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Right to Try

Right to Try

Background

The desire for someone living with ALS to try experimental therapeutics that lack complete knowledge of safety and/or efficacy is understandable. To many, the nature of the disease creates a situation where the risk of benefit often outweighs the risk of getting worse or death.

However,  the  existence  of  a  Right  to  Try  legislation in any  form  often does not  result  in  extensive utilization by a country’s citizens because it also requires the company that owns the treatment to comply.

For Right to Try to result in someone with ALS receiving an unapproved, often experimental, treatment, the following three things must ALL be in place.

    1. Some form of Right to Try legislation must exist, where government regulatory barriers that would normally safeguard citizens against unproven and unapproved treatments are reduced or removed.
      • Some countries have been willing to pass this type of legislation.
    2. An owner of a treatment, often a small or large pharmaceutical company, is willing to provide the treatment, usually while still in the clinical trial process.
      • Many companies with experimental treatments will hesitate to provide it through Right to Try while it remains in clinical development as any adverse events or misuse of the treatment under non-controlled conditions could jeopardize their program and potential to bring the treatment to market. Such a situation may not only be detrimental for the company/owner, but everyone living with ALS if an effective treatment is called into question due to a Right to Try issue.
    3. Cost coverage for the treatment must be identified, either through the owner providing it free of charge or the recipient paying out-of-pocket.
      • No oversight from government can allow for an owner to set their own price, which may be very high and exclusionary to some or many.
      • An owner may set prices at very high levels to compensate for risk of providing the treatment during an ongoing clinical development program.
      • Owners who don’t wish to make individuals pay out-of-pocket for what are often very expensive treatments to manufacture and deliver, often won’t be able to afford provision of the treatment to all through Right to Try.
      • Owners who decide to provide a limited amount of free treatment to a select group of people would likely be subject to scrutiny in a potential future market for them.

An additional, potential consequence of Right to Try legislation is the reduced level of protection against illegitimate companies/owners with products that may be unsafe or knowingly ineffective. Providing government legislated access to dubious treatments may assign a level of legitimacy to them on par with companies/owners who are taking the appropriate clinical trial steps to prove safety and efficacy of their product.

Recommendation

The SAC recommends that members of the Alliance should refrain from making any opinion-based statements about Right to Try and should refer those with questions to this document. Members should be aware of the realities of Right to Try (listed above) and compassionately provide information that balances an understanding of the position people with ALS are in, with the reasons why Right to Try legislation is not the only step in accessing experimental or unapproved treatments. Medical advice regarding any specific treatment should be deferred to an individual’s own clinician. Further objective information about what is known regarding a specific treatment in development may be sought through the SAC.

Further information

Often companies want to help people with ALS and truly care about developing and providing an effective therapy as quickly as possible. It is common for prominent clinical trials to offer extension studies, where all participants, regardless of whether they were on treatment or placebo during the trial, can receive the treatment at the company expense. These mechanisms allow for the treatment to continue in a controlled situation where risk is minimized and safety data can be collected.

 

International Alliance of ALS/MND Associations
June 2020

 


The original language of communication is English and any translation cannot be guaranteed for accuracy of messaging.

Primary Sidebar

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

    Susan Anderson, Diagnosed 2014 – Hope Loves Company, USA

  • 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

  • Oliver Juenke, DGM, Germany

    Oliver Juenke, DGM, Germany

  • Karl Hughes, Diagnosed 2010 - IMNDA, Ireland

    Karl Hughes, Diagnosed 2010 – IMNDA, Ireland

  • Sally Pauls, Diagnosed 2006 - Les Turner ALS Foundation

    Sally Pauls, Diagnosed 2006 – Les Turner ALS Foundation

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

    Liong Ting Ngu, MND Malaysia, Diagnosed 2014, Malaysia

  • Kirsten Harley, Diagnosed 2013 - Australia

    Kirsten Harley, Diagnosed 2013 – Australia

  • Wilfried Leusing, Diagnosed 2010 - DGM, Germany

    Wilfried Leusing, Diagnosed 2010 – DGM, Germany

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

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

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

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

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

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

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

    Brigitte Wernli, Association ALS Switzerland, Diagnosed 2014, Switzerland

  • Brian Lovell, Diagnosed 2011 - MND Australia

    Brian Lovell, Diagnosed 2011 – MND Australia

  • David Watson, MND Scotland, Diagnosed 2018, Scotland

    David Watson, MND Scotland, Diagnosed 2018, Scotland

  • Steven Spencer, Diagnosed 2014 - MND New Zealand

    Steven Spencer, Diagnosed 2014 – MND New Zealand

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

    Antonio Ventriglia, ALS Liga Belgium, Diagnosed 2013, Belgium

  • Philip Brindle, MND Association, Diagnosed 2015, England

    Philip Brindle, MND Association, Diagnosed 2015, England

  • Carlos Gomez Matallanas, Diagnosed 2014 - FUNDELA, Spain

    Carlos Gomez Matallanas, Diagnosed 2014 – FUNDELA, Spain

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

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

  • 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

  • Zelina-Brito-Diagnosed-2018-Brazil

    Zelina-Brito-Diagnosed-2018-Brazil

  • Monica Soriano, Diagnosed 2011 - Asociación ELA Argentina

    Monica Soriano, Diagnosed 2011 – Asociación ELA Argentina

  • Maurice LeClerc, ALS Canada

    Maurice LeClerc, ALS Canada

  • Dorette Lüdi, Diagnosed 2014 - ALS Schweiz, Switzerland

    Dorette Lüdi, Diagnosed 2014 – ALS Schweiz, Switzerland

  • Lachlan Terry, MND Australia, Diagnosed 2015, Australia

    Lachlan Terry, MND Australia, Diagnosed 2015, Australia

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

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

  • Jon Newsome, Les Turner ALS Foundation, USA

    Jon Newsome, Les Turner ALS Foundation, USA

  • Shay Rishoni, Diagnosed 2011 - Prize4Life, Israel

    Shay Rishoni, Diagnosed 2011 – Prize4Life, Israel

  • Ian Roberts

    Ian Roberts

  • Frank "Papa" Taylor

    Frank “Papa” Taylor

  • Denis Blais, Diagnosed 2015 - ALS Canada

    Denis Blais, Diagnosed 2015 – ALS Canada

  • Irene McCaughey, Diagnosed 2011 - MND Australia

    Irene McCaughey, Diagnosed 2011 – MND Australia

  • Mike Rannie, ALS Canada, Diagnosed 2017, Canada

    Mike Rannie, ALS Canada, Diagnosed 2017, Canada

  • 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

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

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

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

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

  • Angie Bordaen, Diagnosed 2014 - ALS Liga België, Belgium

    Angie Bordaen, Diagnosed 2014 – ALS Liga België, Belgium

  • Orlando Ruiz, Diagnosed 2001 - ACELA, Colombia

    Orlando Ruiz, Diagnosed 2001 – ACELA, Colombia

  • Inta Grubb, Diagnosed 2014 - MND Australia

    Inta Grubb, Diagnosed 2014 – MND Australia

  • Duncan Bayly - MND Australia

    Duncan Bayly – MND Australia

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

    Rolf Mauch, Association ALS Switzerland, Diagnosed 2015, Switzerland

  • Eddy LeFrançois, Diagnosed 1992 - ALS Canada

    Eddy LeFrançois, Diagnosed 1992 – ALS Canada

  • Soledad Rodriguez, FUNDELA, Diagnosed 2013, Spain

    Soledad Rodriguez, FUNDELA, Diagnosed 2013, Spain

  • Chris McCauley, Diagnosed 2015 - ALS Canada

    Chris McCauley, Diagnosed 2015 – ALS Canada

  • Glen Elison, ALS Hope Foundation, Diagnosed 2019, USA

    Glen Elison, ALS Hope Foundation, Diagnosed 2019, USA

  • Roxana Canova, Diagnosed 2012 - Asociación ELA Argentina

    Roxana Canova, Diagnosed 2012 – Asociación ELA Argentina

  • Joanne Pratt, Diagnosed 2011 - MND Australia

    Joanne Pratt, Diagnosed 2011 – MND Australia

  • Conny van der Meijden, Diagnosed 2001 - ALS Netherlands

    Conny van der Meijden, Diagnosed 2001 – ALS Netherlands

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

    Erwin Coppejans, Diagnosed 2007 – ALS Liga België, Belgium

Learn more about the March of Faces

Advocacy

  • Advocacy Toolkit
  • Rights of People with ALS/MND
  • Biogen – Tofersen
  • BrainStorm Cell Therapeutics – NurOwn
  • Collaborative Medicinal Development – CuATSM
  • Cytokinetics – Reldasemtiv
  • Right to Try
  • Unproven (Off-Label) Treatments
  • Amylyx – AMX0035
  • Kadimastem – AstroRx

Allied Professionals Forum Registration

The International Alliance of ALS/MND Associations will host a virtual Allied Professionals Forum spread over two days to accommodate time zones on 3 – 4 December 2020.

Click here to register for the Allied Professionals Forum

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