Clinical trials in rare diseases are more challenging than clinical trials in more frequent diseases for many factors such as the trial design, the number of patient populations available for recruitment, require regulatory support, etc. Applying a perfect clinical development strategy may help in overcoming these challenges and accelerate the drug development process in Orphan […]

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For the approximately 7,000 known rare diseases, less than 10% of diseases have an FDA-approved treatment available. Due to a small number of patients, limited understanding of the variability and progression of the diseases, developing a drug or treatment for rare diseases is often challenging. Rare diseases currently affect 3.5% – 5.9% of the worldwide […]

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Many people with rare diseases struggle to find the right diagnosis and right treatment for years. The lack of awareness about these conditions make it much harder for them to get the right treatment. Rare Disease Day is all about raising awareness for rare diseases. And, this year, you can join these efforts too. The […]

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In recent years there is a huge leap in the pharma industry towards rare diseases and orphan drug developments due to the great support from regulatory authorities such as faster approval timelines, supportive regulatory frameworks, financial incentives, and many more. Currently, large pharmaceutical companies are extremely active in the orphan drugs market. Novartis, GSK, Roche, […]

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In a way to support and encourage pharma companies to work upon developing drugs or medical devices in rare disease, several country regulatory agencies are coming up with various benefits such as faster approvals, fee concessions and financial benefits etc. These countries include the US, Europe, Japan, Australia, South Korea and most recently, India. Incentives […]

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Financial benefits, faster approvals, and less stringent clinical data requirements are some significant benefits that regulatory agencies across the world offer as incentives for the development of orphan drugs for rare diseases. These countries include the US, Europe, Japan, Australia, South Korea, Brazil, and most recently, India. These incentives have been so supportive and convincingly […]

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Rare disease drug development is gaining interest in pharma industries. Large pharmaceutical companies have been working for many years, and now startups, small and mid-range pharma companies are also gaining traction in rare diseases drug development.  Developing medicines or drug products in rare diseases is always challenging for both the scientific and clinical development team […]

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The development of drugs in rare diseases provides great opportunities to pharma companies along with challenges that are required to overcome during drug discovery, pre-clinical, and clinical stages of drug development. Commercially rare diseases drug development differs from traditional diseases, as there are considerably few patients to take the drug, therefore pharmaceutical companies should consider […]

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Rare disease drug development is a rapidly expanding field with growing pressure for getting a more orphan drugs approved to treat rare diseases. There is a vast consensus among patients, health care bodies, governments, etc. to expedite drug development in this field. Some wonderful numbers that attest to growing interest in rare diseases and orphan […]

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