The global rare disease drugs market is one that is driven by innovation which is vital for maintaining the rich pipeline of rare diseases drugs seen in the market today. There is an increasing focus to identify and treat rare diseases that currently are undertreated which creates a strong platform for both start-ups and larger pharmaceutical companies to create new drugs that are not already on the market.
The lead analyst of the report commented "It is difficult to investigate rare diseases as the pool of patients is very small and often results in inadequate clinical knowledge. As a result, rare diseases lack published data on long-term outcomes of treatment and are often characterized incompletely. This requires exploring international and regional research partnerships, collaborating with doctors working on any rare disease, and with patient groups and families dealing with the consequences of these disorders."
Leading companies featured in the report AbbVie, Bristol-Myers Squibb (BMS), Celgene, Merck & Co., Inc., Novartis, Pfizer, Roche, Sanofi, Takeda, Teva and other companies.