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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

  • David Bishop

    David Bishop

  • Jason Goodman, Les Turner ALS Foundation, USA

    Jason Goodman, Les Turner ALS Foundation, USA

  • Monica Soriano, Diagnosed 2011 ,  Asociación ELA , Argentina

    Monica Soriano, Diagnosed 2011 , Asociación ELA , Argentina

  • Oliver Juenke, Germany

    Oliver Juenke, Germany

  • Laurie Petit-Jean, Diagnosed 2012 , ARSLA, France

    Laurie Petit-Jean, Diagnosed 2012 , ARSLA, France

  • Claudia Cominetti, Associazione conSLAncio Onlus,  Italy

    Claudia Cominetti, Associazione conSLAncio Onlus, Italy

  • Eric Von Schaumburg, USA

    Eric Von Schaumburg, USA

  • Leon Ryba, Argentina

    Leon Ryba, Argentina

  • Hanne Stenmose, Muskelsvindfonden, Denmark

    Hanne Stenmose, Muskelsvindfonden, Denmark

  • Willi Klein

    Willi Klein

  • Jeff Sutherland

    Jeff Sutherland
    jspic

  • Luis Antonio Pimenta Lima, Brazil

    Luis Antonio Pimenta Lima, Brazil

  • Fabio Correia

    Fabio Correia

  • Dawn Morton, Diagnosed 2014 , MND Scotland, UK

    Dawn Morton, Diagnosed 2014 , MND Scotland, UK

  • Jean Waters, Diagnosed 2004, MND Association of England, Wales and N Ireland

    Jean Waters, Diagnosed 2004, MND Association of England, Wales and N Ireland

  • Diana Fernandez, Diagnosed 2009 , Asociación ELA Argentina

    Diana Fernandez, Diagnosed 2009 , Asociación ELA Argentina

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

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

  • Steve Lufkin, USA

    Steve Lufkin, USA
    IMG_3993

  • Carlos Alberto Arango, Colombia

    Carlos Alberto Arango, Colombia

  • 83

    83

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

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

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

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

  • Kris Van Reusel, Belgium

    Kris Van Reusel, Belgium

  • Mauril Bélanger, Diagnosed 2015 , ALS Canada

    Mauril Bélanger, Diagnosed 2015 , ALS Canada

  • Eddy LeFrançois, Diagnosed 1992,  ALS Canada

    Eddy LeFrançois, Diagnosed 1992, ALS Canada

  • Natalya Rybakova, Russian Charity ALS Foundation

    Natalya Rybakova, Russian Charity ALS Foundation

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

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

  • Steven Spencer, Diagnosed 2014 , MND New Zealand

    Steven Spencer, Diagnosed 2014 , MND New Zealand

  • João Marcos Andrietta, Diagnosed 2008 , ABrELA, Brazil

    João Marcos Andrietta, Diagnosed 2008 , ABrELA, Brazil

  • Duncan Bayly , MND Australia

    Duncan Bayly , MND Australia

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

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

  • Maurice Leclerc, Canada

    Maurice Leclerc, Canada

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

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

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

    Guoqiang Xu, Diagnosed 2016 , Shaanxi ALS Association, China

  • Liam Dwyer, England

    Liam Dwyer, England

  • Dad

    Dad

  • Brigitte Wernli,  Association ALS Switzerland,  Diagnosed 2014

    Brigitte Wernli, Association ALS Switzerland, Diagnosed 2014

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

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

  • Jose Rivero Muñoz, Diagnosed 2015, FYADENMAC, Mexico

    Jose Rivero Muñoz, Diagnosed 2015, FYADENMAC, Mexico

  • Wendy Hendrickson, ALS Hope Foundation, USA

    Wendy Hendrickson, ALS Hope Foundation, USA

  • England-Lee-Millard, UK

    England-Lee-Millard, UK

  • Jette Odgaard Villemoes, Muskelsvindfonden, Denmark

    Jette Odgaard Villemoes, Muskelsvindfonden, Denmark

  • Malcolm Buck, Australia

    Malcolm Buck, Australia

  • Horacio Fritzer, Argentina

    Horacio Fritzer, Argentina

  • Oliver Juenke, DGM, Germany

    Oliver Juenke, DGM, Germany

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

    Angie Bordaen, Diagnosed 2014, ALS Liga België, Belgium

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

    Bruno Leanza Mantegna, Diagnosed 1999 , AISLA Onlus, Italy

  • Tammy Moore and Eddy Lefrancois

    Tammy Moore and Eddy Lefrancois

  • Timothy Holman, Switzerland

    Timothy Holman, Switzerland

  • Alfredo Santos, Diagnosed 2013 , ACELA, Colombia

    Alfredo Santos, Diagnosed 2013 , ACELA, Colombia

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