Let the drugs no longer be “rare” ——RDPAC and CHARD jointly support innovation and availability of drugs for rare diseases


There are up to 7000 types of rare diseases with more than 250 million people who suffer from these diseases. However, only less than 10% of rare diseases have approved treatment drugs or regimens available, and almost none of these diseases can be cured. With over 20 million Chinese patients with rare diseases, the rare diseases in China are actually not “rare” due to the large population base. Although rare diseases are receiving more and more attention, patients with rare diseases are still facing the practical problems such as “inability to afford medication” and “lack of availability of drugs”.

In recent years, the Party and our country attach great importance to the management of rare diseases. National Health Commission (NHC), National Medical Products Administration (NMPA), National Healthcare Security Administration (NHSA) and other relevant departments have taken a series of measures to improve the diagnosis and treatment capacity of rare diseases, the review and approval of drugs, and the availability of drugs, so that the drugs for rare diseases are no longer “rare”.

In the “opinions of the CPC Central Committee and the State Council on deepening the reform of the healthcare security system” released in March this year, it was clearly proposed to “promote the development of multi-level healthcare security system” and “explore the security mechanism for rare disease drugs”. Against this background, R&D-Based Pharmaceutical Association Committee (RDPAC) and China Alliance of Rare Diseases (CHARD) have established a long-term partnership, and jointly initiated a research on Medical Insurance Access and Payment Mechanism and Impact on Basic Medical Insurance Fund of Rare Disease Drugs in China, and its Project Launch Meeting was held online recently. KANG Wei, Managing Director of RDPAC, LI Linkang, Executive Director of CHARD, and IQVIA Real-World Solutions Team, as well as over ten experts in the field of pharmacoeconomics and healthcare security attended the online meeting. Experts actively discussed the research program and offered suggestions on promoting the establishment of healthcare security system for rare diseases and accelerating the availability of innovative drugs for rare disease patients.

As a non-profit industry organization made up of 42 multinational pharmaceutical companies with leading R&D capabilities in the world, RDPAC and its member companies continue to pay attention to the availability of drugs for patients with rare diseases, and are committed to promoting the research, development and introduction of drugs for rare diseases. CHARD, gathering supports from multiple parties such as the government and the society, is aimed to improve the level of diagnosis and treatment for rare diseases and promote the collaborative innovation of clinical and scientific research and orphan drug development of rare diseases. 

To provide decision-making basis for the exploration of security mechanism for rare disease drugs

In the past two years, the introduction of favorable policies related to rare diseases has been accelerated. In 2018, the NHC, together with five ministries and commissions, published the First Batch of Rare Disease List, including 121 diseases, which provided an important reference for the research and development direction, clinical diagnosis and treatment, and medical insurance access of rare disease drugs. In addition, medical products administrations have also issued a series of policies to accelerate the review and approval of rare diseases drugs, especially in two lists of 66 drugs urgently needed in clinical settings issued by Center for Drug Evaluation of NMPA, 38 of which were drugs involved in rare diseases therapy. According to incomplete statistics, with reference to 121 diseases included in the First Batch of Rare Disease List, 162 drugs have been approved in the United States, the European Union or Japan. Of the 61 drugs marketed in China, 36 drugs have been included in the National Reimbursement Drug List (NRDL).

With more and more rare disease drugs marketed in China, what pressure will the medical insurance fund face? How to balance the welfare of patients with rare diseases and the treatment needs of other patients? This study, carried out around the above problems, will estimate the impact of medical insurance fund by inclusion of rare disease drugs in the NRDL, and analyze experiences of domestic and international medical insurance access and payment systems for rare disease drugs, which may provide a basis for decision-making for the relevant departments to formulate policies of healthcare security system for rare disease drugs.

LI Linkang, Executive Director of CHARD, emphasized the importance of the availability and affordability of rare diseases drugs for the treatment in patients with rare diseases. He said: “the NHSA has been trying to solve the problem of healthcare security for patients with rare diseases. Through the implementation of a series of policies, the rare disease patients in China really have had a sense of gain, happiness and security. Since 2018, the two adjustments of NRDL have given policy preference to the inclusion of rare disease drugs, which made some patients with rare disease no longer give up treatment due to inability to afford, improved patients’ quality of life, and brought them hope for survival.”

To support innovation of drugs for rare diseases to benefit more patients as soon as possible

Under the guidance of the concept of “healthier china through innovation”, RDPAC and its member companies have been committed to the R&D and introduction of innovative drugs, so as to let Chinese patients benefit from the world-class achievements of drug innovation. Taking “the First Batch of Rare Disease List” as an example, about 60% of the 61 rare diseases drugs that have been marketed in China are from RDPAC member companies.

The research on Medical Insurance Access and Payment Mechanism and Impact on Basic Medical Insurance Fund of Rare Disease Drugs in China will combine the innovation advantages of RDPAC member companies, the platform advantages of CHARD and the data analysis advantages of IQVIA. KANG Wei, Managing Director of RDPAC, said that “this research co-sponsored by RDPAC, CHARD and IQVIA, as well as various experts has not only practical significance for improving the status of drugs available to patients with rare diseases and saving patients’ lives in China, but also great significance for the development of healthcare system and the pharmaceutical industry of rare diseases.”

The experts also agreed that in order to enable patients with rare diseases in China to access innovative drugs in time, in addition to the long-term attention and efforts of all sectors of the society, it is necessary to focus on building short- and medium-term and medium- and long-term policy mechanism of accelerating approval process for innovative drugs, the diagnosis of rare diseases and the healthcare security system for medications of patients with rare diseases. For example, we should further improve the evaluation criteria and mechanism of rare diseases list and ensure that it is updated in a timely manner, and put forward the evaluation process and standards applicable to rare diseases drugs in the process of NRDL evaluation.

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