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International Alliance of ALS/MND Associations

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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.

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Advocacy

  • Advocacy Toolkit

  • Marcelo Farinelli, Diagnosed 2006, ABrELA, Brazil

    Marcelo Farinelli, Diagnosed 2006, ABrELA, Brazil

  • Liam Dwyer, England

    Liam Dwyer, England

  • Juvenal Bayona Romero

    Juvenal Bayona Romero

  • Orlando Ruiz, Diagnosed 2001,  ACELA, Colombia

    Orlando Ruiz, Diagnosed 2001, ACELA, Colombia

  • Yolanda Armendariz, Diagnosed 2017 , FYADENMAC, Mexico

    Yolanda Armendariz, Diagnosed 2017 , FYADENMAC, Mexico

  • Emilienne Verhaegen, ALS Liga Belgium, Diagnosed 2014

    Emilienne Verhaegen, ALS Liga Belgium, Diagnosed 2014

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

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

  • Antonio Ventriglia,  ALS Liga Belgium,  Diagnosed 2013

    Antonio Ventriglia, ALS Liga Belgium, Diagnosed 2013

  • March of Faces Photo Submission_ALEX_ELA ARGENTINA

    March of Faces Photo Submission_ALEX_ELA ARGENTINA

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    727747090571358167

  • Maria Santos Garcia Tellez, Diagnosed 2017 , FYADENMAC, Mexico

    Maria Santos Garcia Tellez, Diagnosed 2017 , FYADENMAC, Mexico

  • Dick Dayton, USA

    Dick Dayton, USA

  • Brigitte Wernli,  Association ALS Switzerland,  Diagnosed 2014

    Brigitte Wernli, Association ALS Switzerland, Diagnosed 2014

  • Hollister

    Hollister
    hollister

  • Calum Ferguson, Diagnosed 2010 , MND Scotland, UK

    Calum Ferguson, Diagnosed 2010 , MND Scotland, UK

  • Joanne Pratt, Diagnosed 2011 , MND Australia

    Joanne Pratt, Diagnosed 2011 , MND Australia

  • Ana María Zavala, FYADENMAC, Diagnosed 2019, Mexico

    Ana María Zavala, FYADENMAC, Diagnosed 2019, Mexico

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

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

  • Lachlan Terry,  MND Australia,  Diagnosed 2015

    Lachlan Terry, MND Australia, Diagnosed 2015

  • Duncan Bayly , MND Australia

    Duncan Bayly , MND Australia

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

    Erwin Coppejans, Diagnosed 2007 , ALS Liga België, Belgium

  • Shay Rishoni, Diagnosed 2011 - Prize4Life, Israel

    Shay Rishoni, Diagnosed 2011 – Prize4Life, Israel

  • Jo Knowlton and her dog, Scotland

    Jo Knowlton and her dog, Scotland

  • Timothy Holman, Switzerland

    Timothy Holman, Switzerland

  • Dorette Lüdi, Diagnosed 2014 , ALS Schweiz, Switzerland

    Dorette Lüdi, Diagnosed 2014 , ALS Schweiz, Switzerland

  • Carlos Gomez Matallanas, Diagnosed 2014 , FUNDELA, Spain

    Carlos Gomez Matallanas, Diagnosed 2014 , FUNDELA, Spain

  • Rosie Riley, Les Turner ALS Foundation, USA

    Rosie Riley, Les Turner ALS Foundation, USA

  • Natalya Rybakova, Russia

    Natalya Rybakova, Russia

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

    Sharon Corosanite, Diagnosed 2014 , ALS Hope Foundation, USA

  • JP

    JP

  • Alan Liz Ogg 29042016 000799 lo res

    Alan Liz Ogg 29042016 000799 lo res

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

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

  • Graham Johnson, MND Australia

    Graham Johnson, MND Australia

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

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

  • Catherine Pearce, Australia

    Catherine Pearce, Australia

  • Rolf Mauch, Association ALS Switzerland, Diagnosed 2015

    Rolf Mauch, Association ALS Switzerland, Diagnosed 2015

  • Maria Lucia Wood Saldanha, Associação Pró-Cura da ELA, Brazil

    Maria Lucia Wood Saldanha, Associação Pró-Cura da ELA, Brazil

  • Joyce Rusinak, Forbes Norris ALS Center, USA

    Joyce Rusinak, Forbes Norris ALS Center, USA

  • unnamed

    unnamed

  • Oliver Juenke, Germany

    Oliver Juenke, Germany

  • Soledad Rodriguez, FUNDELA, Diagnosed 2013, Spain

    Soledad Rodriguez, FUNDELA, Diagnosed 2013, Spain

  • Debbie Craghill, USA

    Debbie Craghill, USA

  • Jose Espinosa, Argentina

    Jose Espinosa, Argentina

  • Ali Var, Turkey

    Ali Var, Turkey

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

    Danny Reviers, Diagnosed 1979 , ALS Liga België, Belgium

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

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

  • Mark Miller

    Mark Miller

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

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

  • Bjarne Hytjanstorp, ALS Norge, Norway

    Bjarne Hytjanstorp, ALS Norge, Norway

  • Bob Simonds and Drew O'Neill , Les Turner ALS Foundation, USA

    Bob Simonds and Drew O’Neill , Les Turner ALS Foundation, USA

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