Moving with increasing urgency to encourage the development of drugs to treat rare diseases, Chinese regulators are planning for the first time to authorize real-world studies of orphan drugs.
Technical guidelines released in October by the National Medical Products Administration’s Center for Drug Evaluation set parameters for the studies. The guidelines also encourage the use of reports filed directly by patients on the efficacy of the drugs, calling such Patient-Reported Outcomes powerful tools to inform clinicians, policymakers, and pharmaceutical developers.
The technical guidelines also lay out specific steps for the clinical development of orphan drugs that are distinct from the research and development of other pharmaceuticals. Regulators say the rules for orphan drug development take into account the complex nature of rare diseases, and the obstacles to diagnosing them.
China has been prioritizing the development of treatments for rare diseases for the past four years, publishing its first national list of the conditions in 2018 and working steadily since then to increase the availability of treatments to Chinese patients.