The expanded role of rare disease patients is crucial for drug development according to the authors of a commentary published in Nature Reviews – Drug Discovery. The authors elucidate how patient engagement can enhance the drug development at each stage (Phase). According to them, in the early stages of drug development, information on the burden of disease as well as the progression of disease symptoms is essential to assess the benefit-risk profiles. This information, according to them, can be most accurately provided by the rare disease patients themselves. Constructing a body of knowledge in this manner to create patient-reported outcomes or clinician-reported outcomes is substantially important later in the drug development process.
The authors believe that early patient engagement is also essential for establishing appropriate end-points and recruiting suitable patients. At later stages it is important to estimate from patients the reasonable goals of the therapy relative to the risks to determine the safety and efficacy of the drugs.
Finally, the authors recommend standardization of “use and review of information collected from patients in regulatory review.”
OrphaNews September 17, 2016