The U.S. Active Pharmaceutical Ingredients (API) market is set to expand from $67.11 billion in 2024 to $117.86 billion by ...

According to DataM Intelligence, the Orphan Drugs Market Size was USD 223.76 billion in 2023 and is forecasted to soar to USD 486.51 billion by 2032, expanding at a strong CAGR of 9.1% between 2024 ...

The Centers for Medicare and Medicaid Innovation (CMMI) plans to use outcomes-aligned payments (OAPs) to cover the cost of ...

In this podcast episode, we discuss the journey of novel modalities in rare disease treatment to patients, particularly advancements in oncology and the increasing role of patients' families in ...

The Orphan Drug Act has catalyzed innovation in rare disease treatments, but the law’s shortcomings — particularly around ...

Massimo Radaelli, PhD, is a European pharmaceutical industry leader and entrepreneur who has devoted more than 30 years to the innovation of therapies to treat rare diseases. He is President and CEO ...

Usually, bad news gets all the airtime, but this summer brought us a hopeful headline: A new innovation hub will accelerate the development of treatments and products for rare diseases. I have been ...

A recent WHO report outlined concerning changes in the antibacterial pipeline — namely, that the pipeline is shrinking and ...

Your editorial on “Why Vinay Prasad Had to Go at FDA” should be a wake-up call for anyone who cares whether federal bureaucrats get to decide if promising new medicines are available to desperately ...

The National Organization for Rare Disorders (NORD(R)) today urged Congress to reauthorize the Rare Pediatric Disease Priority Review Voucher (RPD PRV) program by year-end, warning that continued ...

Some of the most ambitious cancer science is happening in a disease few outside of oncology can name, and it’s revealing a future where cancer is no longer a death sentence. Multiple myeloma (MM) is a ...