Tanapol Dokkaew, kidney disease survivor and avid advocate for rare diseases

Tanapol has dedicated himself to addressing rare diseases since 2019, predating the Covid-19 pandemic. His efforts have identified over 300 rare diseases in Thailand. Currently, individuals with rare diseases in Thailand have access to medications and treatments for 24 diseases, covering more than 40 diseases.

As a kidney policy advocator, Tanapol has seen the trend of a rare and often overlooked condition called Atypical Hemolytic Uremic Syndrome (aHUS), which has proved to be silently lethal for some patients. This uncommon condition triggers tiny blood clots in the body’s small vessels, significantly affecting critical organs like the kidneys, leading to irreversible damage and eventual kidney failure.

Highlighting another rare disease with potential implications for kidney health in Thailand, Tanapol spotlights Atypical Hemolytic Uremic Syndrome (aHUS). This disorder disrupts the normal functioning of the complement system, leading to red blood cell rupture, platelet formation, and urea accumulation in the blood, often manifesting through paleness and weakness symptoms.

Currently considered a rare disease in Thailand and globally, aHUS present a considerable challenge in screening and diagnosis due to the lack of definitive clinical practice guidelines and laboratory tests. This delay contributes to late diagnosis of patients, with up to 79% loss of life or progress to end-stage renal disease within three years of diagnosis.

Considering the hurdles, Tanapol stands as a prominent advocate in Thailand, urging the government to adopt a comprehensive approach to rare disease screening akin to Taiwan’s model. Taiwan achieves a 100% screening rate for rare diseases in newborns, supplemented by follow-up screenings over 6-8 years.

Taiwan, with a population of 23 million, boasts 25 centres dedicated to supporting individuals with rare diseases across the country. In contrast, Thailand, with a larger population of 67 million, faces the challenge of providing comprehensive care for those with rare diseases. Currently, individuals with rare diseases in Chiang Mai may need to undertake impractical journeys to Khon Kaen for treatment due to the limited availability of specialised centres in their region.

Malaysia’s Ministry of Health a dedicated fund and budget for the management of rare diseases, aimed at supporting the treatment of rare diseases, aiming to support follow-up treatment for diagnosed patients and initiate treatment for those on waiting lists.

Tanapol underscores the significance of proactive measures within Thailand’s public healthcare system. These measures involve implementing early screening for rare diseases from infancy, guaranteeing equitable access to treatment, and establishing support mechanisms for patients in their homes. Central to this approach are specialised healthcare professionals and well-equipped facilities tailored to address the unique challenges of rare diseases.

He added, “Firstly, my primary concern is implementing 100% proactive measures in Thailand’s public healthcare system, similar to Taiwan. We aim to avoid relying solely on screening when diseases emerge. Secondly, we advocate for quicker screenings and diagnoses to ensure prompt patient treatment. Establishing a supportive unit with a comprehensive drug system that adheres to standards for referrals and medication is crucial. The entire system should be streamlined for efficiency and effectiveness.”

In Tanapol’s vision, tackling rare diseases is not just a medical challenge but a societal responsibility. He envisions a scenario where every individual receives timely and effective care regardless of their condition. Tanapol is committed to safeguarding the right to standardised treatment and actively advocates for the rare disease community and society.

In his pursuit, Tanapol extends a warm welcome to individuals affected by rare diseases and everyone else to join his association. This inclusive approach emphasises the importance of collective efforts in ensuring equitable access to care for all.