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The Rarest of Them All: Rare Diseases

The Rarest of Them All: Rare Diseases

The voice of the patient is critical in healthcare. Nowhere is this more true than in the area of rare diseases.”Myshko, PharmaVoice. 2014 Feb; 26

When a person is in rare form, that person is at his or her best. When a disease is a rare disease, its treatment is at its best when patient voices are heard. A disease is considered rare in the United States if it affects fewer than 200,000 people in the country at any given time, according to Rare Disease Day. There are more than 6,000 rare diseases, and half of them affect children.

Patient voices and patient advocacy groups are important in the development of orphan drugs, drugs for rare diseases. The importance of patient voice and patient advocacy groups can be seen through patient recruitment and trial design.

Patient voices and patient advocacy groups raise awareness and are a necessity for patient recruitment in clinical trials. This helps patients receive not-yet-on-the-market medication and enables pharmaceutical companies to meet their patient requirement need for such trials. This is especially important with rare diseases because there is a smaller pool of eligible patients to recruit. According to Premier Research, 60% of companies developing orphan drugs used patient advocacy groups to assist with recruitment, and 42% described the patient advocacy groups as “successful” or “vital.”

Before patient enrollment can begin, the clinical trials must be designed. The natural history of a disease influences such designs. Because the natural history of most rare diseases is not well known, patient voices are critical in filling that void. The FDA recently announced support for a new rare disease database in which patients enter information about their experiences with their disease. This can teach industry leaders which aspects of the disease require treatment, how to identify patient subpopulations and which variables, biological or environmental, correlate with different stages of the disease. All of these factor into better clinical trial designs for orphan drugs.

Patient recruitment and trial design are areas that prove the necessity of including patient voices and patient advocacy groups in the development of orphan drugs and other treatment for rare diseases. With such inclusion, the market for orphan drugs will continue to grow. The quality of life for people living with rare diseases will improve; with that, both the industry and the patients will be in rare form.

By | 2018-11-11T04:42:33+00:00 November 21st, 2014|Blog|0 Comments

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