According to a study conducted by the Economist on the diagnosis and treatments of rare diseases and the time lag between licensing and pricing of 8 specific orphan drugs, South Korea and Taiwan are at the bottom of the table of 7 countries in the study. In South Korea and Taiwan, patients have no say in policies or decisions concerning rare diseases and orphan drugs.
Despite the fact that Taiwan is the 5th country in the world to make a dedicated law for rare diseases and was invited to share the experiences in the UN, the NHIA now gets into dispute with hospitals over the NHI global budget. In order to haggle with the NHIA, the hospital representatives propose zero growth for the special fund for rare diseases. The final decision will be determined by the MOHW Minister.
The Economist compared the rare diseases systems in France, Germany, the UK, Australia, Japan, South Korea and Taiwan. Germany, Japan and the UK all have a special pathway for expediting the licensing and pricing of orphan drugs. In these 3 countries, most orphan drugs in the study were licensed and were priced within a year. In France and Australia, most orphan drugs were licensed and priced, but it took about 2-4 years. However, in South Korea and Taiwan, only about 2 or 3 drugs in the study were licensed and priced, and it took about 30 months for a licensed drug to be priced by the NHI.
According to a campaigner, the NHI in Taiwan only provides about 15% of orphan drugs. Many life-saving orphan drugs are just beyond patients’ reach. Taiwan is seriously behind other countries in providing public-funded treatments for rare diseases.
The study also pointed out the challenges faced by orphan drug developers in meeting the HTA requirement because of the lack of clinical evidences from a usually rather small patient population. Not to mention that orphan drugs often fail to satisfy the ICER/QALY assessment due to their high costs.
An ICER specialist, Dr Pu Rong-Fang, pointed out that the NHIA usually refuses to list orphan drugs on grounds of uncertain efficacy. Dr Pu said that the government should not focus only on cost and benefits, instead, patients’ quality of life and social justice should also be taken into account.
Dr Lee Po-Chang, the Director General of the NHIA expressed that price review is more efficient if it is supported by the HTA. The dispute on whether to list an orphan drug usually happens in the NHI Drug Dispensing Items and Fee Schedule Joint Establishment Meeting where doctors with different background have different points of view.
The new MOHW Minister, Hsieh Jui-Yuan, expressed his concerns and instructed the agencies in charge to improve the accessibility of orphan drugs. He requested the CDE to review the HTA requirement, the TFDA to review the licensing process and the NHIA to schedule the reimbursement of orphan drugs. All these are with a view to improving patients’ access to orphan drugs. The MOHW Minister stressed that caring for patients with rare diseases is a commitment to life.
【2022-09-30 / Unite Daily】
