Navigating the Landscape of Compassionate Use

Requests for compassionate use of investigational drugs have increased dramatically in recent years.

In a newly released white paper by Siren Interactive, produced from input by rare disease patient advocacy group leaders and patient opinion leaders, including PPMD Founding President & CEO Pat Furlong, Siren examines the issues around compassionate use and the need for proactive approaches by industry to both developing policies and communicating those policies to all interested stakeholders.

New Guidance from FDA

In May 2013, the FDA released “Guidance for Industry about Expanded Access to Investigational Drugs for Treatment Use.” This document contains details on the process for expanded access submissions and explains:

  • What should be contained in the two types of access submissions – protocol amendment to an existing or new IND submission
  • Who can request expanded access – the IND sponsor (pharmaceutical company) or the patient’s physician – and what are the limitations and requirements for each
  • Reasons the FDA may deny expanded access – risk versus benefit
  • Process for emergency access
  • How the FDA determines that expanded access won’t interfere with an ongoing clinical trial


It’s important to note that the FDA cannot compel a company to provide expanded access to its drug. Company participation is voluntary.

The Dilemma for Pharmaceutical Companies Investigating Orphan Drugs

Often companies receive requests for compassionate use, which they must refuse because of concerns that:

  • Clinical supplies may be limited due to high cost of production
  • Adverse events with patient populations outside a clinical trial could delay development and approval
  • The compassionate use option may limit enrollment in clinical trials due to patients’ fear of being put on a placebo
  • Responding to pressure from isolated patients may create an ethical dilemma if it results in a flood of requests that can’t be met


Even when these concerns are realistic, companies can encounter problems if they have not clearly and respectfully communicated their policy in advance.

Recommended Action

Navigating the landscape of compassionate use requires a proactive approach to both developing policies
and communicating those policies to all interested stakeholders. Click here to view the six best-practice recommendations Siren identified.

We hope these guidelines help illuminate the issue, and that industry will benefit from its insights and case studies as they go about designing and implementing an appropriate approach and policy for individual companies.

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