What is a “Rare Disease” and How We are Addressing an Unmet Need

Posted by: DaVita Clinical Research

There are over 7,000 rare diseases that impact an estimated 30 million people in the United States.   Many people with these rate conditions often feel that the pharmaceutical and medical industry does not focus enough on these conditions because there is such a focus on more common conditions that impact people in larger numbers.   In fact, the issue has been such a concern that the FDA has implemented special incentives for drug companies so that they continue to focus on these rare but important conditions that affect so many.

A rare disease, by definition, is a conditions that impacts less than 200,000 people in the United States.   These smaller populations can make the conduct of clinical trials more difficult due to the overall complexity of enrolling subjects.  The Orphan Drug Act is a law passed by Congress in 1983 that incentivizes the development of drugs to treat rare diseases. Companies and other drug developers can request orphan drug designation and the FDA will review and grant this designation if the drug meets specific criteria.  Orphan designation qualifies sponsors for various incentives, including:

  • Tax credits for qualified clinical (in humans) testing
  • Waiver of the Prescription Drug User Fee (currently at $3 million for a new drug)
  • Potential 7 years of market exclusivity after approval

An “orphan drug” is a drug that may have been abandoned by a pharmaceutical company due to a lack of incentive to conduct all of the research involved with approval.   The incentives listed above may change the minds of drug manufactures and can have a significant impact on development plans.   We are, in fact, seeing a number of drugs come to the forefront and enter into the research space that may have otherwise been abandoned.

To facilitate the potential of these therapies, DaVita Clinical Research recently launched a new initiative called the CKD and Rare Disease Network to meet the needs of the pharmaceutical sponsors.   In this model, DaVita will look through the data that we have to find where patients with rare diseases reside.   We will then work with the local physician for these patients to see if they are interested and able to conduct these clinical trials locally.   Once the relationship is established, the physician will have the opportunity for their patients that may qualify to participate in a clinical trial for their condition.

While the research and treatment of rare diseases will remain a challenge, DaVita and other organizations are continuing to develop new models of research to bring these vital medications to the market.   We encourage physicians and patients to consider participation in clinical trials for rare disease as this is the only way these new therapies will be developed.

Visit the following websites for more information:

For Patients- MyKidneyResearch.com

For Physicians and Pharma- DavitaClinicalResearch.com