Developing an Innovative Multi-Layered Security System for Rare Diseases Drugs and Establishing a Drug Value Assessment Mechanism


On December 18 and 19, 2021, China Alliance of Rare Diseases convened 2021 China Conference on Rare Diseases. At the conference, all participants had an in-depth exchange of views on themes relating to rare diseases, including medical care service, healthcare security and drug R&D policies, development and regulation of rare diseases drugs, multi-disciplinary treatment, and fostering a rare disease ecosystem. Ms. Kang Wei, Managing Director of the R&D-based Pharmaceutical Association Committee (RDPAC), was present at the conference upon invitation. She shared her thinking on rare diseases security policy and drug value assessment at the Healthcare Security Policy Sub-Forum on Rare Diseases co-hosted by RDPAC and China Alliance of Rare Diseases. Leaders from National Health Commission (NHC), China Alliance of Rare Diseases, and Beijing Rare Disease Treatment and Protection Association and experts in fields of healthcare security administration, pharmacoeconomics, pharmacy and clinical medicine attended the Sub-Forum.


 According to statistics, there are now as many as 7,000 rare diseases worldwide, and the total number of Chinese patients with rare diseases has reached 20 million. In recent years, multiple measures have been taken to encourage R&D for rare diseases drugs, accelerate review and approval of marketing application for rare diseases drugs, enhance rare disease diagnosis and treatment capacity, and massive include rare diseases drugs into the National Reimbursement Drug List (NRDL). Thanks to these efforts, the accessibility of diagnosis and treatment services to patients suffering from rare diseases has been significantly improved. However, many rare disease patients are still facing difficulties in diagnosis, fewer therapeutic drugs and inadequate security. More support is needed in policies to improve security coverage for patients with rare diseases under the multi-layered healthcare security system and to establish an appropriate mechanism for value assessment of innovative drugs for rare diseases to enhance drug accessibility.

Calling for Improvement of the Multi-Layered Security System to Enhance Patient Access to Rare Diseases Drugs

Since 2018, China has made remarkable progress in security for rare disease patients. The First Batch of Rare Diseases List issued and published by NHC, which is inclusive of 121 rare diseases, has played an important role in the R&D, introduction and improvement of security policies for rare diseases drugs in China. In addition, the National Healthcare Security Administration (NHSA) also gave priority to rare diseases when making adjustment to the NRDL. According to statistics, 52 out of all marketed drugs for rare diseases in China have been included in the NRDL, including the two high-value orphan drugs that were included via NRDL negotiation in 2021.

However, the security for some high-value drugs for rare diseases has not yet been sufficient, a multi-layered security system for patients with rare diseases has not yet been fully established, and the drug accessibility for rare diseases still needs to be improved. At the Sub-Forum, industry experts and government officials elaborated on pricing mechanism for rare diseases drugs, healthcare security for rare disease patients, multi-layered security mechanism and relevant local exploration, and discussed on how to break through security issues for high-value drugs for rare diseases.

Currently, one of the major challenges facing security for patients with rare diseases is the lack of a clear distinction regarding the primary and secondary roles played by each party within the framework of multi-layered healthcare security. The experts believed that basic medical insurance should still play a leading role in the multi-layered security mechanism for patients with rare diseases, and besides, a government-led, multi-payment security mechanism for high-value drugs for rare disease should also be established. For the years to come, the key focuses regarding security for patients with rare diseases will be put on coordinating the exploration of security policies for patients with rare disease across provinces and applying the exploration outcome to the entire nation, as well as developing innovative security policies and mechanism for patients with rare diseases under the leadership of the NHSA.

Ms. Kang Wei said, "there are two key methods to improve the multi-layered security system for patients with rare diseases. on the one hand, more drugs for rare diseases should be included into the NRDL, together with lowering the reimbursement threshold and increasing the reimbursement ratio; on the other hand, rare diseases drugs that pose major medical burden should be included into the scope of assistance to enhance security level by establishing a fund specific to high-value drugs for rare diseases and accelerating the establishment of the 'security and assistance mechanism for patients with serious and critical illnesses'. In addition, supplementation through commercial health insurance may also be investigated."

The Conference on Rare Diseases also witnessed the official launch of the Rare Disease Assistance Fund jointly initiated by China Alliance of Rare Diseases and Chinese Red Cross Foundation (CRCF). The Fund aims to seek a new path for the security for rare diseases in China, bring a promising future to Chinese patients with rare diseases, and propose an updated multi-layered security mechanism for patients with rare diseases.

Recommending on Establishment of a Value Assessment Mechanism for Innovative Drugs for Rare Diseases

Due to the high R&D costs, rarity of patients, high risk of investments, and high costs of manufacturing, rare diseases drugs are often priced high and it is impossible to obtain substantial clinical trial data. In this sense, the commonly used pharmacoeconomic evaluation methods and standard thresholds are not applicable to researches on orphan drugs. On account of this, many countries around the world have introduced special policies for marketing authorization assessment of orphan drugs, with some countries raising their thresholds for ICER and others directly exempting health technology assessment (HTA) of rare diseases drugs or assessing only the impact on their medical insurance funds. In contrast, there are currently no special health economic evaluation methods and models for rare disease drugs in China, nor are there special mechanisms and methods for rare diseases drugs value assessment in the adjustment of NRDL.

At the conference, the participants shared their viewpoints on orphan drug policies worldwide, pharmacoeconomic evaluation, and value assessment of drugs for rare diseases. They also conducted an in-depth discussion on considerations in NRDL negotiation on rare diseases drugs. It is argued by the experts that during the value assessment of rare diseases drugs, comprehensive multidimensional considerations should be given to the clinical and social values of rare diseases drugs, such as assigning a higher weight to the QALY gained and setting a higher threshold for ICER when rare diseases drugs are concerned.

Ms. Kang Wei said, “The level of security for rare disease patients is closely related to local social and economic development. In consideration of this, improved social and humanistic care in tune with economic growth should be available. Specifically, when a country's GDP per capita exceeds 10,000 dollars, it should consider introducing some special security policies for the treatment of rare diseases. Based on a meta-analysis of previous studies conducted by RDPAC member companies, we found that it would take ten to twenty-seven years to develop a new drug for rare diseases. This process is not only time-consuming but requires huge capital investments. With effective policy support and incentives in place, the motivation for R&D and manufacturing of drugs for rare diseases would be greatly inspired. In addition to optimizing the medical insurance reimbursement system and accelerating review and approval procedures, other initiatives such as enhancing doctors' and patients' knowledge of rare disease diagnosis and treatment and strengthening patent protection for innovative drugs for rare diseases are also crucial to improving the accessibility of innovative drugs for rare diseases. To achieve this, joint efforts from all parties concerned is indispensable. Therefore, based on China's current pace of economic development, we expect that the Chinese government could introduce more incentive policies that are conducive to drug development and marketing authorization for rare diseases, and explore special security policies to benefit more rare disease patients.”

As the conference came to an end, the participants expressed their expectations regarding security for patients with rare diseases in China, hoping that the Chinese government could pay more attention and care to the group of rare disease patients. The whole community should work together to promote the development of security policies on treatment of rare diseases and formulate a Chinese security scheme for patients with rare diseases, so that rare disease patients can have access to medical care services, drugs and healthcare security.

Related articles: