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Mar 10 2011

Therapeutics for Rare and Neglected Diseases – How Rare is Rare?

Logo of the Office of Rare Diseases, part of t...

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The U.S. National Institutes of Health (NIH) classifies a rare disease as affecting less than 200,000 individuals in the United States.  As insignificant as this classification may sound, collectively there are more than 25 million people in the United States affected by approximately 6,800 rare diseases.

These rare diseases are alarmingly overlooked and under researched. Less than 300 of the 6,800 rare diseases have active industry research programs devoted to finding new therapeutics.  Recently, NIH has embarked on a renewed effort to find treatments for these rare diseases, as well as for diseases considered to be neglected.  Neglected diseases have limited or no treatment options and frequently effect disenfranchised populations in developing nations (e.g., tropical parasitic diseases). To undertake this effort, NIH  launched a program, Therapeutics for Rare and Neglected Diseases (TRND), in 2009 dedicated solely to the development of new treatments for rare and neglected disorders.  TRND is overseen by the Office of Rare Diseases Research (ORDR).

Amazingly, TRND is the first big movement for rare diseases since the Orphan Drug Act of 1983.  That Act provides incentives such as tax breaks and marketing exclusivity for the development of treatments for rare conditions. Since being passed, the FDA has approved more than 300 treatments under direction from the Act.  Although this has raised awareness and increased effort to study rare diseases, the causes behind and effective treatments for the vast majority of these diseases remain completely unknown.

The TRND program is designed to boost the drug pipeline within NIH using its unique structure of partnering with biopharmaceutical companies, academic scientists, and non-profit organizations. The program is not a grant application, but an application to collaborate with experts and gain access to resources. Below are a few resources that the TRND program provides (based on stage and needs):

  • Biomarker development
  • Production of dosage forms
  • IND filing advice
  • Development of analytical methods for bulk substances
  • Planning of clinical trials
  • IND-directed toxicology, with correlative pharmacology and histopathology
  • Development of pharmacology assays

TRND is focused on bringing early-stage products to an IND filing, in particular through work on pharmaceutical development and nonclinical pharmacology and toxicology.  There are five pilot projects underway in schistosomiasis/hookworm, Neimann-Pick Type C, hereditary inclusion body myopathy, sickle cell disease, and chronic lymphocytic leukemia.

An application process is used by TRND to select the best suited collaborators. TRND evaluates applications for the program based on the following criteria:

  1. Target and therapeutic validation (30%)
  2. Strength of current data package (30%)
  3. Feasibility to reach First in Human (20%)
  4. Medical impact relative to current Standard of Care (10%)
  5. Likelihood of external adaption (10%)

Attend a TRND “Road Show” session to learn about progress of the TRND program; how collaborations operate; and how to submit an application to work with the TRND program. The Road Show is visiting Philadelphia, Boston, Irvine, San Diego, and New York March 15th to 24th.

This is a post by Nicole Corrado.  Nicole is a Project Coordinator in Cato Research‘s Washington, DC office.