The Karnataka Vision Group on Biotechnology has now put in place the draft of the Rare Diseases and Orphan Drugs Development Act. Efforts are on to enforce the Act by the year end.

“There is need for a dedicated Act considering the burden of the rare diseases impacting Indian population. There is a huge financial burden on the families of the patients who succumb to the condition for poor access to care. Therefore we were devising ways to support this segment of the population affected with rare diseases”, Kiran Mazumdar-Shaw, head, Vision Group on Biotechnology, government of Karnataka and chairman-managing director, Biocon Limited told Pharmabiz on the sidelines of the 15th edition of Bangalore India Bio.

Rare diseases cover lysosomal storage disorder (LSD) and a group of over 45 rare genetic disorders covering Gaucher disease, MPS I, MPS II, pompe disease, fabry disease and MPS VI occurring due to the deficiency of specific enzymes in special compartments or lysosomes of cells. Every one out of 20 people are known to report LSD. Karnataka has around 50 known patients suffering from this condition according to Lysosomal Storage Disorder Support Society (LSDS).

“There is need to strengthen diagnostics and look at newer therapies to provide relief to the scores of patients suffering from rare diseases. The Vision Group which is manned by industry and academia has research experts who have been involved in the development of the Rare Diseases and Orphan Drugs Development Act to ensure that the country takes the first step in this direction to put in a system for drug access and early detection,” said the Vision Group chief.

Further, there is need to provide access to the available treatment option: ERT or enzyme replacement therapy which is the lone medication to pause the progression of these genetic diseases, she said.

India needs an orphan drug status in India for ERT to enable medication is provided. With an Act in place, it could streamline the process of diagnostics and drug accessibility. The problem of low awareness even among the medical professionals could be improved with the general physicians to enable early detection. Moreover, it would also create a registry of rare diseases for bio-pharma researchers to assess the condition and treatment options further, she said.

We are first looking to passing this Act before the year-end in the state legislature. There is no need for comments from the industry on its draft because the Vision Group has a team of experts who have comprehended the criticality of the condition, said Shaw.

In an earlier interaction, the LSDSS indicated that to treat paediatric population, the annual drug costs would be Rs.40,000 to Rs.1.3 crore and was dependent on the patient’s weight.

Out of the 7,000 rare diseases, 500 have a drug and the remaining have no medication but depend on supportive therapy. Therefore the Rare Diseases and Orphan Drugs Development Act would provide opportunities for global companies like Genzyme, Shire and BioMarin engaged in the research and production of the drug to consider Indian bio-pharma companies to manufacture the same. This would drastically bring down cost of the drugs and enable easy access to therapy, said Prasanna Kumar B Shirol, founder director, Organization for Rare Diseases India.

Startup companies like for instance Aten Biotech in the Bommasandra Industrial Area, Bengaluru engaged in research of a gene therapy rare diseases, could also immensely benefit. Moreover, it would allow the government to create a fund for treatment, added Shirol.