PPMD Supports Advancing Targeted Therapies for Rare Diseases Act of 2015 (S. 2030)

For over a year, PPMD has been working with congressional champions and industry partners to help lead an effort to streamlining the regulatory pathways and review processes for targeted therapeutics (which would include such things as 'follow on exons') by clarifying the FDA’s existing authority to leverage data previously used in the approval of a targeted product when approving a new therapy.

These efforts have yielded a provision within HR 6 the 21st Century Cures Act which was approved this past summer, as well as a Senate bill called the Advancing Targeted Therapies for Rare Diseases Act of 2015 (S. 2030).  

For that reason, we collaborated with the National Organization for Rare Disorders (NORD), the Muscular Dystrophy Association (MDA), the Duchenne Alliance, and many others to demonstrate the value of this bill and the power of our rare disease community.

Please read the letter that PPMD is proud to be a signer on:

(download PDF)

November 12, 2015

The Honorable Lamar Alexander, Chairman
Senate Committee on Health, Education, Labor and Pensions
428 Dirksen Senate Office Building
Washington, D.C. 20510

The Honorable Patty Murray, Ranking Member
Senate Committee on Health, Education Labor and Pensions
428 Dirksen Senate Office Building
Washington, D.C. 20510

Dear Chairman Alexander and Ranking Member Murray:

The undersigned organizations, representing millions of Americans living with a rare disease, write today to express our strong support for the Advancing Targeted Therapies for Rare Diseases Act of 2015 (S. 2030). If enacted, this legislation could greatly accelerate the pace of therapeutic development for rare diseases and their sub-populations by streamlining the regulatory review process, without compromising safety and efficacy standards.

This legislation clarifies FDA’s existing authority to leverage data previously used in the approval of a targeted drug when approving a new therapy that “incorporates or utilizes the same or similar genetically targeted technology, or the same variant protein targeted technology”.

This authority is critically important to accelerating the development of treatments and cures for the numerous devastating rare diseases or subsets of rare diseases that otherwise have little hope of a treatment or cure due to their extremely small population size. By clarifying that the FDA is allowed to use previously accepted data in certain circumstances, this provision will help facilitate the development and approval of therapies for these diseases.

This bill does not jeopardize intellectual property or patent protections, as only the sponsor that generated the original data may leverage that same data for future uses, unless the sponsor grants permission otherwise. Additionally, this legislation does not bind the FDA in its review processes, and instead reiterates existing flexibility in the data they are permitted to accept.

This bill is supported throughout the rare disease stakeholder community, and we urge the Senate Committee on Health, Education, Labor and Pensions to include this bill in the Senate Innovation for Healthier Americans Initiative.

Thank you for your continued dedication to the rare disease community, and we look forward to working with you to ensure this provision is enacted.


  • AKU Society of North America
  • Alpha-1 Foundation
  • ALS Association
  • Alternating Hemiplegia of Childhood Foundation
  • American Behcet’s Disease Association (ABDA)
  • American Brain Tumor Association
  • American Multiple Endocrine Neoplasia Support (AMEN Support)
  • Association for Frontotemporal Degeneration (AFTD)
  • Association of Gastrointestinal Motility Disorders, Inc. (AGMD)
  • Association for Glycogen Storage Disease
  • Batten Disease Support and Research Association
  • BCC Nevus Syndrome Life Support Network
  • BRBN Alliance
  • Children's PKU Network
  • Cholangiocarcinoma Foundation
  • Chromosome Disorder Outreach Inc.
  • Cicatricial Alopecia Research Foundation
  • Council For Bile Acid Deficiency Diseases
  • CureCADASIL (CADASIL Association Inc.)
  • CureCMD
  • Cure HHT
  • Cutaneous Lymphoma Foundation
  • The Desmoid Tumor Research Foundation, Inc.
  • Dystonia Advocacy Network
  • Dystonia Medical Research Foundation
  • dystrophic epidermolysis bullosa research association of America (debra of America)
  • The Erythromelalgia Association
  • Everylife Foundation for Rare Diseases
  • Foundation for Ichthyosis & Related Skin Types, Inc.
  • Foundation for Prader-Willi Research
  • Foundation to Eradicate Duchenne (FED)
  • Friedreich's Ataxia Research Alliance (FARA)
  • GBS/CIDP Foundation International
  • The Global Foundation for Peroxisomal Disorders
  • The Guthy – Jackson Charitable Foundation
  • Hermansky-Pudlak Syndrome Network Inc.
  • Histiocytosis Association
  • HLRCC Family Alliance
  • The Huntington’s Disease Society of America
  • HypoPARAthyroidism Association
  • Immune Deficiency Foundation
  • Indian Organization for Rare Disorders
  • The International Advocate for Glycoprotein Storage Diseases
  • International FOP Association
  • International Foundation for CDKL5 Research
  • International Myeloma Foundation
  • International Pemphigus and Pemphigoid Foundation (IPPF)
  • International WAGR Syndrome Association
  • Jack McGovern Coats’ Disease Foundation
  • Kennedy's Disease Association
  • LAL Solace
  • The Life Raft Group
  • Lymphangiomatosis & Gorham's Disease Alliance
  • The Marfan Foundation
  • MEBO Research
  • MitoAction
  • Moebius Syndrome Foundation
  • The Morgan Leary Vaughan Fund
  • Mucolipidosis Type IV Foundation
  • Muscular Dystrophy Association (MDA)
  • The Myositis Association
  • National Adrenal Diseases Foundation
  • National Alopecia Areata Foundation
  • National Ataxia Foundation
  • National Eosinophilia Myalgia Syndrome Network
  • National Lymphedema Network (NLN)
  • National MPS Society
  • National Organization for Rare Disorders (NORD)
  • National PKU Alliance
  • National Spasmodic Dysphonia Association
  • National Tay-Sachs & Allied Diseases Association, Inc. (NTSAD)
  • NBIA Disorders Association
  • NephCure Kidney International
  • Neuroendocrine Tumor Research Foundation
  • Neurofibromatosis Network
  • The Oley Foundation
  • Organic Acidemia Association
  • Osteogenesis Imperfecta Foundation
  • Oxalosis and Hyperoxaluria Foundation
  • Parent Project Muscular Dystrophy (PPMD)
  • Parents and Researchers Interested in Smith-Magenis Syndrome (PRISMS)
  • PKD Foundation
  • Prader-Willi Syndrome Association (USA)
  • PRP Alliance
  • Pulmonary Hypertension Association
  • RASopathies Network USA
  • Rett Syndrome Research Trust
  • Scleroderma Foundation
  • Spastic Paraplegia Foundation
  • Sturge-Weber Foundation
  • Tarlov Cyst Disease Foundation
  • Tuberous Sclerosis Alliance
  • United Leukodystrophy Foundation
  • The United Mitochondrial Disease Foundation
  • US Hereditary Angioedema Association Vasculitis Foundation
  • VHL Alliance
  • Williams Syndrome Association
  • Wilson Disease Association
  • Worldwide Syringomyelia & Chiari Task Force
  • XLH Network

For additional information, contact:

Annie Kennedy, Senior Vice President Legislation & Public Policy, Parent Project Muscular Dystrophy (PPMD), annie@parentprojectmd.org, (443) 813-0998

Paul Melmeyer, Associate Director of Public Policy, National Organization for Rare Disorders (NORD), pmelmeyer@rarediseases.org, (202) 588-5700 ext. 104.

Kristin Stephenson, Vice President Policy & Advocacy, Muscular Dystrophy Association (MDA), kstephenson@mdausa.org, (202) 321-2959

CC: Members of the Senate Health, Education, Labor and Pensions (HELP) Committee 

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Comment by John Loehlein on November 16, 2015 at 5:52pm

will this legislation allow for the elimination of the use of placebos in clinical trials for MD drugs?

Given the fact that there is a huge amount of data already collected about the progression of DMD and its natural history this legislation should allow for use of that data as a standard that all clinical trials could use. The elimination of the placebo requirement would be a huge step forward in this effort to speed up and streamline research. 

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