ORPHAN DRUGS AND CHALLENGES

Описание: Complete information about Orphan Drugs and challenges faced by the world and India.

Orphan drugs are medicinal products intended for diagnosis, prevention or treatment of life-threatening or very serious diseases or disorders that are rare. In USA, the Orphan Drug Act was passed in 1983 to give drug companies incentives to develop treatments for rare diseases. India does not have laws on orphan drugs though CDSCO defined orphan drugs as those intended to treat a condition which affects fewer than 200,000 people.

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It is estimated that globally around 6000 to 8000 rare diseases exist with new rare diseases being reported in the medical literature regularly. However, 80% of all rare disease patients are affected by approximately 350 rare diseases.

India, like many other developing countries, currently has no standard definition of rare diseases and data on prevalence. Since there is no epidemiological data, there are no figures on burden of rare diseases and morbidity and mortality associated with them. If we apply the international estimate of 6% to 8% of population being affected by rare diseases, to India, we have between 72 to 96 million people affected by rare diseases in the country, which is a significant number. However, this is at best a general estimate and India will need to arrive at its own estimate and definition of rare diseases, derived chiefly from prevalence data, which is currently lacking. So far only about 450 rare diseases have been recorded in India from tertiary care hospitals.

The most common rare diseases include Haemophilia, Thalassemia, Sickle-cell Anaemia and Primary Immuno Deficiency in children, auto-immune diseases, Lysosomal storage disorders such as Pompe disease, Hirschsprung disease, Gaucher’s disease, Cystic Fibrosis, Hemangiomas and certain forms of muscular dystrophies.

Lack of epidemiological data
Data on how many people suffer from different rare diseases in India is lacking. The cases identified so far have been diagnosed at tertiary hospitals. The lack of epidemiological data on incidence and prevalence of rare diseases impedes understanding of the extent of the burden of rare diseases and development of a definition. It also hampers efforts to arrive at correct estimation of the number of persons suffering from these diseases and describe their associated

Skyscraping Pricing – Increasing opportunities
Every time, high drug prices are not due to patent of medicines. Just remember Infamous Martin Shkreli, who purchased Turing Pharmaceuticals which is involved in production of Daraprim and subsequently hiked price of medicine by 5500 percent. The world took note on that price hike. It became a hike controversy when Turing raised the price of a dose of Daraprim in the U.S. market from US$13.50 to US$750.

Patients whose liver does not produce one of the two primary bile acids and results in a critical condition that causes neurological problems, seizures and even dementia. For the past half century, it was relatively easy to treat such patients at a cost of €500 per annum by using Chenofalk (chenodeoxycholic acid), an off-label drug. In 2008, Leadiant acquired the rights to Chenofalk, and developed its own version, known as CDCA Leadiant. Then, very smartly in 2014, it got the EU to classify its version as an “orphan drug”, giving Leadiant exclusive marketing rights for 10 years. Immediately thereafter, it jacked up the price of CDCA Leadiant to about €150,000 for a year’s treatment.

Cydan, which helps launch startups focused on orphan-drug development, have decided to steer clear of them. That strategy is beginning to pay off. According to Adams, the company has successfully spun out three startups. The first of these, Vtesse, launched in January 2015 to develop a treatment for Niemann-Pick type C, a rare lysosomal storage disorder, using sugar molecules called cyclodextrins. It was acquired by Sucampo Pharmaceuticals for $200 million last April 2017.

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