Rare disease is an issue in multiple perspectives:
Rare disease is an issue of people’s livelihood. There is an urgency for solution in the background of China’s “Completing the Building of a Moderately Prosperous Society” and “Win the battle against poverty” momentum. In the context of the inaccessibility and unaffordability of drugs especially the premium drugs, it is still a common phenomenon that patients become disabled or poor again due to the diseases. Urgent actions should be made to precisely support the rare disease patients and avoid their “poverty again” issue, and to let these people share the result of China’s economic miracle. Under the background of the high attention paid by the CPC Central Committee and the State Council to people’s livelihood and fairness, rare diseases to an extent also reflect the room that still exists in enhancing the operation efficiency of multipartite supporting mechanisms of the society and the intensity of promotion also embodies the social value orientation and process of civilization.
Rare disease is an issue of healthcare industry development. Research and development in rare disease is a key for China healthcare industry to incubate global leading R&D capability and participate in the high end of global biopharmaceutical value chain. Echoing the momentum “making China a country of innovators” from the 19th National Congress of the CPC, China biopharmaceutical industry aims to bring more true innovation and breakthroughs. In the era of precision medicine, rare disease is an epitome of human diseases and a key breakthrough for deciphering the mystery of human health, and an important lead for promoting the development of the human researches on medical science. On the one hand, rare disease is a global challenge with huge unmet medical needs. There are more than 7,000 known rare diseases around the globe, with only less than 10% of them having treatment drugs or other methods that have been approved, leaving sufficient space for discovery and innovation for China biopharmaceutical industry. With blooming genetic diagnostics and gene therapies, development of curative therapeutics for rare disease is an opportunity for China biopharmaceutical industry to take global leadership in innovation. Compared to the fast-follower driven development model in oncology and common diseases, China biopharmaceutical industry can benefit from its effort on rare disease. On the other hand, rare disease is often a cradle for true innovator drugs that applied to a whole “disease group”, hence a cradle for drug discovery and innovative biopharmaceutical companies. Every disease may be a rare disease when it is first discovered. Behind every common disease, there stands a queue of invisible rare diseases; behind every rare disease, there also stands a queue of invisible common diseases. A focused research on the pathology, mechanism of action and the metabolic pathway for a rare disease empowers the drug discovery in the corresponding disease group. Numerous blockbuster drugs were initiated as rare disease treatment, like most of the top 10 best-seller drugs globally gained orphan drug designation in their earliest indications. Data from IQVIA Institute demonstrated >50% newly approved drugs in US and EU in the past 5 years are orphan drugs, and most of the innovator drugs in the near future are orphan drugs on rare targets from emerging biopharmas. Finally, world largest rare disease patient population in China builds a foundation for scientific and clinical research globally, and for new solutions for worldwide patients. China’s boosting development in life sciences, genetic diagnosis and biopharmaceutical development has established a solid base for the generation of true innovators.
Rare disease is an issue of public governance. It is a problem that the regulators have to think about: how to guarantee the equal health rights of citizens, promote the focus of medical researches on the group of rare diseases, overcome the payment difficulty of public health, ensure the accessibility and affordability of treatment drugs in the context of the high prices of treatment drugs and balance the sustainable development of assurance funds.
Rare disease is in the community of shared future for mankind and closely related to everyone. Every human individual is a potential rare disease patient. In the more than 7,000 rare diseases already known around the world, 80% are genetic diseases caused by gene defects. The inheritance of life is fragile: In the genes of every person, defects exist in 7 ~ 10 groups of genes on average. So long as life is multiplying, it is possible for rare diseases to occur and everyone may be a patient or carrier of a rare disease. From a certain perspective, rare disease patients bear the disease risks of the whole mankind.
Resource: <China Rare Disease Drug Accessibility Report 2019>