Indian and Dutch Medical Collaboration Secures Rare Thyroid Disorder Medication for Children

A collaborative effort between Indian and Dutch physicians, along with civil society groups, has arranged a six-month supply of the rare medication Tiratricol for seven children in India suffering from a genetic thyroid disorder. The initiative bypasses high international commercial costs through legally approved personal use imports and compounded pharmacy alternatives.

A group of seven children in India diagnosed with a rare thyroid-related genetic disorder are set to receive a six-month supply of the medication Tiratricol later this month. The supply is being secured through a collaboration involving Dutch and Indian physicians, supported by civil society organizations.

Paediatrician Sivaranjani Santosh stated that the medicine is being imported from the Netherlands through legally approved channels designated for personal use. Dr. Santosh noted that Tiratricol is currently unavailable in India, and its international commercial price can reach approximately ₹4 crore per patient annually. In contrast, the imported compounded version costs about ₹2.5 lakh a year. Funds to support the patient families have been raised independently.

Dr. Santosh's patient, an approximately 20-month-old child named Arav, requires the medication lifelong. The genetic defect prevents certain thyroid hormones from reaching the brain, which impacts the child's development and metabolism. Egetis Therapeutics, a Swedish company, markets Tiratricol as a thyroid hormone receptor agonist used to treat MCT8 deficiency, also known as Allan–Herndon–Dudley syndrome.

Wilbert Bannenberg, Chairperson of the Pharmaceutical Accountability Foundation (PAF), stated that Tiratricol is an existing drug that was repurposed in the Netherlands and sold to the commercial company. PAF noted that while the drug costs nearly €180 per tablet in Europe, it can be compounded by a pharmacist in the Netherlands for a fraction of that price. PAF and Universities Allied for Essential Medicines Netherlands are actively raising funds for the children's year-long treatment.

Chetali Rao of India's Working Group on Access to Medicines and Treatments noted that while compounding is recognized in India, it is not actively pursued.

According to PAF, Tiratricol has been known since 1953 and was available in France for thyroid conditions beginning in 1974. In 2014, publicly funded researchers at Erasmus University Medical Center found that the drug could treat AHDS. In 2017, Erasmus MC sold the related intellectual property rights to Egetis Therapeutics without a fair price clause, according to PAF, which noted the drug costs about €441,000 per year in Germany.

Egetis was approached for comment via email but a response is awaited. PAF alleged that Egetis conducted a clinical trial for the drug in India but previously declined a request from Dr. Santosh for compassionate use for her patient.

The year-long treatment cost for the seven children totals €14,200. While €5,600 has been raised in India, PAF aims to secure the remaining €8,600 through crowdfunding in the Netherlands.

"This case highlights significant structural challenges in the global pharmaceutical market, particularly regarding access to repurposed medications for rare diseases. When intellectual property rights are transferred without fair pricing frameworks, patients in developing regions face extreme financial barriers. Collaborative cross-border efforts between civil society groups, medical professionals, and alternative compounding channels provide a vital workaround for essential treatments, emphasizing the need for accessible healthcare frameworks globally." — Dr. Shishir Gupta, Founder & CEO, StartupLanes