Orphan drugs, by nature, remain commercially underdeveloped, and as demand in the market increases, patients in clinical trials need to be considered.
The rapid growth in the market for rare disease treatments has placed substantial burdens on patients, and the expansion of drug development to meet the rare disease market has increased pressure on patients and in the drug regulatory process.
Drug regulatory agencies now make substantial efforts to include patient perspectives and involve patients in the drug review process. This is based on a recognition that, especially in life threatening and rare diseases, there is often an additional perspective that the patient provides on the disease that is not captured by the medical environment. Patients generally participate in the regulatory process as representatives of specific rare disease advocacy organisations providing insight on what their organisation considers are key points about the disease to account for in drug approval, or as an individual patient expert in their therapy area, giving insight on what they, as consumers, feel is most important in the specific treatment for their disease.
Study sponsors need to consider the patient perspective in the study design from the earliest stage of study concept and protocol development. This can be done using information from medical literature, qualitative patient interviews, patient questionnaires, and patient workshops.
Rare disease patients are most likely to find out about a trial from their treating physician, and typically, rare disease physicians will be in a practice specialising in rare disease treatments and have multiple trial opportunities. Study sponsors need to ensure that physicians at enrolling sites are able to describe the study value to patients and communicate how the trial process has been designed to ensure minimal patient discomfort and lifestyle disruption. Participation by patients requires study sponsors to arrange for travel and lodging, and provide other support services based on a comprehensive assessment of that patient and their family’s individual needs and circumstances.
ICON’s experts can help you navigate the clinical development challenges in rare and orphan diseases. To speak with one of our experts, please contact us.
This blog is an edited version of a media article written by ICON experts William Maier and Kelly Franchetti, “Patient voices in the industry”, which is published in the October 2019 edition of European Biopharmaceutical Review, pages 28-32. © Samedan Ltd.Download Media Article
Rare and orphan diseases insights
ICON's Rare and Orphan Diseases team provides analysis including whitepapers, blogs and contributions to media and industry conversations relating to all aspects of rare and orphan diseases in clinical trials.Read more