Rare Disease Care Across Asia Pacific

Rare Disease Care Across Asia Pacific

February 2023

“Rare diseases” are conditions that affect relatively few people compared to the general population, making them a complex challenge for national healthcare systems, which must determine how to allocate resources to suitably tackle the problem. Though approximately 8,000 rare diseases have been identified, merely an estimated five percent currently have treatments. Furthermore, perhaps 80 percent or more of rare diseases have been identified as genetic in origin, which provides insight into the biological drivers of many conditions. As hereditary conditions can be more common in some genetic populations than others, it is critical for nations to create rare disease strategies targeted to local conditions.

The Role of Governments in Rare Disease Support

Given the expense of treatment and potentially life-threatening health consequences of rare diseases, there is a serious need for state-level support for these challenging conditions. Definitions of what constitutes a rare disease vary by jurisdiction, with typical criteria including the total number of patients with the disease, its prevalence in the population, and the availability or scarcity of treatments. These national definitions are critical, as they often determine the level of state support for the R&D and production of medicines and treatments that would otherwise be unprofitable given the relatively small groups of affected patients.

Across the Asia Pacific region, rare diseases were estimated to affect around 258 million people in 2020, around half of which are children. At the same time, many lingering issues in national healthcare systems continue to hinder the treatment of rare diseases, including healthcare practitioner (HCP) knowledge. In 2020, 34 percent of regional HCPs did not know if their market had a unified definition of rare diseases and 35 percent did not know if their jurisdiction had a rare disease policy. A dearth of available treatment options and limited funding are also perennial problems across much of Asia Pacific. Some recent policy initiatives to tackle rare diseases in jurisdictions across the region are highlighted below.

Regional Policy Highlights

• Australia: In 2022, the Australian Federal Government announced that it will support and resource a Health Technology Assessment (HTA) Policy and Method Review (Review) to keep pace with rapid advances in health technology, innovation, and research to provide patients with faster access to affordable medicines. The review is expected to significantly strengthen the healthcare delivery for all Australians, including those with rare diseases. It follows the government’s 2020 release of the National Strategic Action Plan for Rare Diseases, which marked the country’s first nationally coordinated effort to address rare diseases.
• India: In 2021, the National Policy of Rare Diseases was approved, aiming to support domestic research, lower the cost of treatment, and screen and detect rare diseases at an early age.
• Japan: In 2015, the Intractable Disease Health Care Act entered into effect, defining rare diseases as any condition affecting fewer than 50,000 people domestically or disorders deemed to be intractable or difficult and affecting up to 180,000 people domestically.
• Singapore: In 2019, the Ministry of Health defined rare diseases in a press release as conditions affecting fewer than 1 in 2,000 people, however, there continues to be no formal legal definition.
• South Korea: In 2015, the Rare Disease Management Act was passed, allocating greater R&D support, creating plans for the prevention, diagnosis, and treatment of rare, diseases, and defining rare diseases as those affecting fewer than 20,000 people in the country.
• Taiwan: In 2000, the Rare Disease and Orphan Drug Act was promulgated, with 225 diseases recognized as rare diseases, granting support for research, development, and accessibility of orphan drugs and special nutritional foods, strengthening the prevention and early diagnosis of rare diseases, and enhancing intensive care for patients with rare diseases.

China’s System in Focus

Given China’s huge scale, rare diseases present a particularly noteworthy healthcare policy challenge, as even a small percentage of the total population represents a very large group. According to an official announcement, it is estimated more than 20 million people have rare diseases in the country. The government is tackling this significant health burden through a variety of efforts.

Determining Rare Diseases

In May 2018, the National Health Commission and other authorities jointly released China’s first Rare Disease List (RDL), including 121 diseases and conditions, a milestone event in the treatment of these conditions in the country. Still, official criteria for determining a rare disease in China remains undefined.

Medicine Access

At the same time, the government is working to broaden access to rare disease medicines. Increasingly, they are being granted priority for marketing review and approval, listed in China’s clinically urgently needed medicines, and being introduced through some pilot zones. From 2018 to 2022, a total of 56 medicines for rare diseases were approved in China. From 2018 to 2020, the government released lists of 81 clinical-urgently needed medicines, including 39 for rare diseases. By March 2022, 23 out of the 39 had applied for marketing registration, with 22 finishing the process. Despite the current efforts in priority review and approval, the marketing time of rare disease medicines in China still takes 9.5 years longer than in the EU.

Insurance Coverage

In China, since 2020, city-level commercial insurance programs guided by the government and operated by insurance companies have seen explosive growth. While more than 200 prefectural-level cities in 27 provinces have announced such programs so far, many programs cover medicines for rare diseases. For example, Shanghai included five medicines with high prices for three rare diseases into its program, providing 30 percent payment coverage for those with existing conditions and 70 percent for those without, for a total payment of up to RMB 1 million. While some local authorities have extended medical assistance to rare disease patients, there is no such directive on the national level. In principle, some beneficiaries of the central government’s other assistance programs (such as poverty alleviation) are guaranteed a 70 percent reimbursement rate for rare disease medicines. Thanks to increased government efforts, private companies, non-profit organizations, and academics alike are increasingly engaged.

In October 2022, the government announced that 45 medicines targeting 26 rare diseases listed in the RDL were added to the National Reimbursement Drug List (NRDL), following 19 such additions since 2018. Due to the government’s efforts negotiating with enterprises, newly added medicines achieved price cuts of 52.6 percent, benefiting even more patients. To include more medicines in the NRDL, the government set up a separate track and granted priority review for such medicines, resulting the inclusion of another 7 medicines into the 2023 NRDL.

Manufacturing support

In May 2022, China’s government released a draft policy that has yet to be officially promulgated, granting rare disease medicine MAHs a market exclusiveness period of seven years. This decision is very much in line with the global practice and grants a market boost for biopharmaceutical companies with products in this domain.

Looking ahead

As governments across the Asia Pacific region continue to prioritize strategies for tackling rare diseases, the large and diffuse population of patients across the region can benefit tremendously.

As more jurisdictions create official definitions for rare diseases, comprehensive support will be significantly enhanced. Innovations in pricing mechanisms also stand to broaden access to the critical therapies and treatments needed for these harmful conditions. It is also crucial to recognize the importance of reforming and streamlining the avenues of primary care delivery for rare diseases. This includes telehealth, virtual care, or the entitlement of pharmacists to prescribe medications to patients, making it easier and faster for patients to access life-saving medicines.

MNCs with related products are well advised to monitor the latest updates regarding government initiatives for rare diseases across the region in the coming years to best utilize the growing support to facilitate patients with the latest and most innovative medicines and treatments.

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