The Challenges of Drug Development for Rare Diseases

Challenges Development

Understanding the Challenges of Drug Development for Rare Diseases

Drug development for rare diseases can present a number of unique challenges. As the name implies, rare diseases, which the National Institutes of Health classifies as those that affect fewer than 200,000 people in the United States, can leave affected individuals without a cure or treatment options and without the financial support or recognition by larger drug developers.

Unique Challenges Presented by Rare Diseases

  • Financial Costs: Drug development is incredibly costly and the market for rare diseases may not be financially attractive to many of the large pharmaceutical companies that are capable of funding such projects.
  • Difficulty with Studies: Even if a drug is produced, finding enough participants to take part in clinical studies can be difficult subject to the number of individuals living with a particular rare disease in any given community.
  • The Orphan Drug Act: The FDA’s 1983 act created incentives for rare drug development in the US, but still more funding and support are needed in order to encourage the growth of treatments and cures for rare diseases.

The Negative Impact on Health

The lack of solutions to treat rare diseases affects the overall health of society. For those living with a rare disease, lack of treatment options can have a significant impact on their quality of life and for those who rely on medical advances for treatment, a lack of solutions can be life threatening. The development of drugs for rare diseases can also lead to breakthroughs in treatments for more common diseases and illnesses, so it is important to continue to find solutions to these unique issues.

Measures Taken to Support Drug Development

The National Institutes of Health (NIH), National Institutes of Health Rare Diseases Clinical Research Network (RDCRN), and the Orphan Drug Act are all dedicated to finding treatments for rare diseases and helping those affected by them. In addition, many nonprofits have been created to bring awareness to specific rare diseases, provide support to patients, and assist in drug development.


The challenges of drug development for rare diseases are many but so too are the efforts of those who are striving to find treatments. From professional organizations and U.S. government initiatives to non-profits and support groups, communities around the world are dedicated to improving the lives of those affected by rare diseases and helping to find cures for those who suffer.

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