Planning and Expanded Access Program
As an investigational drug progresses through Phase 2 and Phase 3 clinical development, interest from physicians and patients often grows well ahead of regulatory approval. Planning an Expanded Access Program early — not reactively — is one of the most important steps a sponsor can take to manage this demand effectively and ethically.
Develop an EA Policy Early
Before individual requests begin arriving, sponsors should have a written EA policy that defines eligibility criteria, the process for requesting access, and how decisions will be made. Making this policy publicly available — through your company website or a platform such as Reagan Udall's navigator — builds trust with the clinical community and reduces ad hoc requests that are difficult to manage.
Key Planning Considerations
Drug Supply
Do you have sufficient investigational drug supply to support an EA program alongside ongoing clinical trials? Supply constraints are one of the most common reasons EA requests must be declined, and should be assessed before any program is launched.
Regulatory Requirements by Country
Each country has its own EA framework, submission requirements, timelines, and reporting obligations. A program that operates across multiple jurisdictions requires careful coordination and country-specific submissions.
Clinical Trial Integrity
EA programs must be designed so they do not interfere with ongoing trials. Patient eligibility criteria, dosing regimens, and data collection should be clearly differentiated from those used in clinical investigations.
Workflow and Operational Infrastructure
Processing EA requests — from initial physician enquiry through regulatory submission, drug supply, and patient monitoring — requires a defined, documented workflow. Without this, programs become reactive and difficult to scale.
Start-Up and Close-Out Planning
Both the launch and the wind-down of an EA program require deliberate planning. Close-out in particular is often overlooked: what happens to patients currently receiving treatment when a program ends? This needs to be addressed in the program design from the outset.
Real-World Data Collection
EA programs generate valuable real-world data on safety, tolerability, and preliminary efficacy across patient populations that may not be represented in clinical trials. Establishing a systematic approach to data collection from the start maximises the scientific and regulatory value of the program.
When to Seek External Support
EA programs sit at the intersection of regulatory affairs, clinical operations, ethics, and patient advocacy. For many sponsors — particularly smaller biotechs — managing this complexity alongside an active clinical development program is challenging. Langlands & Associates brings deep hands-on experience in designing, launching, and managing EA programs across multiple therapeutic areas and jurisdictions.