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International Alliance of ALS/MND Associations

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US FDA Orphan Drug Designation

US FDA Orphan Drug Designation

Background

As our understanding of the underlying biology of ALS has improved, multiple experimental treatment approaches have been identified. Currently, there are numerous interventional drug trials testing a diverse set of targets using multiple drug approaches (e.g. small molecules, antibodies, stem cells, gene interface/silencing technologies). Drug regulators have also set up programs to provide special incentives for companies to develop drugs and biologicals for rare diseases that have a small market (fewer than 200,000 people such as the ALS community). One such program is the orphan drug designation (or sometimes “orphan status”) administered at the Food and Drug Administration (FDA) of the United States. Created in 1983, this program provides financial incentives and research subsidies such as partial tax credit for clinical trial expenditures, waived user fees, and eligibility for market exclusivity for that drug. The European Medicines Agency (EMA) of the European Union also has a similar program and defines a drug as “orphan” for a rare disease affecting fewer than 5 in 10,000 people. As of 2020, over 80 drugs have been designated by the FDA with the orphan drug status for ALS including recent example,s Cytokinetics’ drug reldesemtiv and Neuropore’s drug NPT520-34.

Benefit for patients
The National Organization for Rare Disorders along with many other organizations led the lobbying efforts for the passage and formation of this program. Companies will typically shy away from developing drugs for a rare disease area due to financial considerations, limited profit margins, as well as the scientific, ethical and operational complexities of conducting clinical research in small niche patient populations. This program motivates, supports and incentivizes drug companies to invest their research and development capital (both scientific and financial) with the hope that more medical breakthroughs will be made available for patients with rare diseases than otherwise would have been achieved.

Benefit for companies/sponsors
Companies benefit from this program as it allows them exclusive marketing and development rights and allows them to partially recover the costs of research and developing the drug. In addition to cost reductions, the FDA provides streamlining of regulatory processes and guidance for those drugs with such a designation. Additionally, investors in pharmaceutical companies often view orphan designation as a signal of higher company value, thus bringing in more capital needed for the drug discovery program.

What orphan drug designation means and does not mean
While the orphan drug designation can be an important milestone for a drug company, it is important to note that orphan drugs, like non-orphan drugs, are still required to show safety and efficacy prior to approval for use as a therapeutic. Orphan drug designations are typically given early on in development and orphan drugs must still follow the proper clinical development and regulatory process. It is also important to state that orphan drug designation should not be confused with other regulatory designations such as fast-track designation. Fast-track designation does not provide drug companies with any explicit financial benefits for developing its drug, however, it does provide more frequent meetings with the FDA, as well as an expedited review when submitting an application to bring a new drug to market.

Recommendation

The SAC hopes that drug companies take advantage of incentives and expedited pathways set up by regulatory agencies such as the orphan drug designation to discover and rigorously test new treatments for people with ALS.

 

International Alliance of ALS/MND Associations
July 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

  • Amparo Muriel Engativa, Colombia

    Amparo Muriel Engativa, Colombia

  • Greg Heydet, ALS Hope Foundation, USA

    Greg Heydet, ALS Hope Foundation, USA

  • Bob Simonds and Drew O'Neil, USA

    Bob Simonds and Drew O’Neil, USA

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

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

  • Wiebke Braach, Deutsche Gesellschaft für Muskelkranke, Germany

    Wiebke Braach, Deutsche Gesellschaft für Muskelkranke, Germany

  • Carlos Alberto Arango, Colombia

    Carlos Alberto Arango, Colombia

  • 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

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

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

  • Ali Var, Turkey

    Ali Var, Turkey

  • Carlos Alberto Báez Murillo, ACELA, Colombia

    Carlos Alberto Báez Murillo, ACELA, Colombia

  • Bjarne Hytjanstorp, ALS Norge, Norway

    Bjarne Hytjanstorp, ALS Norge, Norway

  • Jette Odgaard Villemoes, Muskelsvindfonden, Denmark

    Jette Odgaard Villemoes, Muskelsvindfonden, Denmark

  • Lin Yong Yi, Taiwan MND Association, Diagnosed 2004

    Lin Yong Yi, Taiwan MND Association, Diagnosed 2004

  • Alan Liz Ogg 29042016 000799 lo res

    Alan Liz Ogg 29042016 000799 lo res

  • Luis Antonio Pimenta Lima, Brazil

    Luis Antonio Pimenta Lima, Brazil

  • Michel Perrozzo, ARSLA, Diagnosed 2015, France

    Michel Perrozzo, ARSLA, Diagnosed 2015, France

  • Gudjon Sigurdsson, Diagnosed 2004 , MND Association of Iceland

    Gudjon Sigurdsson, Diagnosed 2004 , MND Association of Iceland

  • Conny van der Meijden, Diagnosed 2001,  ALS Netherlands

    Conny van der Meijden, Diagnosed 2001, ALS Netherlands

  • Enzo Maccarrone, AISLA ONLUS, Italy

    Enzo Maccarrone, AISLA ONLUS, Italy

  • Imelda Arenas, ACELA, Colombia

    Imelda Arenas, ACELA, Colombia

  • Guido De Mets, Belgium

    Guido De Mets, Belgium

  • Francisco Perez Palop, Diagnosed 2013 , FUNDELA, Spain

    Francisco Perez Palop, Diagnosed 2013 , FUNDELA, Spain

  • Maurice LeClerc, ALS Canada

    Maurice LeClerc, ALS Canada

  • John Dinon, MND Australia

    John Dinon, MND Australia

  • Frank "Papa" Taylor, USA

    Frank “Papa” Taylor, USA

  • Joanne Pratt, Diagnosed 2011 , MND Australia

    Joanne Pratt, Diagnosed 2011 , MND Australia

  • 393647_2252248542053_984912751_n

    393647_2252248542053_984912751_n

  • Ana María Zavala, FYADENMAC, Diagnosed 2019, Mexico

    Ana María Zavala, FYADENMAC, Diagnosed 2019, Mexico

  • Claire Garry, USA

    Claire Garry, USA
    20200117_214643

  • Soledad Rodriguez, FUNDELA, Diagnosed 2013, Spain

    Soledad Rodriguez, FUNDELA, Diagnosed 2013, Spain

  • Chun Ju Xiao, China

    Chun Ju Xiao, China

  • Paul Launer, USA

    Paul Launer, USA

  • Rolf Mauch, Association ALS Switzerland, Diagnosed 2015

    Rolf Mauch, Association ALS Switzerland, Diagnosed 2015

  • Brigitte Wernli,  Association ALS Switzerland,  Diagnosed 2014

    Brigitte Wernli, Association ALS Switzerland, Diagnosed 2014

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

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

  • Eddy LeFrançois, Diagnosed 1992,  ALS Canada

    Eddy LeFrançois, Diagnosed 1992, ALS Canada

  • Roxana Canova, Diagnosed 2012 ,  Asociación ELA Argentina

    Roxana Canova, Diagnosed 2012 , Asociación ELA Argentina

  • Juvenal Bayona Romero

    Juvenal Bayona Romero

  • Hans Dieter Olszewski, Diagnosed 2010 , DGM, Germany

    Hans Dieter Olszewski, Diagnosed 2010 , DGM, Germany

  • Antonio Ventriglia,  ALS Liga Belgium,  Diagnosed 2013

    Antonio Ventriglia, ALS Liga Belgium, Diagnosed 2013

  • Andrea Zicchieri, Associazione conSLAncio Onlus, Italy

    Andrea Zicchieri, Associazione conSLAncio Onlus, Italy
    AndreaZicchieri_conSLAncioItaly

  • Denis Blais, Diagnosed 2015 , ALS Canada

    Denis Blais, Diagnosed 2015 , ALS Canada

  • Steve Lufkin, USA

    Steve Lufkin, USA
    IMG_3993

  • Daniel Hare

    Daniel Hare

  • Natalya Rybakova, Russian Charity ALS Foundation

    Natalya Rybakova, Russian Charity ALS Foundation

  • Jo Knowlton and her dog, Scotland

    Jo Knowlton and her dog, Scotland

  • 727747090571358167

    727747090571358167

  • Roy

    Roy
    roy

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

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

  • March of Faces Photo Submission_ALEX_ELA ARGENTINA

    March of Faces Photo Submission_ALEX_ELA ARGENTINA

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