Addressing 'Orphaning' of Rare Diseases

To tackle any disease requires sustained research and development (R&D), to help us learn more about it and to create effective treatments. However, when it comes to R&D around rare diseases, there are few key challenges which ultimately impact patient outcomes.

- Ravi Shet

The most important challenge is how unevenly spread rare diseases are among populations. We don’t have a complete picture of how many people are affected by rare diseases in India, because most of the time these are not reported properly from the patient or their relatives. It becomes more difficult for the pharmaceutical companies to prioritise, since most of the rare diseases are clinically dissimilar and require unique treatments.

Another big challenge is funding to develop drugs for treating these rare diseases. Since it affects a small number of people in comparison with cancer, cardiovascular disease and diabetes, they are often considered low priority when it comes to allocating funds. Drugs needed to treat rare diseases are known as Orphan Drugs which essentially means that they treat medical conditions so rare that it would not be profitable to produce and develop them.