in all countries of interest.
WEP has developed an extensive database that outlines the unique pre-approval, expanded access regulatory processes and importation laws in over 120+ countries. For help with understanding and developing your regulatory plan, reach out to our team at:
6. Decide if and what data to collect
The main purpose of an EAP is always to provide treatment to patients. However, there are additional benefits to running an EAP, including the opportunity to collect Real World Data (RWD) from patients and physicians enrolled in the program. Companies can collect this data to support their clinical trial data or to gain a better understanding of their drug in a wider patient population outside the clinical trial. It is important to understand what data can be collected in an EAP, how much can be collected and what tools can be used to collect it.
WEP has a free resource companies can use to begin strategizing their data collection goals and needs: