24 rare diseases included in universal coverage, but challenges remain

Despite the high-cost therapies, the National Health Security Office (NHSO), in 2020, introduced treatments for 24 rare diseases known as inherited metabolic disorder or inborn errors of metabolism, under the benefits package of the Universal Coverage Scheme.

A person diagnosed with one of those diseases will be transferred to one of seven tertiary hospitals with clinical genetics experts and advanced healthcare technologies.

The hospitals, mainly based in Bangkok, are: Chulalongkorn Hospital, Thammasat Hospital, Siriraj Hospital, Ramathibodi Hospital, Phramongkutklao Hospital, Queen Sirikit National Institute of Child Health, and Srinagarind Hospital in Khon Kaen province.

The move has been made possible by the collaboration among players, including patient groups, clinicians, the Public Health Ministry and the NHSO. However, there are still huge challenges as there are more than 6,000 rare diseases for which many patients still cannot access treatment.

On February 28, all players joined a seminar for the recognition of Rare Disease Day at Centara Government Complex Hotel and Convention Centre in Chaeng Watthana, Nonthaburi province. The event was jointly organised by NHSO, the Public Health Ministry and Thai Rare Disease Foundation. Several experts who play vital roles in pushing forward rare-disease care at a policy level, including Prof Duangrurdee Wattanasirichaigoon from Mahidol University's Faculty of Medicine Ramathibodi Hospital and also President of Medical Genetics Network, Genetic Society of Thailand; Prof. Kanya Suphapeetiporn from Chulalongkorn University's Faculty of Medicine; Asst. Prof. Achara Sathienkijkanchai, and Prof. Cherdchai Nopmaneejumruslers from Mahidol University's Faculty of Medicine Siriraj Hospital and the chairman of NHSO's working committee on rare-disease care, took part in the seminar.

NHSO secretary-general Sakchai Kanjanawatana and Vice Minister at the Office of the Prime Minister, Prapon Tangsrikertikul, gave the opening speech at the event.

■ Gain public attention

Health experts estimate that 5 per cent of the Thai population have one of the rare diseases, meaning that three million out of more than the 65 million Thai population have some of those diseases. However, only around 20,000 of them receive treatment.

“Three million is a lot. Why aren't rare diseases a matter of concern for most people? One of the answers is, we don’t have a good strategy to raise this issue and gain public attention,” said Somchai Jitsuchon, an economic researcher at Thailand Development Research Institute Foundation.

He admitted that if his daughter did not have a rare disease, he might not care about it like many others.

“As an economist, I'm interested in the lost opportunity cost for Thailand due to the illness caused by rare diseases. We should have more studies on that, so that society will see the need to address this issue.”

Many technocrats express concern about the financial burden on the government if it provides free treatment to patients with rare diseases. Currently, Civil Servant Medical Benefits Scheme fully covers rare-disease care costs, while there is a gap for patients under the UCS and Social Security Scheme.

Somchai believes that Thai society has the ability to handle the cost if it can have an efficient tax collection system.

■ Driving policy with data

Data on rare diseases are scarce due to inadequate research and a small number of patients.

“When there is no data, patients miss the opportunity," said Asst. Prof. Thipwimol Tim-Aroon, a clinical geneticist from Ramathibodi Hospital. “Data is essential for convincing policymakers to address rare diseases in healthcare policy. We need the registry system for rare-disease patients.”

Empowering patient groups is the first step to collect data, as it creates a hub for data-sharing among patients, she said.

One success story is the hemophilia patient club, which gathered data and case studies from its networks and used them to convince the NHSO and the Thai government to include hemophilia treatment in the UCS benefits package.

Asst. Prof. Prapat Suriyaphol from Siriraj Hospital, Mahidol University, said during the seminar that digital technology could improve collection of data on rare diseases.

His team is adopting Fast Healthcare Interoperability Resources -- a standard describing data formats, elements and an application programming interface for exchanging electronic health records -- for the development of Thai patients’ dashboard containing data of their health status and medical records.

He hopes that the dashboard, expected to be launched by next year, will point out the need to address rare diseases at a policy level.

■ Patient empowerment

The voice of patients has a large influence on Thai health policy and the UCS. Genetic LSD Foundation is one of many patient groups that plays an important role in pushing the government to provide rare-disease medication and treatment.

Established in 2009, the foundation focuses on empowering patients by expanding their knowledge about Lysosomal Storage Diseases, the complex and multi-systemic genetic disorders that affect various organs throughout the body. It raises funds and organises activities in which patients can meet, comfort each other, and work together on pushing health policy.

The foundation’s president, Boon Puttipongthanachote, recalled his group's success in 2013 in convincing the government to add Gaucher disease medication to the National List of Essential Medicines provided to patients under three healthcare schemes.

“We highlighted the fact that the drug was highly effective. Providing it to patients would help their families avoid bankruptcy,” said Boon, adding that his group is currently pushing for access to medication for Pompe disease.

■ Improve regulatory system

The regulatory system is an important component that drives the policy to develop rare disease innovations and products.

Prof John CW Lim, founding Executive Director of the Centre of Regulatory Excellence (CoRE) at Duke-National University of Singapore Medical School, raised three key challenges that regulators and governments face in the Asia Pacific region.

First is insufficient regulatory knowledge and capacity, especially in smaller developing regulatory agencies; second, fragmentation in the regulatory systems that industry faces when it tries to bring new products to the market. And thirdly, the lack of platforms for regulatory science and policy innovation.

CoRE was set up in 2014 with support from Singapore's Ministry of Health to address the three challenges by using several approaches, including increasing institution regulatory talent, offering capacity building programmes to regulatory authorities, and establishing networks to bring together stakeholders from across government, industry, academia and research.

“At the end of the day, regulation can never exist in a vacuum. The overall government regional and global systems all come into play,” said Lim, adding that rare diseases are being discussed in regional frameworks, including the Asia-Pacific Economic Cooperation Action Plan for Rare Diseases which guides the coordination of different stakeholders across sectors.

“But ultimately, the patient's voice and patient-centric models are not only important but will become increasingly important across all areas of health and social systems, not only in promoting regulatory agility and regulatory innovation but to make sure that the patient's voice and the patient-centric model form the basis and are at the heart of how we approach rare diseases going forward,” Lim said.