6 hours Ago By Rema Nagarajan
Representative Image On World Rare Diseases Day observed on February 28, the focus in India is on the struggle of patients with rare diseases to access treatment, which is often too expensive because of exorbitant monopoly pricing of the drugs needed. Despite the National Policy for Rare Diseases mentioning facilitating of indigenous manufacture, the government seems reluctant to exercise this option as demonstrated by the fight of a patient with spinal muscular atrophy to access treatment. Why is the treatment for rare diseases so expensive? Rare diseases are debilitating lifelong conditions with very low prevalence.
Such diseases being rare, the patient pool is very small and hence drug companies do not find it commercially viable to do research on finding drugs for them. When companies do discover a drug, they charge hefty amounts claiming that they need to recover the cost of R&D though none of them ever reveal the R&D cost incurred. The prices are beyond reach for most, if not all, patients suffering from the disease.
Getting data on the prevalence is also difficult since diagnosing is difficult as doctors are not trained adequately to identify them, except in a few tertiary care centres. What is India’s policy on rare diseases? The policy envisages creating a registry of rare diseases, measures to help early detection and a fund to give financial assistance to help patients access treatment. It divides rare diseases into three categories—group one constitutes diseases that have a one-time curative treatment like a surgery or organ transplant or a one-time expensive drug, group two includes diseases requiring long term or lifelong treatment of relatively low cost or disorders that can be managed with special dietary formulae or food for special medical purposes and group three includes diseases with definitive but exorbitant treatment which would be needed lifelong.
Since most of the diseases are prohibitively expensive to treat, especially for group 3 diseases, the policy outlines many strategies which includes focusing on lowering incidence and prevalence of such rare diseases and making drugs for such diseases affordable. Strategies to make drugs affordable include taking legal and legislative measures “to create a conducive environment for indigenous manufacture of drugs for rare diseases”. Why was a patient with spinal muscular atrophy (SMA) forced to approach courts despite being given Rs 50 lakh as financial assistance? The patient, 24-year-old Seba PA, pointed out that the drug she needed, Risdiplam , would cost her roughly Rs 18 lakh per month and hence with the Rs 50 lakh cap on financial assistance, she would not be able to afford prolonged treatment.
She has already exhausted the Rs 50 lakh given for her treatment. Her petition pointed out that the medicine she needed had a patent monopoly, which prevented local manufacture, and hence was priced very high due to lack of competition. According to drug pricing expert Dr Melissa Barber in Yale University, Risdiplam, which at its current price would cost about Rs 72 lakh per year per patient, could be produced for as little as Rs 3,000 per year if India allowed local manufacture.
If the manufacturer, Roche, seeks fair compensation, they must provide transparent accounts of their expenditure on bringing this drug to the market, argued Dr Barber. Understanding that it might not be possible for the government to support her indefinitely to access such an exorbitantly priced drug, the petitioner sought directions from the court for the government to implement the national policy by issuing a compulsory licence to enable indigenous manufacture, which could make the drug easily affordable for all SMA patients. Patent law in India allows compulsory licences for “government use” and for selling “on a non-commercial basis”.
What was the central government’s objection to supporting the continuation of Seba’s treatment? The government argued that in resource-constrained settings, it was “crucial to balance the competing interests of public health in order to achieve the optimal outcomes for the resources allocated”. Pointing out that there are more than 3,000 registered patients with different rare disease registered across India, the government stated that funding their treatment could cost Rs 6,400 crore to Rs 34,000 crore annually as the treatment could be prohibitively expensive, which would place substantial strain on public health resources. It further stated that continuing support for Seba might set a precedent with other patients demanding similar relief.
What has been the result of Seba’s petition? The Kerala high court ordered the central government to continue supporting Seba’s treatment while asking the government to file an affidavit on what action it has taken based on the clause in the national policy on rare diseases. The clause states that the department of pharmaceuticals and the department for promotion of industry and internal trade will be requested to promote local development and manufacture of drugs for rare diseases by public and private sector pharmaceutical companies at affordable prices and necessary legal and legislative measures will be taken for creating a conducive environment for indigenous manufacturing of drugs for rare diseases at affordable prices. The government challenged the HC order in the Supreme Court, which stayed the order and remarked that granting a compulsory licence might have “international ramifications”.
Public health activists argued that this could prove to be a death sentence for Seba and others like her with the government not even considering the option of indigenous generic production of the medicine, which could have helped thousands of SMA patients. They argued that it was tantamount to denying her the right to life enshrined in the constitution. Stay informed with the latest India news, updates on public holidays , and bank holidays in March .
.
