• Skip to primary navigation
  • Skip to main content
  • Skip to primary sidebar
  • Skip to footer
  • Email
  • Facebook
  • Instagram
  • 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 of People with ALS/MND and Caregivers
    • Research
      • 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
    • 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
    • Drugs in Development
      • AB Science – Masitinib
      • Amylyx – AMX0035
      • Biogen – Tofersen
      • BrainStorm Cell Therapeutics – NurOwn
      • Clene Nanomedicine – CNM-Au8
      • Collaborative Medicinal Development – CuATSM
      • Cytokinetics – Reldesemtiv
      • Kadimastem – AstroRx
      • Mitsubishi Tanabe Pharma America – Oral Edaravone
      • Orphazyme – Arimoclomol
      • T Regulatory Cell Therapies
    • Approved Drugs
      • Radicava/Edaravone
      • Neudexta
      • Riluzole/Tiglutik
  • Support for Health Professionals
    • How to Break the News in ALS/MND
  • Events/Programs
    • Calendar of Events/Programs
    • Alliance Webinars
    • Alliance Meeting
    • Allied Professionals Forum
    • ALS/MND Connect
    • Global Day
    • March of Faces
    • International Symposium on ALS/MND
  • About
    • Who We Are
    • Board of Directors
    • Committees/Advisory Councils
      • Scientific Advisory Council
      • PALS and CALS Advisory Council
      • Innovation and Technology Council
      • Governance Committee
      • Budget and Finance Committee
    • Staff
    • History
    • Archives
      • Newsletters
      • Meetings
    • Awards
      • Student Award Nomination
      • Forbes Norris Award Nomination
      • Humanitarian Award Nomination
      • Allied Health Professional Award Nomination
  • 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.

For example, a drug normally undergoes three clinical trial phases in progressively larger groups of people before it is approved for clinical use, but Amylyx is trying to get their ALS/MND drug, AMX0035, to people sooner. AMX0035 slows down nerve cell death. In a phase 2 trial, the drug slowed ALS progression, and in a three-year open-label trial, the drug reduced the death rate by 50 percent. The company is going to apply to enter the drug into an expanded access program before it completes its phase 3 trial.

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

  • Roy

    Roy
    roy

  • Mark Miller

    Mark Miller

  • Erwin Coppejans, Diagnosed 2007 - ALS Liga België, Belgium

    Erwin Coppejans, Diagnosed 2007 – ALS Liga België, Belgium

  • Irene McCaughey, Diagnosed 2011 - MND Australia

    Irene McCaughey, Diagnosed 2011 – MND Australia

  • Jon Newsome, Les Turner ALS Foundation, USA

    Jon Newsome, Les Turner ALS Foundation, USA

  • Danny Reviers, Diagnosed 1979 - ALS Liga België, Belgium

    Danny Reviers, Diagnosed 1979 – ALS Liga België, Belgium

  • Orlando Ruiz, Diagnosed 2001 - ACELA, Colombia

    Orlando Ruiz, Diagnosed 2001 – ACELA, Colombia

  • Joyce Rusinak, Forbes Norris ALS Center, USA

    Joyce Rusinak, Forbes Norris ALS Center, USA

  • Francisco Perez Palop, Diagnosed 2013 - FUNDELA, Spain

    Francisco Perez Palop, Diagnosed 2013 – FUNDELA, Spain

  • Fernando Ocampo Cardona, Colombia

    Fernando Ocampo Cardona, Colombia

  • Zabun Nassar, MND Association, Diagnosed 2016, England

    Zabun Nassar, MND Association, Diagnosed 2016, England

  • Soledad Rodriguez, FUNDELA, Diagnosed 2013, Spain

    Soledad Rodriguez, FUNDELA, Diagnosed 2013, Spain

  • Sanjay Kumar Srivastava, Asha Ek Hope Foundation for ALS/MND, Diagnosed 2018, India

    Sanjay Kumar Srivastava, Asha Ek Hope Foundation for ALS/MND, Diagnosed 2018, India

  • Dorette Lüdi, Diagnosed 2014 - ALS Schweiz, Switzerland

    Dorette Lüdi, Diagnosed 2014 – ALS Schweiz, Switzerland

  • Inta Grubb, Diagnosed 2014 - MND Australia

    Inta Grubb, Diagnosed 2014 – MND Australia

  • Mary Thomas, Diagnosed 2013 - MND Australia

    Mary Thomas, Diagnosed 2013 – MND Australia

  • Luis Antonio Pimenta Lima, Brazil

    Luis Antonio Pimenta Lima, Brazil

  • Colm Francis Davis, Ireland

    Colm Francis Davis, Ireland

  • Susan Anderson, Diagnosed 2014 - Hope Loves Company, USA

    Susan Anderson, Diagnosed 2014 – Hope Loves Company, USA

  • Marcel R. Wernard, Diagnosed 2016 - ALS Patients Connected, The Netherlands

    Marcel R. Wernard, Diagnosed 2016 – ALS Patients Connected, The Netherlands

  • England-Lee-Millard, UK

    England-Lee-Millard, UK

  • Juvenal Bayona Romero

    Juvenal Bayona Romero

  • Joanne Pratt, Diagnosed 2011 - MND Australia

    Joanne Pratt, Diagnosed 2011 – MND Australia

  • Liong Ting Ngu, MND Malaysia, Diagnosed 2014, Malaysia

    Liong Ting Ngu, MND Malaysia, Diagnosed 2014, Malaysia

  • Lachlan Terry, MND Australia, Diagnosed 2015, Australia

    Lachlan Terry, MND Australia, Diagnosed 2015, Australia

  • Willi Klein

    Willi Klein

  • Phil Rossall, MND-Association, UK

    Phil Rossall, MND-Association, UK

  • Conny van der Meijden, Diagnosed 2001 - ALS Netherlands

    Conny van der Meijden, Diagnosed 2001 – ALS Netherlands

  • Cath Muir

    Cath Muir
    Cath

  • John and Loretta Russo, USA

    John and Loretta Russo, USA
    final3878

  • Chen Chun-Chin

    Chen Chun-Chin

  • Fabio Correia

    Fabio Correia

  • Eddy LeFrançois, Diagnosed 1992 - ALS Canada

    Eddy LeFrançois, Diagnosed 1992 – ALS Canada

  • Rolf Mauch, Association ALS Switzerland, Diagnosed 2015, Switzerland

    Rolf Mauch, Association ALS Switzerland, Diagnosed 2015, Switzerland

  • Brian Lovell, Diagnosed 2011 - MND Australia

    Brian Lovell, Diagnosed 2011 – MND Australia

  • Valdomiro Xavier Honório, Brazil

    Valdomiro Xavier Honório, Brazil

  • Carlos Alberto Arango, Colombia

    Carlos Alberto Arango, Colombia

  • Kirsten Harley, Diagnosed 2013 - Australia

    Kirsten Harley, Diagnosed 2013 – Australia

  • Amparo Muriel Engativa, Colombia

    Amparo Muriel Engativa, Colombia

  • Steven Spencer, Diagnosed 2014 - MND New Zealand

    Steven Spencer, Diagnosed 2014 – MND New Zealand

  • Fabrice Kamp, Germany

    Fabrice Kamp, Germany

  • Carlos Gomez Matallanas, Diagnosed 2014 - FUNDELA, Spain

    Carlos Gomez Matallanas, Diagnosed 2014 – FUNDELA, Spain

  • Timothy Holman, Switzerland

    Timothy Holman, Switzerland

  • David Watson, MND Scotland, Diagnosed 2018, Scotland

    David Watson, MND Scotland, Diagnosed 2018, Scotland

  • Norm MacIsaac, ALS Society of Canada, ALS Society of Quebec, Diagnosed 2014, Canada

    Norm MacIsaac, ALS Society of Canada, ALS Society of Quebec, Diagnosed 2014, Canada

  • Daniel Hare

    Daniel Hare

  • Oliver Juenke, DGM, Germany

    Oliver Juenke, DGM, Germany

  • Robbie Caliste, UK

    Robbie Caliste, UK

  • Art Eggert, USA

    Art Eggert, USA

  • Josée Kolijn-de Man, Diagnosed 2015 - ALS Patients Connected, The Netherlands

    Josée Kolijn-de Man, Diagnosed 2015 – ALS Patients Connected, The Netherlands

Learn more about the March of Faces

Latest Tweets

  • Just now

Subscribe to our Bi-Monthly Newsletter

Fill out the form below to subscribe to the Alliance’s bi-monthly newsletter, packed with updates from members associations from around the world!

Footer

Keep in touch

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
  • Instagram
  • Twitter
  • YouTube
Return to top of page

Contact | Disclaimer | Privacy Notice & Cookies | Sitemap

Copyright © 2023 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