Report on the Implementation of National Reimbursed Orphan Drugs in China (2023) Released in Shanghai: Multi-Stakeholder Collaboration to Address the 'Last-Mile' Access Barriers

ShanghaiOctober 18, 2023/PR Newswire/ -- On October 14, 2023, the release event for the report "Breaking Through the Last Mile: Research on the Implementation of Nationally Negotiated Rare Disease Drugs in China and Policy Recommendations (2023)" (hereinafter referred to as the "report"), hosted by the Cord Rare Disease Foundation and supported by enterprises such as Hansoh Pharma, was held in Shanghai. The report shows that compared with other negotiated drugs, nationally negotiated rare disease drugs in China are characterized by slower progress in medical institution allocation, lower regional coverage, and poorer affordability. Therefore, more than ten experts from hospitals, health insurance, and pharmaceutical fields present at the event called for increased attention to the issue of medication difficulties faced by rare disease patients, working together to resolve the "last mile" bottleneck in the implementation of nationally negotiated rare disease drugs.

Difficulties in the Implementation of Nationally Negotiated Drugs for Rare Diseases

In recent years, to ensure that patients with rare diseases have access to medical care, appropriate medications, insurance coverage, and guaranteed benefits, the government has formulated corresponding safeguarding and incentive policies covering various aspects such as the research and development, market approval, regulation, pricing, reimbursement, and supply of drugs for rare diseases. Among these, national medical insurance negotiations play a crucial role in promoting the inclusion of rare disease drugs into the medical insurance system. To date, a total of 30 rare disease drugs have been included in medical insurance through national negotiations, with 26 of them still within the agreement period during the survey period of this report. These 30 negotiated drugs for rare diseases cover 18 types of rare diseases, corresponding to nearly 800,000 patients with rare diseases in China.

However, the successful negotiation of rare disease drugs and their inclusion in the national medical insurance is only the first step in improving the accessibility of rare disease drugs. The actual implementation of nationally negotiated rare disease drugs still requires going through processes such as online procurement and hospital access.

China’s Rare Disease Diagnosis and Treatment Collaboration Network is a medical cooperation system specially established to strengthen the management of rare diseases and improve the level of diagnosis and treatment. It consists of 324 hospitals distributed across provinces, regions, and cities in China. These hospitals generally lead the development of rare disease treatment standards in China and profoundly influence the clinical use and promotion of innovative therapies and drugs for rare diseases in the country. However, in this survey, the highest availability rate of national negotiation drugs for rare diseases covered only 37% of the hospitals in the collaboration network, while the lowest availability rate had a coverage rate as low as 3%. This indicates that even in the relatively advanced hospitals within the rare disease collaboration network, patients still face significant challenges in terms of accessibility and convenience for long-term medication.

In addition to the difficulty of entering hospitals, rare disease drugs negotiated at the national level in China also face many practical problems, such as insufficient outpatient drug coverage, restrictions on inpatient drug use due to DRG/DIP payment systems, and the lack of smooth implementation of the "dual channel" policy for purchasing drugs at pharmacies.

Joint Efforts to Break Through the Last Mile

As the primary responsible party for the clinically appropriate use of negotiated drugs, medical institutions generally have a low level of provision for rare disease drugs negotiated at the national level. Since some rare disease drugs (such as inebilizumab for the treatment of NMOSD) are the only specific medications covered by medical insurance in China for their respective conditions, their accessibility significantly impacts the treatment of patients with rare diseases.

To this end, the report recommends that local medical insurance departments should clarify that nationally negotiated drugs for rare diseases are not subject to the total medical insurance cap of medical institutions and can be separately accounted for and paid; local health departments should ensure that nationally negotiated drugs for rare diseases are not restricted by assessment indicators such as the drug-to-revenue ratio of medical institutions; encourage hospitals in the rare disease collaboration network to fully equip nationally negotiated drugs for rare diseases, while ensuring the continuity of these drugs entering the hospital.etc.。

In response to the insufficient outpatient medication security for rare diseases, the report recommends that provincial medical insurance departments establish special disease outpatient security systems. It also suggests including nationally negotiated rare disease drugs in the outpatient special drug list, expanding outpatient coverage for rare disease patients, reducing unnecessary hospitalizations of rare disease patients, and avoiding waste of medical resources.

For the issue of rare disease patients' inpatient medication being restricted by DRG/DIP payment, the report suggests including national negotiation drugs for rare diseases in a separate payment or exclusion payment category, without DRG settlement.

Moreover, considering the current difficulties in the inclusion of rare disease drugs negotiated at the national level into hospitals and the incomplete alignment of policy systems, the important role of retail pharmacies as a supplementary channel for drug supply can be further leveraged. However, issues such as low levels of information transparency, unclear drug delivery and infusion processes, and ambiguous responsibility divisions in "dual-channel" pharmacies in some regions have made it difficult for rare disease patients to access medications through this channel. To address this, the report recommends that local medical insurance departments promptly include rare disease drugs negotiated at the national level in the "dual-channel" drug list, promote the flow of electronic prescriptions for these drugs from hospitals to pharmacies, clarify the responsibilities of all parties involved in the dispensing of injectable rare disease drugs via the "dual-channel" system, and ensure patient medication safety.