Suggestions for promoting domestic clinical trials of medicinal products for rare diseases / 国际药学研究杂志

ABSTRACT

Regardless of rare or common diseases, it is a common desire to shorten the time period of drug devel-opment. This is of particular importance for rare diseases because the most of rare diseases are lack of sufficient therapeu-tic methods. The incentive policy for the development of rare disease drugs(also called orphan drugs)was first adopted by the United States in the Orphan Drug Act in 1983, and correspondingly, most of clinical trials of the orphan drugs are seen in the United States. The clinical trials of orphan drugs are also increasingly on the rise in China. The main target diseases of the major portion of orphan drugs in clinical trials are mainly the rare cancers, and most of them are in the phaseⅡtrials. The clinical trial is a particular step in the orphan drug development. Besides the need of policy support, it still suffers from severe challenges in the clinical trial design and the clinical trial implementation, including the insuffi-ciency in the understanding of natural history and mechanism of the diseases, the impossibility to conduct the random-ized controlled clinical trial, the requirement for innovated design and more sensitive outcome measures to evaluate effi-cacy, the difficulty in enrolling patients to participate in the trials, and the ethical conflict. The solutions to these prob-lems will require the joint efforts of all parties involving the government, pharmaceutical companies, medical institu-tions, and patient organizations.