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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

  • Lombana, Spain

    Lombana, Spain

  • Dorette Lüdi, Diagnosed 2014 , ALS Schweiz, Switzerland

    Dorette Lüdi, Diagnosed 2014 , ALS Schweiz, Switzerland

  • Graham Johnson, MND Australia

    Graham Johnson, MND Australia

  • Norm MacIsaac,  ALS Society of Canada,  ALS Society of Quebec,  Diagnosed 2014

    Norm MacIsaac, ALS Society of Canada, ALS Society of Quebec, Diagnosed 2014

  • Willi Klein

    Willi Klein

  • Verónica Isabel Castro Molina, Diagnosed 2014, Argentina

    Verónica Isabel Castro Molina, Diagnosed 2014, Argentina

  • Timmy, ALS Liga

    Timmy, ALS Liga

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

    Susan Anderson, Diagnosed 2014 , Hope Loves Company, USA

  • Liong Ting Ngu, MND Malaysia, Diagnosed 2014

    Liong Ting Ngu, MND Malaysia, Diagnosed 2014

  • Steve Gallagher, ALS Society of Canada

    Steve Gallagher, ALS Society of Canada
    Picture1

  • Ian Roberts

    Ian Roberts

  • Valdomiro Xavier Honório, Brazil

    Valdomiro Xavier Honório, Brazil

  • Juvenal Bayona Romero

    Juvenal Bayona Romero

  • England-Lee-Millard, UK

    England-Lee-Millard, UK

  • Elisabeth Zahnd, Switzerland

    Elisabeth Zahnd, Switzerland

  • Fabrice Kamp, Germany

    Fabrice Kamp, Germany

  • Mary Thomas, Diagnosed 2013 , MND Australia

    Mary Thomas, Diagnosed 2013 , MND Australia

  • Dr Shelly Hoover

    Dr Shelly Hoover

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

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

  • Wiebke Braach, Deutsche Gesellschaft für Muskelkranke, Germany

    Wiebke Braach, Deutsche Gesellschaft für Muskelkranke, Germany

  • Mahmood Anwar, UK

    Mahmood Anwar, UK

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

    Angie Bordaen, Diagnosed 2014, ALS Liga België, Belgium

  • Bob Simonds and Drew O'Neil, USA

    Bob Simonds and Drew O’Neil, USA

  • David Watson,  MND Scotland,  Diagnosed 2018

    David Watson, MND Scotland, Diagnosed 2018

  • Jose Rivero Muñoz, Diagnosed 2015, FYADENMAC, Mexico

    Jose Rivero Muñoz, Diagnosed 2015, FYADENMAC, Mexico

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

    Erwin Coppejans, Diagnosed 2007 , ALS Liga België, Belgium

  • Duncan Bayly , MND Australia

    Duncan Bayly , MND Australia

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

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

  • Orlando Ruiz, Diagnosed 2001,  ACELA, Colombia

    Orlando Ruiz, Diagnosed 2001, ACELA, Colombia

  • Hans Dieter Olszewski, Diagnosed 2010 , DGM, Germany

    Hans Dieter Olszewski, Diagnosed 2010 , DGM, Germany

  • Dad

    Dad

  • Alejandro Aquino, Diagnosed 2011 , Asociación ELA Argentina

    Alejandro Aquino, Diagnosed 2011 , Asociación ELA Argentina

  • Bayley, Australia

    Bayley, Australia

  • Maurice Leclerc, Canada

    Maurice Leclerc, Canada

  • Dick Dayton, USA

    Dick Dayton, USA

  • Steve

    Steve

  • 393647_2252248542053_984912751_n

    393647_2252248542053_984912751_n

  • Steven Gallagher, Canada

    Steven Gallagher, Canada

  • Horacio Fritzer, Argentina

    Horacio Fritzer, Argentina

  • Jason Goodman, Les Turner ALS Foundation, USA

    Jason Goodman, Les Turner ALS Foundation, USA

  • Carlos Gomez Matallanas, Diagnosed 2014 , FUNDELA, Spain

    Carlos Gomez Matallanas, Diagnosed 2014 , FUNDELA, Spain

  • Zelina Brito, Diagnosed 2018, Brazil

    Zelina Brito, Diagnosed 2018, Brazil

  • Claudette Sturk, ALS Society of Canada

    Claudette Sturk, ALS Society of Canada
    Picture2

  • Cassio Fernando da Silva, Diagnosed 2013 , ABrELA, Brazil

    Cassio Fernando da Silva, Diagnosed 2013 , ABrELA, Brazil

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

    Danny Reviers, Diagnosed 1979 , ALS Liga België, Belgium

  • Fabio Carvalho

    Fabio Carvalho

  • Mark Miller

    Mark Miller

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

    Bruno Leanza Mantegna, Diagnosed 1999 , AISLA Onlus, Italy

  • JP

    JP

  • Imelda Arenas, ACELA, Colombia

    Imelda Arenas, ACELA, Colombia

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