Approximately, 300 million people, around the world, are living with one or other 6,000 identified rare diseases. Countries worldwide follow different criteria to classify rare diseases. In the United States, a rare disease affects fewer than 200,000 people (Orphan Drug Act). While in the European Union, a rare disease is a disease that affects fewer than 1 in 2,000 people.
The rare disease possesses a significant challenge to the patient in terms of screening, diagnosis, and treatment availability. Similarly, at the medication and research front, the time and the cost of clinical trials discourage potential sponsors and pharma enterprises to invest in the rare disease segment.
However, today several pharma companies at the global level such as Celgene, Takeda, Eli Lilly and Company, Novartis, Bristol Myers Squibb, Alexion Pharmaceuticals, Sanofi, Vertex Pharmaceuticals, Roche, Bluebird Bio, Amgen, Biogen, and many others are working towards the development of novel approaches and novel orphan drugs for rare diseases to bring relief to the affected patients.
For more details, visit: Challenges in Rare Diseases Therapeutics Market