Principal Investigator (PI): Ying BIAN, Associate Professor
Year: 2024
“The difficulty in evaluating orphan drugs lies in the difficulty of conducting routine randomized controlled double-blind clinical trials. First, there is no standard reference treatment method or lack of appropriate reference medicines; second, there is only evidence from a small number of clinical cases; third, due to uncertainty in the evidence, it is difficult to evaluate their additional benefits, especially the long-term effects; fourth, predicting the price of new orphans is difficult, and therefore pricing orphan drugs is also difficult.”
Regulatory Science Challenge
Drugs for rare diseases once received less attention from pharmaceutical companies due to large investment, difficult research and development (R&D), and low demand, so they are called “orphan drugs” (OD). Countries and regions such as the United States and the European Union have enacted special legislation to incentivize pharmaceuticals for the R&D and production of OD. The most powerful measure is market exclusivity. However, market exclusivity is prone to abuse in practice, which leads to a series of problems such as ultra-high pricing of OD, repeated acquisition of market exclusivity, and staggering profits for pharmaceutical companies. The OD industry in China is still in its infancy, there is a need in introducing the market exclusivity measure in legislation to incentivize pharmaceuticals. In the meantime, the intervention mechanism for OD pricing must be established, OD identification standards should be reasonably set, market exclusivity grant conditions must be strictly enforced, exceptions for market exclusivity should be set up, and timely adjustments to the protection period of market exclusivity should be allowed based on practical changes in market conditions. There are two major ways to solve the problems in treatment of rare disease patients. First is to encourage pharmaceuticals to invest in the R&D and production of drugs for rare diseases, increase the supply of rare disease drugs through policy and legal support in national level. Second is to integrate rare diseases into the social security system and improve the accessibility of drugs for rare disease patients.
Project Description
This study intends to review the decision-making principles of international OD pricing policies and their impact on the international pharmaceutical market as basis, to analyze how China’s OD pricing policy-making principles will be responded to future changes in China’s medical service market due to the huge population and serious population ageing in China, to analyze the gap between supply and demand in the OD market based on the real data from the centralized procurement of drugs and to put forward corresponding policy recommendations.
This study is a combination of quantitative and qualitative research. Based on the analysis of policy influencing factors, it analyzes the gap between supply and demand in the OD market according to the actual data from the centralized procurement of drugs, and conducts model research to try to find out the maximum value of the influencing factors.
