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International Alliance of ALS/MND Associations

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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.

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

  • Fundamental Rights of People with ALS/MND and Caregivers
  • Advocacy Toolkit
  • Understanding ALS/MND Research
  • Drug Pathways
  • Right to Try
  • US FDA Orphan Drug Designation
  • Unproven (Off-Label) Treatments
  • Open Label Extension
  • Expanded Access

  • ALS-0571

    ALS-0571

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

    Susan Anderson, Diagnosed 2014 – Hope Loves Company, USA

  • Lachlan Terry, MND Australia, Diagnosed 2015, Australia

    Lachlan Terry, MND Australia, Diagnosed 2015, Australia

  • 20200117_214643

    20200117_214643

  • Phil-Rossall-MND-Association

    Phil-Rossall-MND-Association

  • Inta Grubb, Diagnosed 2014 - MND Australia

    Inta Grubb, Diagnosed 2014 – MND Australia

  • Ann-Nicol

    Ann-Nicol

  • Dorette Lüdi, Diagnosed 2014 - ALS Schweiz, Switzerland

    Dorette Lüdi, Diagnosed 2014 – ALS Schweiz, Switzerland

  • 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

  • Joy Blakeley, Diagnosed 2017 - MND Australia

    Joy Blakeley, Diagnosed 2017 – MND Australia

  • Brigitte Wernli, Association ALS Switzerland, Diagnosed 2014, Switzerland

    Brigitte Wernli, Association ALS Switzerland, Diagnosed 2014, Switzerland

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

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

  • Alejandro Aquino, Diagnosed 2011 - Asociación ELA Argentina

    Alejandro Aquino, Diagnosed 2011 – Asociación ELA Argentina

  • Elkin-Ramiro-Gaviria-Muñoz-diagnosed-Dec-2018-v3

    Elkin-Ramiro-Gaviria-Muñoz-diagnosed-Dec-2018-v3

  • pALS-Guido-De-Mets-ALS-Liga-Belgium

    pALS-Guido-De-Mets-ALS-Liga-Belgium

  • Antonio Ventriglia, ALS Liga Belgium, Diagnosed 2013, Belgium

    Antonio Ventriglia, ALS Liga Belgium, Diagnosed 2013, Belgium

  • Richard Clark, MND New Zealand, Diagnosed 2011, New Zealand

    Richard Clark, MND New Zealand, Diagnosed 2011, New Zealand

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

    Erwin Coppejans, Diagnosed 2007 – ALS Liga België, Belgium

  • Zabun Nassar, MND Association, Diagnosed 2016, England

    Zabun Nassar, MND Association, Diagnosed 2016, England

  • Shay Rishoni, Diagnosed 2011 - Prize4Life, Israel

    Shay Rishoni, Diagnosed 2011 – Prize4Life, Israel

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

    Danny Reviers, Diagnosed 1979 – ALS Liga België, Belgium

  • Duncan Bayly - MND Australia

    Duncan Bayly – MND Australia

  • Ailsa Malcolm-Hutton, Diagnosed 2013 - MND Association of England, Wales and N Ireland

    Ailsa Malcolm-Hutton, Diagnosed 2013 – MND Association of England, Wales and N Ireland

  • Fabio-Carvalho3

    Fabio-Carvalho3

  • Monica Soriano, Diagnosed 2011 - Asociación ELA Argentina

    Monica Soriano, Diagnosed 2011 – Asociación ELA Argentina

  • MoF-Christian-Bär

    MoF-Christian-Bär

  • Valdomiro-Xavier-Honorio-ABrELA

    Valdomiro-Xavier-Honorio-ABrELA

  • Willi-Klein

    Willi-Klein

  • Carlos Gomez Matallanas, Diagnosed 2014 - FUNDELA, Spain

    Carlos Gomez Matallanas, Diagnosed 2014 – FUNDELA, Spain

  • Orlando Ruiz, Diagnosed 2001 - ACELA, Colombia

    Orlando Ruiz, Diagnosed 2001 – ACELA, Colombia

  • Mike Rannie, ALS Canada, Diagnosed 2017, Canada

    Mike Rannie, ALS Canada, Diagnosed 2017, Canada

  • Steven Spencer, Diagnosed 2014 - MND New Zealand

    Steven Spencer, Diagnosed 2014 – MND New Zealand

  • 9B63A612-09B4-453E-8430-B76DB0AB5196

    9B63A612-09B4-453E-8430-B76DB0AB5196

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

    Rolf Mauch, Association ALS Switzerland, Diagnosed 2015, Switzerland

  • Maurice LeClerc, ALS Canada

    Maurice LeClerc, ALS Canada

  • Sally Pauls, Diagnosed 2006 - Les Turner ALS Foundation

    Sally Pauls, Diagnosed 2006 – Les Turner ALS Foundation

  • Philip Brindle, MND Association, Diagnosed 2015, England

    Philip Brindle, MND Association, Diagnosed 2015, England

  • Len Johnrose, MND Association, Diagnosed 2017, England

    Len Johnrose, MND Association, Diagnosed 2017, England

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

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

  • IMG_3993

    IMG_3993

  • Wilfried Leusing, Diagnosed 2010 - DGM, Germany

    Wilfried Leusing, Diagnosed 2010 – DGM, Germany

  • Fabio-Correia-2

    Fabio-Correia-2

  • Margreth Burger-Saile, Diagnosed 2011 - ALS Schweiz, Switzerland

    Margreth Burger-Saile, Diagnosed 2011 – ALS Schweiz, Switzerland

  • Mary Thomas, Diagnosed 2013 - MND Australia

    Mary Thomas, Diagnosed 2013 – MND Australia

  • Brian Lovell, Diagnosed 2011 - MND Australia

    Brian Lovell, Diagnosed 2011 – MND Australia

  • Luis-Antonio-Pimenta-Lima-ABrELA

    Luis-Antonio-Pimenta-Lima-ABrELA

  • Jo-and-her-dog-2

    Jo-and-her-dog-2

  • Eddy LeFrançois, Diagnosed 1992 - ALS Canada

    Eddy LeFrançois, Diagnosed 1992 – ALS Canada

  • jspic

    jspic

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

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

Learn more about the March of Faces

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