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. As the disease is rare, finding sites to conduct clinical […]

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Most of the companies are spending a significant amount of cost and manpower to manage clinical trial activities as database building, monitoring, and management of data using a variety of EDC systems as Inform, Rave, Oracle Clinical Remote Data Capture, Veracity, etc. Many software, web-based applications, and cloud source management tools are being used in […]

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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 makes 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 disease research 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 like Novartis, GSK, Roche, and Pfizer are extremely active in […]

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Regulatory agencies of various countries are providing benefits & regulatory incentives for orphan drugs such as faster approvals, fee concessions, and financial benefits, and many more. These incentives are to support and encourage pharma companies to work upon developing orphan drugs or medical devices for rare diseases. The countries that provide regulatory incentives include the […]

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Many regulatory agencies across the world are offering incentives for the development of orphan drugs for rare diseases. These incentives include financial benefits, faster approvals, less stringent clinical data requirements, and many more. The countries that are offering the regulatory incentives for orphan drugs include the US, Europe, Japan, Australia, South Korea, Brazil, and most […]

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Clinical supply chain teams manage some typical challenges almost every day of their work in handling clinical trial logistics for local or global clinical trials. These include those related to regulatory compliance, temperature control (or, excursions), labeling, actual shipment, or last-mile delivery. These challenges are often compounded multi-fold when clinical trials are related to a […]

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Rare diseases are rapidly becoming a high priority therapeutic area for our industry. This is needed, especially when fewer than 5% of approx 7,000 rare diseases have drug therapies available, and many of these provide limited benefit. There is also a business case to this, due to a supportive reimbursement environment, particularly in the US. […]

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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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Over a few years, the rate of drug development in rare diseases is increasing, particularly in those countries where the regulators are offering various incentives for drug development in rare diseases. Over the past five years, one-third of the approved drugs were to treat rare diseases. The huge leap of the pharma industry into the […]

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