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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

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    Armando González Gómez, ACELA, Colombia

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    Ana Lilia RodriguezApoyo Integral Gila A.C., Diagnosed 2018, Mexico

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    Imelda Arenas, ACELA, Colombia

  • Stephanie Christiansen Hall, Canada

    Stephanie Christiansen Hall, Canada

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    IMG_2658

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

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

  • Jorge Melo, ABrELA, Brazil

    Jorge Melo, ABrELA, Brazil

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

    Guoqiang Xu, Diagnosed 2016 , Shaanxi ALS Association, China

  • Joy Blakeley, Diagnosed 2017 , MND Australia

    Joy Blakeley, Diagnosed 2017 , MND Australia

  • Juvenal Bayona Romero

    Juvenal Bayona Romero

  • Ismail Gokcek, Turkey

    Ismail Gokcek, Turkey
    ismail_gokcek_alsmnd_tr

  • Rolf Mauch, Association ALS Switzerland, Diagnosed 2015

    Rolf Mauch, Association ALS Switzerland, Diagnosed 2015

  • Anita Forte, Les Turner ALS Foundation, USA

    Anita Forte, Les Turner ALS Foundation, USA

  • Ian Roberts

    Ian Roberts

  • Jack Buzby, USA

    Jack Buzby, USA

  • Phil Rossall, MND-Association, UK

    Phil Rossall, MND-Association, UK

  • H. Todd Kelly, Diagnosed 2013 , ALS Hope Foundation, USA

    H. Todd Kelly, Diagnosed 2013 , ALS Hope Foundation, USA

  • Eddy LeFrançois, Diagnosed 1992,  ALS Canada

    Eddy LeFrançois, Diagnosed 1992, ALS Canada

  • Ali Var, Turkey

    Ali Var, Turkey

  • Purningam Jacob, Diagnosed 2012 , Asha Ek Hope Foundation, India

    Purningam Jacob, Diagnosed 2012 , Asha Ek Hope Foundation, India

  • Jo Knowlton and her dog, Scotland

    Jo Knowlton and her dog, Scotland

  • Duncan Bayly , MND Australia

    Duncan Bayly , MND Australia

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

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

  • Diana Fernandez, Diagnosed 2009 , Asociación ELA Argentina

    Diana Fernandez, Diagnosed 2009 , Asociación ELA Argentina

  • Chen Chun-Chin

    Chen Chun-Chin

  • Bruno Leanza Mantegna, Diagnosed 1999 , AISLA Onlus, Italy

    Bruno Leanza Mantegna, Diagnosed 1999 , AISLA Onlus, Italy

  • Claudia Gotti, Brazil

    Claudia Gotti, Brazil

  • JP

    JP

  • Ada Garrido Benavidez, Diagnosed 2016,  FYADENMAC, Mexico

    Ada Garrido Benavidez, Diagnosed 2016, FYADENMAC, Mexico

  • Xian-Zhang Niu, Diagnosed 2006 , Shaanxi ALS Association, China

    Xian-Zhang Niu, Diagnosed 2006 , Shaanxi ALS Association, China

  • Luis Antonio Pimenta Lima, Brazil

    Luis Antonio Pimenta Lima, Brazil

  • 83

    83

  • Yolanda Armendariz, Diagnosed 2017 , FYADENMAC, Mexico

    Yolanda Armendariz, Diagnosed 2017 , FYADENMAC, Mexico

  • Carlos Alberto Báez Murillo, ACELA, Colombia

    Carlos Alberto Báez Murillo, ACELA, Colombia

  • Sally Pauls, Diagnosed 2006 , Les Turner ALS Foundation

    Sally Pauls, Diagnosed 2006 , Les Turner ALS Foundation

  • Oliver Juenke, DGM, Germany

    Oliver Juenke, DGM, Germany

  • Mary Thomas, Diagnosed 2013 , MND Australia

    Mary Thomas, Diagnosed 2013 , MND Australia

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    Steve

  • Dr Shelly Hoover

    Dr Shelly Hoover

  • Dick Dayton, USA

    Dick Dayton, USA

  • Colm Francis Davis, Ireland

    Colm Francis Davis, Ireland

  • Karl Hughes, Diagnosed 2010 , IMNDA,  Ireland

    Karl Hughes, Diagnosed 2010 , IMNDA, Ireland

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    727747090571358167

  • Jan Zuring, Diagnosed 2010 , The Netherlands

    Jan Zuring, Diagnosed 2010 , The Netherlands

  • Chun Ju Xiao, China

    Chun Ju Xiao, China

  • Bob Simonds and Drew O'Neil, USA

    Bob Simonds and Drew O’Neil, USA

  • Bayley, Australia

    Bayley, Australia

  • Ann Nicol

    Ann Nicol

  • Gudjon Sigurdsson, Diagnosed 2004 , MND Association of Iceland

    Gudjon Sigurdsson, Diagnosed 2004 , MND Association of Iceland

  • Len Johnrose,  MND Association,  Diagnosed 2017,  England

    Len Johnrose, MND Association, Diagnosed 2017, England

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