• SKIP TO CONTENT
  • SKIP NAVIGATION
  • Patient Resources
    • COVID-19 Patient Resource Center
    • Clinical Trials
    • Search Clinical Trials
    • Patient Notification System
    • What is Clinical Research?
    • Volunteering for a Clinical Trial
    • Understanding Informed Consent
    • Useful Resources
    • FDA Approved Drugs
  • Professional Resources
    • Research Center Profiles
    • Clinical Trial Listings
    • Market Research
    • FDA Approved Drugs
    • Training Guides
    • Books
    • eLearning
    • Events
    • Newsletters
    • JobWatch
    • White Papers
    • SOPs
    • eCFR and Guidances
  • White Papers
  • Trial Listings
  • Advertise
  • COVID-19
  • iConnect
  • Sign In
  • Create Account
  • Sign Out
  • My Account
Home » Compassionate use and the impact on clinical research

Compassionate use and the impact on clinical research

April 11, 2016
CenterWatch Staff

Requests for compassionate use of drugs not yet approved by the FDA have been making headlines, and experts predict this trend will continue. Last year, the FDA received 1,262 expanded access requests from drug companies, on behalf of patients, to use unapproved drugs outside of clinical trial programs.

Since 2010, the number of compassionate use requests has risen by nearly 25%. Many of these requests are for rare diseases with small patient populations, with highly engaged patients and caregivers connected to advocacy groups. With the increase in targeted therapies, the question is not whether research sponsors will have to address a request, but when and how prepared they are to respond.

The FDA has clear guidelines on requirements for meeting expanded access, but the onus is on companies to determine which cases to support and which to deny. Many companies are unprepared to respond quickly, having no formal process to follow. This approach has left the corporate reputations of several companies vulnerable.

cww2014_20160411_outkUs-6

For example, several years ago BioMarin was petitioned for access to its experimental phase III ovarian cancer drug. Like many pharma companies, BioMarin refused access. Andi Sloan, the petitioner, began a social media campaign in attempt to prolong her life with treatment. Word spread quickly through Facebook and Twitter, generating major media coverage critical of BioMarin, celebrity support from Wynonna Judd and Newt Gingrich and a Change.org petition signed by 150,000 people. A rival firm anonymously donated its own ovarian drug candidate to Sloan, but her condition worsened and she died.

As healthcare has become more consumer-driven, policy makers have stepped into the fray, proposing legislation aimed at cutting through government red tape that prevents access to investigational drugs in a timely manner. These “Right to Try” bills have made their way to legislatures in half the states in the U.S.

Some argue that these bills are empty legislation designed to raise the hope that patients have new rights. In fact, patients already have a path to request access to these drugs under current FDA rules, and over 98% of applications to the FDA to use experimental drugs are approved. These new laws contain no provision that compels companies developing experimental drugs to approve a patient’s request.

The public at large may not understand the impact of compassionate use on clinical research, but it has the potential to compromise the clinical trial process. The essence of right-to-try laws is to eliminate the role of the FDA from the process, which flies in the face of public safety.

Furthermore, the easier it is for patients to get access to experimental therapies, the less inclined they may be to enroll in a clinical trial. This is particularly troubling for rare diseases with small patient populations, where enrolling enough patients to generate significant comparative data is already a challenge.

Granting compassionate use of an investigational compound is a question of both good ethics and good business. Currently, the FDA offers no guarantee that an adverse outcome related to a compassionate use case does not threaten an ongoing clinical trial. At what cost is an individual’s access to therapy worth the ability to bring that therapy to hundreds or thousands of patients in a safe way?

Regardless of how the right-to-try movement plays out, this is an area that clinical research sponsors need to pay attention to. Experts forecast the global orphan drugs market to grow 10.05% annually between now and 2019. Companies like Shire, Baxalta and PTC Therapeutics, who have growth strategies focused on rare diseases, should anticipate an increase in compassionate use requests, and have appropriate decision systems in place. Instead of ad hoc evaluations of requests, they may want to test pilot programs that equitably and transparently distribute an experimental drug outside of its clinical trial program. 

 

Matthew Howes is executive vice president, Strategy & Growth, for PALIO, an inVentiv Health company. Email matthew.howes@inventivhealth.com.

This article was reprinted from CWWeekly, a leading clinical research industry newsletter providing expanded analysis on breaking news, study leads, trial results and more. Subscribe »

Upcoming Events

  • 24May

    Powering an Effective Oversight Strategy with Clinical and Operational Insights

  • 25May

    2022 WCG Avoca Quality & Innovation Summit: Own the Future

  • 28Jun

    Effective Root Cause Analysis and CAPA Investigations for the Life Sciences

  • 16Oct

    WCG MAGI's Clinical Research Hybrid Conference - 2022 West

Featured Products

  • Spreadsheet Validation: Tools and Techniques to Make Data in Excel Compliant

    Spreadsheet Validation: Tools and Techniques to Make Data in Excel Compliant

  • Surviving an FDA GCP Inspection

    Surviving an FDA GCP Inspection: Resources for Investigators, Sponsors, CROs and IRBs

Featured Stories

  • Protocol-360x240.png

    Avoid Deviations by Making Protocol Review a Team Effort

  • SelectionProcess-360x240.png

    Give Us a Voice: Sites Clamor for a Say on Vendor Selection

  • Convince-360x240.png

    Use Data and Details to Convince Site Leadership to Add Staff

  • AsktheExpertsBadge-360x240.png

    Ask the Experts: Listing Trial Staff and Others on the Statement of Investigator

Standard Operating Procedures for Risk-Based Monitoring of Clinical Trials

The information you need to adapt your monitoring plan to changing times.

Learn More Here
  • About Us
  • Contact Us
  • Privacy Policy
  • Do Not Sell My Personal Information

Footer Logo

300 N. Washington St., Suite 200, Falls Church, VA 22046, USA

Phone 617.948.5100 – Toll free 866.219.3440

Copyright © 2022. All Rights Reserved. Design, CMS, Hosting & Web Development :: ePublishing