Why is the World of Orphan Haemato-Oncology Agents Changing?

As an increasing number of targeted oncology and haematology products are getting approved by EMA and FDA, the orphan space is becoming more and more crowded in these therapeutic areas.

Patients with a growing number of rare orphan diseases are thankfully seeing their lifespans and quality of life improved, in some cases dramatically. This phenomenon is especially due to the advent of targeted new medicines. Our expanding knowledge of biology and medicine means that there will undoubtedly be many more new products coming through development for rare cancers, and their sponsors will want to have these designated as Orphan Drugs.

The increasingly crowded Haematology and Oncology Orphan space has major implications for the development and registration of these products, as well as for the incentives provided by the Regulators, particularly EMA and FDA, for Orphan Drugs. Market Access is also becoming an increasing challenge.