A drug, custom-made in The Hague, for 7 kids in India

The drug, tiratricol, is goi­ng to be compounded by Tr­ansvaal Pharmacy for 1.8 euro per capsule (Representative image)

NEW DELHI: Dutch and Indian physicians and civil society organisations have come together to ensure access to a rare disease drug for seven children in India, by compounding the drug in the Hague and sending it to India. Compounding is the process by which a medicine is custom-made in small quantities.The drug, tiratricol, is going to be compounded by Transvaal Pharmacy for 1.8 euro per capsule, which is 99 times cheaper than the commercial drug sold by the Swedish company Egetis in Germany for 180 euro (over Rs 19,000) per tablet.Currently, the family of one of the children is importing the medicine from Germany. It costs them Rs 3 lakh per month. Since it is prescribed for life, buying it is proving to be unsustainable.The seven children suffer from a rare disease, Allan Herndon-Dudley Syndrome (AHDS), also known as MCT8 deficiency. Due to a genetic defect, their thyroid hormones are unable to reach their brain cells effectively, leading to severe developmental issues. Research by the Erasmus University Medical Centre (Erasmus MC) has confirmed that tiratricol, a medication known since 1953, could reduce the physical manifestations of the disease. Tiratricol has been in the market in France since 1974 to treat patients with thyroid conditions.In 2014, doctors at Erasmus MC, through research which involved 14 million euro of public funding, discovered that tiratricol could also be used to treat AHDS. Erasmus MC sold the intellectual property rights to Egetis without a “fair price clause”. In 2025, Egetis received European marketing authorisation for tiratricol, granting it 10 years of market exclusivity under the European orphan drug legislation. By 2026, the price per tablet of tiratricol had gone up from 0.35 euro, at which it was being sold in France in 2017, to almost 180 euro per tablet in Germany.The Netherland-based Pharmaceutical Accountability Foundation (PAF) which works to ensure access to medicines and medical technologies in a sustainable manner, is coordinating the effort to make the drug available to children in India. Wilbert Bannenberg of PAF termed Egetis’ pricing of tiratricol an “unethical business model”.

A news report on the funding effort, which caught the attention of PAF, kicked off the Dutch-India collaboration.Since compounding by a pharmacy is only an emergency fix, for a long-term solution PAF is working with access to medicine activists and paediatricians in India for affordable production and registration of tiratricol in India, which could help start generic production in a few years.