The Ministry of Health and Family Welfare has recently given its stamp of approval to the National Rare Disease Policy 2021. This comes in the wake of a directive by the Delhi High Court that mandates the Centre to establish a Rare Diseases Committee and Fund and to finalize and notify the National Health Policy for Rare Diseases before the end of March 2021.
Primary Objectives
The aim of this policy is multifaceted: to enhance focus on indigenous research and local production of medicines, to reduce the cost of rare disease treatment, and to ensure early detection of these diseases for their prevention.
Significant Provisions of The Policy
The policy introduces categorization of rare diseases into three groups: Group 1 includes disorders susceptible to one-time curative treatment; Group 2 consists of those necessitating long-term or lifelong treatment, and Group 3 comprises diseases where definitive treatment is available, yet optimal patient selection presents a challenge due to high cost and lifelong therapy.
Financial Support Structure
The policy offers financial support of up to Rs. 20 lakh for patients suffering from rare diseases categorized under Group 1 under the umbrella scheme of the Rashtriya Arogya Nidhi. This Scheme provides financial aid to patients below the poverty line burdened with major life-threatening diseases, offering them the chance to receive medical treatment at government super-specialty hospitals or institutes.
Extended Beneficiary Base
The beneficiaries of such financial assistance won’t be exclusive to below poverty line families. Instead, it will be extended to approximately 40% of the population who are eligible as per Pradhan Mantri Jan Arogya Yojana norms, targeting their treatment in Government tertiary hospitals only.
Alternate Funding Mechanism
The policy also suggests an alternative funding mechanism, voluntary crowdfunding of treatment by establishing a digital platform for individuals and corporate donors to contribute voluntarily to the treatment cost of patients with rare diseases.
Establishment of Centres of Excellence
The policy aims to fortify tertiary healthcare facilities for the prevention and treatment of rare diseases. It proposes to designate eight health facilities as ‘Centres of Excellence’ and provide them a one-time financial support of up to Rs. 5 crore to upgrade diagnostic facilities.
National Registry
It also plans to create a national hospital-based registry of rare diseases, thereby ensuring that adequate data and comprehensive definitions of such diseases are available for research and development purposes.
Potential Concerns
Despite the numerous advantages of the policy, concerns have been raised about sustainable funding. Patients with Group 3 disorders require sustainable treatment support. The lack of such support places these patients, primarily children, at risk and at the mercy of crowdfunding. Another significant concern is the expensive, limited availability of drugs due to the dearth of pharmaceutical companies producing medicines for rare diseases.
Rare Diseases – A Snapshot
Rare diseases are classified into 6,000-8,000 categories, with less than 5% having available therapies. Examples of these diseases include Lysosomal Storage Disorders (LSD), Pompe disease, cystic fibrosis, muscular dystrophy, spina bifida, haemophilia, etc. About 95% of rare diseases have no approved treatment, and less than 1 in 10 patients receive disease-specific treatment.
India has around 50-100 million people affected by rare diseases or disorders, with approximately 80% of these being children leading to high morbidity and mortality rates due to the life-threatening nature of these diseases.
