Beyond customs – Treating the Rare Diseases | 3rdd April 2023 | UPSC Daily Editorial Analysis

What's the article about?

• It talks about the initiatives that the Union government is taking to reduce the burden of rare diseases.

Relevance:

• GS2: Issues Relating to Development and Management of Social Sector/Services relating to Health, Education, Human Resources;
• Prelims

Context:

• Recently, the Central Government has given full exemption from basic customs duty on all drugs and Food for Special Medical Purposes imported for personal use for treatment of all Rare Diseases listed under the National Policy for Rare Diseases 2021 through a general exemption notification.
• In order to avail this exemption, the individual importer has to produce a certificate from Central or State Director Health Services or District Medical Officer/Civil Surgeon of the district.
• This exemption will result in substantial cost savings and provide much needed relief to the patients.
• Along with medicines for rare diseases, the government also removed customs on the cancer immunotherapy medicine Pembrolizumab (Keytruda).

What are rare diseases?

• Rare diseases as the name suggests are conditions that affect very few people.
• The World Health Organisation defines it as any debilitating lifelong disease or disorder with a prevalence of ten or less per 10,000 population; other countries follow standards ranging between 1 and 10 cases per 10,000 to define a condition as a rare disease.
  • Example: Lysosomal Storage Disorders (LSD), Pompe disease, cystic fibrosis, muscular dystrophy, spina bifida, haemophilia etc.
• There are about 7,000 to 8,000 conditions globally that have been defined as rare diseases. The landscape of rare diseases keeps changing, with newer conditions being identified and reported constantly.

Initiatives by the Government for treatment of rare diseases:

• The Government has launched National Policy for Rare Diseases (NPRD), 2021 in March, 2021 for the treatment of rare disease patients.
• The salient features of NPRD, 2021 are as under:
  • The rare diseases have been identified and categorized into 3 groups namely Group 1, Group 2 and Group 3.
    • Group 1:  Disorders amenable to one-time curative treatment.
    • Group-2: Diseases requiring long term/lifelong treatment having relatively lower cost of treatment and benefit has been documented in literature and annual or more frequent surveillance is required.
    • Group 3: Diseases for which definitive treatment is available but challenges are to make optimal patient selection for benefit, very high cost and lifelong therapy.
  • Provision for financial support of up to Rs. 50 lakhs to the patients suffering from any category of the Rare Diseases and for treatment in any of the Centre of Excellence (CoE) mentioned in NPRD-2021, outside the Umbrella Scheme of Rashtriya Arogaya Nidhi.
  • Eight (08) Centres of Excellence (CoEs) have been identified for diagnosis, prevention and treatment of rare diseases.
  • Five Nidan Kendras have been set up for genetic testing and counselling services.
  • The NPRD, 2021 has provisions for promotion of research and development for diagnosis and treatment of rare diseases; promotion of local development and manufacture of drugs and creation of conducive environment for indigenous manufacturing of drugs for rare diseases at affordable prices.
• Department of Pharmaceuticals has initiated the implementation of Production Linked Incentive Scheme for Pharmaceuticals.
  • The Scheme provides for financial incentives to manufacturers selected under the Scheme for domestic manufacturing of various product categories, which also include Orphan drugs.

Analysis:

• While rare diseases are defined by their infrequent occurrence in the population, the sheer number of diseases (estimated between 7,000-8,000 conditions; 450 of them have been reported from hospitals in India), and the number of people with some form of rare diseases in India (an estimated 100 million) make it a problem that cannot be ignored.
• When the NPRD was released, it underlined the magnitude, and specified that demands could only be considered in the context of the available scarce resources that would have to be used judiciously.

Way Forward:

• While striking a note for the goal of affordable health care, the government must ensure that its directions are followed in full, besides staying the course to innovate solutions for this category of patients.