On February 25, 2022, as the 2023 Rare Diseases Day approached, the online release conference of the "2023 China Rare Diseases Industry Trend Observation Report" (hereinafter referred to as the "Report") jointly organized by Frost & Sullivan (abbreviated as "Frost & Sullivan") and the Beijing Pain Challenge Public Welfare Foundation (abbreviated as "Pain Challenge Foundation") was successfully held.
The "2023 China Rare Diseases Industry Trend Observation Report" is the second annual trend observation report following last year's joint release by Frost & Sullivan and the Pain Challenge Foundation. The Report aims to review and sort out the development of rare diseases in China in terms of diagnosis, medication, coverage, special medical foods, and innovative services, outline the problems faced and progress made in this field over the past, and propose prospects for China's comprehensive rare disease service system, hoping to be beneficial to policymakers, advocates, practitioners from relevant institutions, patients with rare diseases, and the general public who care about rare diseases.
China's modernization cannot be separated from human health, which also involves the rights and interests of patients with rare diseases, including medication and coverage.Li Linkang, Executive Chairman of the China Rare Diseases Alliance, delivered an opening speech, calling on everyone to respect every rare life, work together to promote the prevention and treatment of rare diseases in China, and light up the light of life for patients with rare diseases. Mao Hua, Partner and Managing Director of Frost & Sullivan Greater China, attended the release conference and shared insights on the current development status and future trends of the rare disease industry.
The accessibility of rare disease drugs has improved,70% of therapeutic drugs are included in medical insurance
At the release conference, Guo Jinchuan, Information Research Director of the Pain Challenge Foundation, interpreted the Report from a policy perspective. As of now, based on the "First Batch of Rare Diseases Catalog," 103 drugs have been launched in China, involving 47 rare diseases; among them, 73 drugs have been included in medical insurance, covering 31 rare diseases.However, the coverage of high-value drugs outside the catalog remains a core pain point for patients' medication accessibility,Currently, all treatment drugs for 16 rare diseases are not included in medical insurance, and 13 of these drugs are high-value drugs, with annual treatment costs ranging from hundreds of thousands to millions.
From the national level to local areas, various parties have made extensive explorations to solve the problem of medication accessibility for rare diseases, such as dynamic adjustments to the medical insurance catalog, new exploration of local rare disease medication coverage mechanisms, and the participation of public welfare and charitable forces in multi-level coverage for rare diseases.
Rare diseases have become a popular investment track globally, with multiple favorable policies introduced domestically
Mao Hua, Partner and Managing Director of Frost & Sullivan Greater China, stated that currently, patients with rare diseases in China face many difficulties in diagnosis, treatment, payment, and coverage. To solve the urgent medication problem of the rare disease patient group, the Chinese government has introduced six policy measures to encourage the research, development, review, and approval of rare disease drugs: encouraging the research and development of rare disease drugs, prioritizing review and approval, exempting clinical trials, providing tax incentives, and including temporary import and market exclusivity periods.
Mao Hua pointed out that benefiting from favorable policies, China has made certain progress in covering rare disease drug varieties, and domestic pharmaceutical companies are gradually participating in the introduction, research, and imitation of rare disease drugs.Based on the "First Batch of Rare Diseases Catalog," 81 rare disease drugs in China are currently in clinical trials or application stages for marketing, among which 46 drugs are independently developed locally.
On the other hand, the global rare disease field has become a popular investment track, with multinational pharmaceutical companies actively deploying through mergers and acquisitions or research and development cooperation. Data shows that among global pharmaceutical merger and acquisition projects in 2022, rare diseases were the field with the highest capital investment.
In addition, breakthroughs in key technologies have brought new hope to rare disease treatment.Small nucleic acid (SNP) drugs, due to their dual characteristics of gene modification and traditional drugs, have broad application prospects in the field of rare diseases. Currently, there are heavyweight SNP drugs with annual sales exceeding $1 billion in the rare disease field, and more than half of the globally developed SNP drugs are orphan drugs. At the same time, gene therapy also brings hope to patients with rare diseases, with the prices of gene therapies for rare diseases currently on the market often exceeding millions of dollars. China also has many gene therapies for rare diseases under development, but their high prices pose challenges to China's rare disease medication coverage system.
Mao Hua stated that in the future, Frost & Sullivan will continue to follow up on the latest industry trends, pay attention to the field of rare diseases, and continuously release blue books on biomedicine and healthcare industries to help the industry develop healthily and rapidly.
Multiple parties gather to discuss rare disease policies and new directions for the industry
Representatives from policy coverage, industry, and patient organizations gathered together to discuss the direction of rare disease policies and the industry in 2023 based on the Report. The attending guests all emphasized the importance of top-level policy design and took the example of Jiangsu's legislation to determine special fund coverage forms to explore the path of establishing multi-level coverage, hoping to participate in ensuring the last hundred meters of rare disease payment issues through multiple parties to avoid patients with rare diseases missing out on the dividends brought by policies. Industry-related parties also introduced the characteristics of high investment, long cycle, and low short-term returns in rare disease drug research and development, looking forward to more incentive policies.
Finally, everyone expressed their expectations for the Report, hoping to see new changes in the report every year, continuously and intuitively demonstrating the country's attention to rare diseases, benefiting patients with rare diseases, and playing a positive guiding role in the industry.
Frost & Sullivan sincerely looks forward to working with all sectors to pay attention to and jointly ensure the colorful life of the rare disease group; we will also continue to be committed to research in the field of rare diseases, actively fulfill our social responsibilities, and look forward to joining hands with all parties to create a better tomorrow for patients with rare diseases, continuously contributing to the construction of "Healthy China 2030"!
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About Frost & Sullivan's Healthcare Business Unit
The healthcare business unit of Frost & Sullivan has professional analytical capabilities and rich project experience in the field of life sciences. Relying on Frost & Sullivan's global think tank resources and the cross-industry business development platform in Greater China, Frost & Sullivan Healthcare has unique core advantages in healthcare industry investment and financing services. Frost & Sullivan Healthcare has extensive corporate clients in China and has established a large customer network over the past 20 years, accumulating a wealth of project experience in various healthcare sub-sectors.
Project types include knowledge center projects (in-depth content, promotional activities), Pre-IPO projects (DCF valuation, business plan services), IPO listing projects (industry advisor, clinical audit, fundraising writing), market research, market value management, and strategic consulting, etc., and cooperate with well-known domestic and international information platforms and investment and financing institutions to provide one-stop solutions for professional sub-sectors such as pharmaceuticals and medical devices for enterprises, receiving wide attention from investors.
