Drug development and working with specialised Rare Disease networks

There are currently an estimated 300 million people living with one or more Rare Diseases across the world. There are over 7,000 Rare Diseases that have been identified, yet only 5% have treatments.

However, despite the work still left to be done, there are now a growing number of treatments in the pipeline and 2020 proved to be a remarkable year for orphan drug approvals, with the US FDA approving more orphan designated treatments than non-orphan drugs.

In a recent webinar held by pharmaphorum, in partnership with IQVIA, a discussion was held looking at how to make academic and commercial medical research more efficient, while also looking at the opportunity of genomic data to bring benefits to patients and healthcare systems.

Dr Jeff Keefer, VP and Head of the Pediatric and Rare Disease Center of Excellence at IQVIA, was one of the three panellists present at the webinar, and he stated that what makes the space unique is the “strength and depth of the relationships that form within the Rare Disease community.”

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BioPharma Global is a mission-driven corporation, operating like a not-for-profit, dedicated to using our FDA and EMA regulatory expertise and knowledge of various therapeutic areas to help drug developers advance treatments for the disease communities with a high unmet medical need. If you are a drug developer seeking regulatory support for Orphan Drug designation, Fast Track designation, Breakthrough Therapy designation, other FDA/EMA expedited programs, type A, B (pre-IND, EOPs), or C meeting assistance, or IND filings, the BioPharma Global team can help. Contact us today to arrange a 30-minute introductory call.

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