• Skip to primary navigation
  • Skip to main content
  • Skip to primary sidebar
  • Skip to footer
  • Email
  • Facebook
  • LinkedIn
  • Twitter
  • YouTube

International Alliance of ALS/MND Associations

  • Members' Login
  • Contact
  • Join the Alliance
  • Donate
  • What is ALS/MND
  • Find a Member Association
  • Support for PALS & CALS
    • Fundamental Rights for People with ALS/MND and Caregivers
    • Research
      • Voice Preservation
      • Open Science
      • Expanded Access
      • Understanding ALS/MND Research
      • Improving Regulatory Pathways
      • Right to Try
      • US FDA Orphan Drug Designation
      • Unproven (Off-Label) Treatments
      • Open Label Extension
    • Advocacy
      • Advocacy Toolkit
      • Emergency Preparedness Toolkit
      • Equitable Access to Therapies
      • Recommendations for Trial Sponsors
    • Clinical Care
      • Genetic Counselling & Testing
      • Mental Health Support
      • Nursing and Symptom Management
      • Nutrition and Swallowing
      • Occupational Therapy and Activities of Daily Living
      • Physiotherapy and Mobility
      • Respiratory Care
      • Speech Therapy and Communication
      • Support for Family & Caregivers
      • Technology
      • Global Clinic Locator
    • Drugs in Development
      • AB Science – Masitinib
      • BrainStorm Cell Therapeutics – NurOwn
      • Clene Nanomedicine – CNM-Au8
      • Collaborative Medicinal Development – CuATSM
      • ILB – Tikomed
      • Kadimastem – AstroRx
      • Mitsubishi Tanabe Pharma America – Oral Edaravone
      • Neuronata-R/Lenzumestrocel
      • NeuroSense – PrimeC
      • Neuvivo – NP001
      • Prilenia Therapeutics – Pridopidine
      • T Regulatory Cell Therapies
      • SOD1 Therapies & Trials
    • Approved Drugs
      • Nuedexta
      • Radicava/Edaravone
      • Riluzole/Tiglutik
      • Rozebalamin/Methylcobalamin
      • Tofersen/Qalsody
    • Drugs No Longer in Development
      • Amylyx – AMX0035
      • Collaborative Medicinal Development – CuATSM
      • Cytokinetics – Reldesemtiv
      • Orphazyme – Arimoclomol
      • TUDCA Trial
  • Support for Health Professionals
    • Breaking the News in ALS/MND
    • Diagnostic Delay (in development)
  • Events/Programs
    • Calendar of Events/Programs
    • Alliance Meeting
    • Allied Professionals Forum
    • Alliance Webinars
    • ALS/MND Connect
    • Global Day
    • March of Faces
    • Patient Fellows Program
    • Global CRLI
    • International Symposium
  • About
    • Who We Are
    • ALS/MND Health Literacy Map
    • Board of Trustees
    • Advisory Councils/Committees
      • Scientific Advisory Council
      • PALS and CALS Advisory Council
      • Innovation and Technology Council
      • Advocacy and Public Policy Forum
      • Research Directors Forum
      • Governance Committee
      • Finance Committee
    • Staff
    • History
    • Archives
      • Newsletters
      • Meetings
    • Awards
      • Forbes Norris Award
      • Humanitarian Award
      • Allied Health Professional Award
      • Student Innovation Award
  • Members
    • Member Registration
    • Forgot Password

Expanded Access

Every medication or medical device used today underwent extensive testing to ensure its safety and efficacy. The United States set the standard for clinical trials more than 60 years ago, and the process has not changed much since, except to become more complex and lengthy. It can take a decade or more for a drug candidate to reach the clinic, which for many people is too long to wait.

Fortunately, over the past decade, the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have taken steps to expedite certain aspects of clinical trials to more quickly deliver important drugs to people in need. In the US, the expanded access pathway (also called “passionate use”) gives people with life-threatening illness early access to drugs that have not finished clinical testing and, in turn, have not yet been approved by the FDA. The EMA supports expanded access and the programs are operated independently by each member of the European Union.

The Conditions for Compassionate Use

Since compassionate use programs involve giving people drugs that have are not proven safe and effective, the FDA always prefers that people participate in a clinical trial first. The FDA and other regulatory agencies take careful steps to make sure they are not putting patients under undue risk. The FDA only qualifies drugs for expanded access available if:

  • The people who would take it have a immediately life-threatening illness and there are no comparable or effective treatments already available to them
  • These people cannot enroll in a clinical trial, whether it is because no trial is available or the are not eligible for any trials
  • The potential benefits of taking the drug outweigh the potential risks
  • Giving the drug to people will not interfere with an ongoing clinical trial

Expanded access comes in many forms. In some cases, the program is designed to bridge the gap between the end of a clinical trial and marketing approval for a large group of people. In other cases, the pathway is used for a smaller group of people who need a drug that is not actively being developed for clinical use. Finally, a doctor may get permission to give an individual an investigational drug in an emergency setting.

Expanded Access for People with ALS/MND

ALS/MND’s status as a severe, life-threatening disease with no effective treatments might qualify certain experimental ALS/MND drugs for expanded access programs.

 

The Risks Associated with Expanded Access Programs

While the FDA and EMA fully support expanded access programs, they prefer that people enter clinical trials because clinical research offer people more protection if the treatment is unsafe or ineffective. Also, clinical trials are the best way to prove a medication’s safety and efficacy.

It’s important to remember that drugs made available as part of an expanded access program have not been approved for use in the clinic — researchers have not yet proven that it is safe and effective. These drugs make produce unexpected side effects. It is important to discuss the risks with your doctor.

Learn more about expanded access from the FDA here.

Primary Sidebar

Advocacy

  • Advocacy Toolkit

  • Xian-Zhang Niu, Diagnosed 2006 , Shaanxi ALS Association, China

    Xian-Zhang Niu, Diagnosed 2006 , Shaanxi ALS Association, China

  • Soledad Rodriguez, FUNDELA, Diagnosed 2013, Spain

    Soledad Rodriguez, FUNDELA, Diagnosed 2013, Spain

  • Marco Antonio Alvarez Mercado, Mexico

    Marco Antonio Alvarez Mercado, Mexico

  • Roy

    Roy
    roy

  • Richard Clark, MND New Zealand,  Diagnosed 2011

    Richard Clark, MND New Zealand, Diagnosed 2011

  • Sébastien Batiot, Diagnosed 2012 , ARSLA, France

    Sébastien Batiot, Diagnosed 2012 , ARSLA, France

  • Fabio Carvalho

    Fabio Carvalho

  • Christian Bär, Germany

    Christian Bär, Germany

  • Joyce Rusinak, Forbes Norris ALS Center, USA

    Joyce Rusinak, Forbes Norris ALS Center, USA

  • Joy Blakeley, Diagnosed 2017 , MND Australia

    Joy Blakeley, Diagnosed 2017 , MND Australia

  • Maurice Leclerc, Canada

    Maurice Leclerc, Canada

  • Nicholas (Nic) Bowman, MND Association of South Africa,  Diagnosed 2016,  Australia

    Nicholas (Nic) Bowman, MND Association of South Africa, Diagnosed 2016, Australia

  • 727747090571358167

    727747090571358167

  • Aida Trzmiel de Guterman, Asociacion ELA Argentina, Diagnosed 2007, Argentina

    Aida Trzmiel de Guterman, Asociacion ELA Argentina, Diagnosed 2007, Argentina

  • Amparo Muriel Engativa, Colombia

    Amparo Muriel Engativa, Colombia

  • Chen Yin Xue, Taiwan MND Association, Diagnosed 1995, Taiwan

    Chen Yin Xue, Taiwan MND Association, Diagnosed 1995, Taiwan

  • JP

    JP

  • Mike Rannie,  ALS Canada,  Diagnosed 2017

    Mike Rannie, ALS Canada, Diagnosed 2017

  • Jean

    Jean
    jean

  • Lucy Lintott, Diagnosed 2013 , MND Scotland, UK

    Lucy Lintott, Diagnosed 2013 , MND Scotland, UK

  • Alan Liz Ogg 29042016 000799 lo res

    Alan Liz Ogg 29042016 000799 lo res

  • Wiebke Braach, Deutsche Gesellschaft für Muskelkranke, Germany

    Wiebke Braach, Deutsche Gesellschaft für Muskelkranke, Germany

  • Catherine Pearce, Australia

    Catherine Pearce, Australia

  • Mark Miller

    Mark Miller

  • Mauril Belanger

    Mauril Belanger

  • Conny van der Meijden, Diagnosed 2001,  ALS Netherlands

    Conny van der Meijden, Diagnosed 2001, ALS Netherlands

  • Feng Gin Sun, Diagnosed 2014 , Shaanxi ALS Association, China

    Feng Gin Sun, Diagnosed 2014 , Shaanxi ALS Association, China

  • Fabio Correia

    Fabio Correia

  • Marcelo Farinelli, Diagnosed 2006, ABrELA, Brazil

    Marcelo Farinelli, Diagnosed 2006, ABrELA, Brazil

  • Chen Chun-Chin

    Chen Chun-Chin

  • Irene McCaughey, Diagnosed 2011,  MND Australia

    Irene McCaughey, Diagnosed 2011, MND Australia

  • Mary Thomas, Diagnosed 2013 , MND Australia

    Mary Thomas, Diagnosed 2013 , MND Australia

  • Cath Muir

    Cath Muir
    Cath

  • John Dinon, MND Australia

    John Dinon, MND Australia

  • Carlos Gomez Matallanas, Diagnosed 2014 , FUNDELA, Spain

    Carlos Gomez Matallanas, Diagnosed 2014 , FUNDELA, Spain

  • Maurice LeClerc, ALS Canada

    Maurice LeClerc, ALS Canada

  • Duncan Bayly , MND Australia

    Duncan Bayly , MND Australia

  • Ann Nicol

    Ann Nicol

  • Karl Hughes, Diagnosed 2010 , IMNDA,  Ireland

    Karl Hughes, Diagnosed 2010 , IMNDA, Ireland

  • Oliver Juenke, DGM, Germany

    Oliver Juenke, DGM, Germany

  • Frank "Papa" Taylor, USA

    Frank “Papa” Taylor, USA

  • Hanne Stenmose, Muskelsvindfonden, Denmark

    Hanne Stenmose, Muskelsvindfonden, Denmark

  • Den Haag, Diagnosed 2016 , The Netherlands

    Den Haag, Diagnosed 2016 , The Netherlands

  • Graham Johnson, MND Australia

    Graham Johnson, MND Australia

  • John and Loretta Russo, USA

    John and Loretta Russo, USA
    final3878

  • Rolf Mauch, Association ALS Switzerland, Diagnosed 2015

    Rolf Mauch, Association ALS Switzerland, Diagnosed 2015

  • Chih Ching Darren Wong, MND Malaysia

    Chih Ching Darren Wong, MND Malaysia

  • Leon Ryba, Argentina

    Leon Ryba, Argentina

  • David Solomon, Diagnosed 2015, MND Association of England, Wales and N Ireland

    David Solomon, Diagnosed 2015, MND Association of England, Wales and N Ireland

  • Ana Lilia RodriguezApoyo Integral Gila A.C., Diagnosed 2018, Mexico

    Ana Lilia RodriguezApoyo Integral Gila A.C., Diagnosed 2018, Mexico

Learn more about the March of Faces

Latest Tweets

  • Just now

Footer

Subscribe to our Bi-Monthly Newsletter

Sign up to receive updates and to hear what's going on in the International Alliance of ALS/MND Associations.

"*" indicates required fields

 
This field is for validation purposes and should be left unchanged.
  • Email
  • Facebook
  • LinkedIn
  • Twitter
  • YouTube
Return to top of page

Contact | Disclaimer | Privacy Notice & Cookies | Sitemap

Copyright © 2025 The International Alliance of ALS/MND Associations. All rights reserved.


Registered in England: Charity Number 1079504 · Site built by graphics.coop · Powered by WordPress · Members' login