Comparison of research support for rare diseases and orphan drugs innovation in China and the United States / 中国药学杂志

OBJECTIVE: To introduce and compare research support for rare disease and orphan drug innovation in China and United States, and provide reference for relevant policies in China. METHODS: Data of main source of funding for rare disease research in two countries ie. National Institutes of Health, Food and Drug Administration in the United States and National Natural Science Foundation of China were analyzed and compared. RESULTS: US NIH gives substantial support for rare disease research every year with funded capital growing. FDA Orphan Products Grants program provides incentives for sponsors to develop products for rare diseases. In China, however, there is no specific support project for rare disease research, and there is a huge gap in funding efforts for rare disease research between China and the United States. CONCLUSION: China should establish rare disease research center to promote rare disease research and set up specific funding for rare diseases research, increase efforts to support research and innovation for rare diseases and orphan drugs, in order to protect the health interests of patients with rare diseases.

International reimbursement policies on drugs for pulmonary arterial hypertension and enlightenment to China / 中国药学杂志

OBJECTIVE: To introduce international reimbursement policies on drugs for pulmonary arterial hypertension (PAH), and provide a reference for establishing relevant policies in China. METHODS: Reimbursement policies on PAH drugs of Australia, Canada, America and some EU countries were analyzed and compared with the status quo in China. RESULTS: Although expensive, PAH drugs are reimbursed by medical insurance in most countries included in this study. However, the accessibility of PAH drugs in China remains very poor with only a few cities including these drugs in the medical insurance. Patients with PAH face with poverty caused by disease, even abandoning treatment. CONCLUSION: Learning the experience from countries with rare disease act and good medical insurance system and analysing the earliest attempts to reimburse PAH drugs in China will help promote the reimbursement policy on PAH drugs in China.