• The central government is planning to approve generic drugs that are manufactured in the country for rare diseases soon.

• Apart from 13 rare diseases drugs for sickle cell, will also be made available.

• While the first of the four generic drugs made in India will be available soon, four others will be launched next year.

• At present, the drugs used for treating rare diseases are imported. They are very expensive too. Parents of children afflicted with rare diseases are  dependent on crowdfunding. The new drugs will bring down costs. 

• Four drugs have been approved and will soon be available in the market for four rare diseases, which include Tyrosinemia Type 1, Gaucher’s Disease, Wilson’s Disease, and Dravet/ Lennox Gastaut Syndrome-related seizures.

What are rare diseases? 

• WHO defines rare disease as often debilitating lifelong disease or disorder with a prevalence of 1 or less, per 1,000 population. 

• However, different countries have their own definitions to suit their specific requirements and in context of their own population, healthcare system and resources. 

• In the US, rare diseases are defined as a disease or condition that affects fewer than 200,000 patients in the country (6.4 in 10,000 people).

• The European Union defines rare diseases as a life-threatening or chronically debilitating condition affecting no more than 5 in 10,000 people. 

• Japan identifies rare diseases as diseases with fewer than 50,000 prevalent cases (0.04 per cent) in the country.

• The field of rare diseases is complex and heterogeneous. The landscape of rare diseases is constantly changing, as there are new rare diseases and conditions being identified and reported regularly in medical literature. Apart from a few rare diseases, where significant progress has been made, the field is still at a nascent stage. 

• Early diagnosis of rare diseases is a challenge owing to multiple factors that include lack of awareness among primary care physicians, lack of adequate screening and diagnostic facilities.

• So far, only a limited number of diseases has been recorded in India from tertiary care hospitals that are globally considered as rare diseases though ambit may encompass from 7,000 to 8,000 disorders. 

• The commonly reported diseases include Primary immunodeficiency disorders, Lysosomal storage disorders (Gaucher’s disease, Mucopolysaccharidoses, Pompe disease, fabry disease etc.) small molecule inborn errors of metabolism (Maple Syrup urine disease, organic acidemias etc.), Cystic Fibrosis, osteogenesis imperfecta, certain forms of muscular dystrophies and spinal muscular atrophy, etc.

• The government has launched National Policy for Rare Diseases (NPRD), 2021 in March 2021 for the treatment of rare disease patients.

Why are rare diseases called ‘orphan diseases’?

• As the number of persons suffering from individual rare diseases is small, they do not constitute a significant market for drug manufacturers to develop and bring to market drugs for them. 

• For this reason, rare diseases are also called “orphan diseases” and drugs to treat them are called “orphan drugs”. 

• At present, very few pharmaceutical companies are manufacturing drugs for rare diseases globally. Where they do make drugs to treat rare diseases, the prices are extremely high apparently to recoup the cost of research and development. 

• It is estimated that for a child weighing 10 kg, the annual cost of treatment for some rare diseases, may vary from Rs 10 lakh to more than Rs 1 crore per year with treatment being lifelong and drug dose and cost, increasing with age and weight.

Generic drugs

• A generic drug is a medication created to be the same as an already marketed brand-name drug in dosage form, safety, strength, route of administration, quality, performance characteristics, and intended use. 

• A generic medicine works in the same way and provides the same clinical benefit as the brand-name medicine. 

• Generic medicines have the same active ingredient as brand-name medicines and work in the same way.

• Generic alternatives are often cheaper than brand-name medicines. This is because the company that produces the medicine did not need to invest money in developing and marketing it.

• India is one of the largest suppliers of generic medicines. The country manufactures about 60,000 different generic brands across 60 therapeutic categories and accounts for 20 per cent of the global supply of generics.

• Indian pharmaceutical industry plays significant role globally, supplying affordable and low-cost generic drugs to millions of people across the globe.

• Clause 1.5 of the Indian Medical Council (Professional Conduct, Etiquette and Ethics) Regulations, 2002 prescribes that every physician should prescribe drugs with generic names legibly and preferably in capital letters and he/she shall ensure that there is rational prescription and use of drug.

• In order to make quality generic drugs available to citizens of the country at affordable prices, the department of pharmaceuticals has launched Pradhan Mantri Bhartiya Janaushadhi Pariyojana (PMBJP) wherein about 9,668 generic drugs stores have been opened all across the country till July 31, 2023.

• The government plans to increase the number of generic drugs stores to 10,000 by March 2024 in order to accelerate the drive to make quality medicines available at affordable prices to all.

What is PMBJP?

• Jan Aushadhi was launched by the government in 2008 for making quality medicines available at affordable prices for all. The campaign was undertaken through the sale of generic medicines through exclusive outlets namely Pradhan Mantri Janaushadhi Kendra in various districts of the country.

• It was later renamed as Pradhan Mantri Bhartiya Janaushadhi Pariyojana (PMBJP). 

• It aims to bring down the health care budget of citizens by providing quality generic medicines at affordable prices.

• The scheme is being implemented through the Pharmaceuticals & Medical Devices Bureau of India (PMBI), which is working under the administrative control of the department of pharmaceuticals, ministry of chemicals & fertilisers.

Objectives of the scheme:

• To popularise generic medicines among the masses and dispel the prevalent notion that low priced generic medicines are of inferior quality or are less effective.

• To make available quality medicines consumables and surgical items at affordable prices for all and thereby reduce out of pocket expenditure of consumers/patients.

• Generate employment by engaging individual entrepreneurs in the opening of PMBJP Kendras.

Other key points:

• The product basket of the scheme now covers 1,759 medicines and 280 surgical items covering all major therapeutic groups such as cardiovascular, anti-cancers, anti-diabetics, anti-infectives, anti-allergic, gastro-intestinal medicines and nutraceuticals, among others.

• The endeavour of the government is to enhance the product basket of generic medicines and surgicals. It is a continuous process based on feedback of various stakeholders and market demands.

• Under PMBJP, an incentive of Rs 5 lakh is provided to the Janaushadhi Kendras as financial assistance. 

• Also, a one-time additional incentive of Rs 2 lakh as reimbursement for IT and infra expenditure is provided to Janaushadhi Kendras opened in northeast states, Himalayan areas, island territories and backward areas mentioned as aspirational district by NITI Aayog or if opened by women entrepreneur, ex-serviceman, divyang, SC and ST.

• The medicines listed in the product list of PMBJP are procured only from World Health Organisation – Good Manufacturing Practices (WHO-GMP) certified suppliers for ensuring the quality of the products. Apart from this, each batch of the drug is tested at laboratories accredited by the ‘National Accreditation Board for Testing and Calibration Laboratories’ (NABL). Only after passing the quality tests, the medicines are dispatched to PMBJP Kendras.

• A medicine under PMBJP is priced on the principle of a maximum of 50 per cent of the average price of the top three branded medicines. Therefore, the price of Janaushadhi medicines is cheaper at least by 50 per cent and in some cases, by 80 per cent to 90 per cent of the market price of branded medicines.

• A mobile application ‘Janaushadhi Sugam’ has also been launched with user-friendly options like — locate nearby PMBJK (direction guided through Google Maps), search Janaushadhi medicines, etc.

Manorama Yearbook app is now available on Google Play Store and iOS App Store