Policy makers, awash with complex new medical information, face daunting challenges.
OUR SOLUTION: Provide them with evidence-based recommendations that prioritize patient health, safety, and access to treatments and diagnostic tests.
How can we better advance, approve, and monitor medical products?
The development and ultimate approval of new drugs — and the monitoring of drugs after they’re on pharmacy shelves — involves intense scrutiny and regulation by the U.S. Food and Drug Administration (FDA). This is necessary to ensure that medications are safe and effective. Developing the rules and tools to do this is a science in itself — a science that Kathy Giacomini, PhD, aims to improve as co-director of the UCSF-Stanford Center of Excellence in Regulatory Science and Innovation (CERSI).
Giacomini is now leading a major research study looking at 400 of the many inactive ingredients in generic tablets, capsules, and syrups taken orally — ingredients such as coatings, binding agents, and thickeners — that, with the active ingredients, travel to patients’ intestines. There, the active ingredient is absorbed into the bloodstream by intestinal transporters. Do the inactive ingredients, which can make up 90 percent of a drug formulation, hinder how well their active companions are absorbed? She intends to find out. Her results may help the FDA better regulate any inactive ingredients that hinder absorption and ensure that generic drugs used by patients have equivalent biological effects, dose by dose, to their brand-name counterparts.
How can we drive health policy so that genetic testing and the information it reveals benefit patients?
Genetic tests on the market today hold the promise of health care tailored to each patient’s unique biology and situation. But Kathryn Phillips, PhD, knows there’s a wide gap between this promise and a patient’s ability to benefit from it. She’s methodically narrowing that gap by asking hard questions and revealing critical answers, in her role as director of the Center for Translational and Policy Research on Personalized Medicine (TRANSPERS). Her team there recently found that the use of, and insurance coverage for, genomic tests for developmental disabilities such as autism, vary greatly. Instead of benefiting from diagnoses based on genomic testing, many of the one in six children in the U.S. who are developmentally disabled end up on diagnostic odysseys lasting many years. Findings like this — which Phillips and her fellow TRANSPERS researchers then share with policymakers — are essential if personalized medicine is to benefit all patients equitably.
Continue the journey to better medicines:
OUR SOLUTION: Explore molecular targets in daring new ways.medium.com
OUR SOLUTION: Get drugs to exactly where they’re needed.medium.com
OUR SOLUTION: Take into account all the factors that can influence a drug’s effectiveness for each individual — from genetic heritage and lifestyle to age and weight.medium.com
OUR SOLUTION: Start incrementally and locally — then apply the best solutions broadly.medium.com
Contributors: Grant Burningham; Levi Gadye, PhD; Paula Joyce; and Susan Levings