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The U.S. Food and Drug Administration today issued a revised draft guidance, Rare Pediatric Disease Priority Review Vouchers – Guidance for Industry. The revised draft guidance incorporates public comments received on the initial draft and provides FDA’s thinking regarding the provisions of the Advancing Hope Act of 2016, which updated the definition of a rare pediatrics disease as one that is a rare disease and one where the disease is serious or life-threatening with the serious or life-threatening manifestations primarily affecting individuals from age zero to 18.

The US Food and Drug Administration (FDA) recently released guidance to help companies develop new treatments for epidermolysis bullosa (EB), which is a debilitating and often fatal rare disease. The six-page draft guidance offers details on considerations for clinical trial design, including trial population (estimates suggest 25,000-50,000 people in the US have EB), discussion of efficacy endpoints (i.e. effects on patients’ signs or symptoms such as itching, pain, blister prevention and wound healing) and special considerations to maximize patient comfort (i.e. allowing photo or video documentation of wounds during routine dressing changes in the home).

Just moments after it was announced the FDA had approved a new treatment for spinal muscular atrophy, Dr. Peter Bach was warning about a “break down” in the U.S. drug pricing system. It’s unfortunate Dr. Bach would respond to the discovery of a miracle cure for a fatal disease with such doom and gloom. In stark contrast to a patient community who welcomed the “great news” as “an amazing advancement,” Dr. Bach described the search for new treatments for rare diseases as a “problem” that needs to be addressed. But his dire concerns are misplaced for a host of reasons. There is plenty of room for innovative treatments in our health care system. Dr. Bach has been part of the chorus of those warning about “skyrocketing” drug costs for some time, but the facts usually tell a different story. According to the Centers for Medicare & Medicaid Services, spending on retail prescription drugs increased just 0.4% in 2017, which is slower than the growth in spending on hospitals (4.6%), physician services (4.2%) and health care more generally (3.9%).

The Biotechnology Innovation Organization (BIO) released the 2019 Emerging Therapeutic Company Trend Report, highlighting ten years (2009-2018) of biotechnology funding and deal making across five areas: venture capital, Initial Public Offerings (IPOs), follow-on public offerings, licensing, and acquisitions. The report also contains a 2019 snapshot of the industry’s clinical pipeline to highlight the significant contribution of emerging companies.

Shepherding a drug from discovery to the market is a complex process that involves many actors. The process often begins with academic researchers making a breakthrough discovery in the lab and ends with pharmaceutical companies running large-scale clinical trials to demonstrate that the drug is safe and effective. But what about the middle of this process—the point at which the discovery is translated into something that could be meaningful for treating patients? Who is responsible for the translational, or preclinical, part of the process? There is a lack of clarity about who should assume that role; translational research is often too expensive for academics to perform by themselves, but it is too risky for pharmaceutical companies given the uncertainty about the discovery’s safety and market worthiness.