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International Alliance of ALS/MND Associations

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    • Fundamental Rights for People with ALS/MND and Caregivers
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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.

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Advocacy

  • Advocacy Toolkit

  • 83

    83

  • 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

  • Jean

    Jean
    jean

  • Fernando Ocampo Cardona, Colombia

    Fernando Ocampo Cardona, Colombia

  • Horacio Fritzer, Argentina

    Horacio Fritzer, Argentina

  • Shay Rishoni

    Shay Rishoni

  • Armando González Gómez, ACELA, Colombia

    Armando González Gómez, ACELA, Colombia

  • Juvenal Bayona Romero

    Juvenal Bayona Romero

  • Fabio Correia

    Fabio Correia

  • Natalya Rybakova, Russian Charity ALS Foundation

    Natalya Rybakova, Russian Charity ALS Foundation

  • Claire Garry, USA

    Claire Garry, USA
    20200117_214643

  • Jason Goodman, Les Turner ALS Foundation, USA

    Jason Goodman, Les Turner ALS Foundation, USA

  • Teddy Hanono Annie, Apoyo Integral Gila A.C., Diagnosed 2018, Mexico

    Teddy Hanono Annie, Apoyo Integral Gila A.C., Diagnosed 2018, Mexico

  • Lucy Lintott, Diagnosed 2013 , MND Scotland, UK

    Lucy Lintott, Diagnosed 2013 , MND Scotland, UK

  • Osiel Mendoza, Diagnosed 2016 ,  ALS Therapy Development Institute, USA

    Osiel Mendoza, Diagnosed 2016 , ALS Therapy Development Institute, USA

  • Catherine Pearce, Australia

    Catherine Pearce, Australia

  • Jette Odgaard Villemoes, Muskelsvindfonden, Denmark

    Jette Odgaard Villemoes, Muskelsvindfonden, Denmark

  • Mauril Bélanger, Diagnosed 2015 , ALS Canada

    Mauril Bélanger, Diagnosed 2015 , ALS Canada

  • Leon Ryba, Argentina

    Leon Ryba, Argentina

  • Oliver Juenke, DGM, Germany

    Oliver Juenke, DGM, Germany

  • Peng Yi-Wen

    Peng Yi-Wen

  • Mauril Belanger

    Mauril Belanger

  • Phil Rossall, MND-Association, UK

    Phil Rossall, MND-Association, UK

  • Shay Rishoni, Diagnosed 2011 , Prize4Life, Israel

    Shay Rishoni, Diagnosed 2011 , Prize4Life, Israel

  • Alan Liz Ogg 29042016 000799 lo res

    Alan Liz Ogg 29042016 000799 lo res

  • Amparo Muriel Engativa, Colombia

    Amparo Muriel Engativa, Colombia

  • JP

    JP

  • Lombana, Spain

    Lombana, Spain

  • João Marcos Andrietta, Diagnosed 2008 , ABrELA, Brazil

    João Marcos Andrietta, Diagnosed 2008 , ABrELA, Brazil

  • Elisabeth Zahnd, Switzerland

    Elisabeth Zahnd, Switzerland

  • Philip Brindle,  MND Association,  Diagnosed 2015,  England

    Philip Brindle, MND Association, Diagnosed 2015, England

  • Angela Jansen, Deutsche Gesellschaft für Muskelkranke e.V.-DGM, Diagnosed 1995, Germany

    Angela Jansen, Deutsche Gesellschaft für Muskelkranke e.V.-DGM, Diagnosed 1995, Germany

  • Ali Var, Turkey

    Ali Var, Turkey

  • IMG_1211

    IMG_1211

  • Hanne Stenmose, Muskelsvindfonden, Denmark

    Hanne Stenmose, Muskelsvindfonden, Denmark

  • Charlie “Hark” Dourney, Diagnosed 2007 , Hark ALS, USA

    Charlie “Hark” Dourney, Diagnosed 2007 , Hark ALS, USA

  • Calum Ferguson, Diagnosed 2010 , MND Scotland, UK

    Calum Ferguson, Diagnosed 2010 , MND Scotland, UK

  • Paul Launer, USA

    Paul Launer, USA

  • Len Johnrose,  MND Association,  Diagnosed 2017,  England

    Len Johnrose, MND Association, Diagnosed 2017, England

  • Guoqiang Xu, Diagnosed 2016 , Shaanxi ALS Association, China

    Guoqiang Xu, Diagnosed 2016 , Shaanxi ALS Association, China

  • Shera Mukherjee, Diagnosed 2013,  Asha Ek Hope Foundation, India

    Shera Mukherjee, Diagnosed 2013, Asha Ek Hope Foundation, India

  • Alfredo Santos, Diagnosed 2013 , ACELA, Colombia

    Alfredo Santos, Diagnosed 2013 , ACELA, Colombia

  • Bob Simonds and Drew O'Neil, USA

    Bob Simonds and Drew O’Neil, USA

  • Steven Gallagher, Canada

    Steven Gallagher, Canada

  • Conny van der Meijden, Diagnosed 2001,  ALS Netherlands

    Conny van der Meijden, Diagnosed 2001, ALS Netherlands

  • Elkin Ramiro Gaviria Muñoz, Diagnosed December 2018

    Elkin Ramiro Gaviria Muñoz, Diagnosed December 2018

  • Carlos Alberto Báez Murillo, ACELA, Colombia

    Carlos Alberto Báez Murillo, ACELA, Colombia

  • Yessenia Hernandez Mendoza, Apoyo Integral Gila A.C., Diagnosed 2018, Mexico

    Yessenia Hernandez Mendoza, Apoyo Integral Gila A.C., Diagnosed 2018, Mexico

  • Laurie Petit-Jean, Diagnosed 2012 , ARSLA, France

    Laurie Petit-Jean, Diagnosed 2012 , ARSLA, France

  • Sharon Corosanite, Diagnosed 2014 , ALS Hope Foundation, USA

    Sharon Corosanite, Diagnosed 2014 , ALS Hope Foundation, USA

Learn more about the March of Faces

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