Regulatory and Economic Aspects of Precision Medicine
The final session of the meeting focused on the regulatory and economic aspects of precision medicine in oncology. Dr. Andrew Stainthorpe, Director of the National Institute for Health and Care Excellence (NICE) program that receives and reviews Patient Access Schemes into NICE Technology Appraisal and the program that assesses new interventional procedures and devices for use in the UK National Health Service, reviewed the regulatory issues of companion biomarker tests in the United States and the European Union. In the United States, although the FDA has announced plans to expand its regulation to all genetic tests, this expansion has yet to take place. In the interim, the FDA continues to regulate test kits, and has begun to regulate genomics tools in clinical research. In the European Union, different countries have individual regulations about genomic testing, with the United Kingdom and Germany leading the way in evaluating not only clinical application but also cost-effectiveness. Dr. Stainthorpe then reviewed the current status of a number of biomarkers across a broad variety of tumors, when the biomarker tests should and should not be used, who should pay for the testing, and whether there is a reasonable way to decide who should pay. He ended his talk with a controversial question: “Is there a need for biomarker tests to be regulated or should access be unlimited and uncontrolled?” Dr. Stainthorpe’s presentation was followed by Dr. Michael Kolodziej, the National Medical Director, Oncology Solutions, Aetna. Dr. Kolodziej started his presentation by showing that based on the growth and aging of the US population, medical expenditures for cancer in 2020 are projected to reach at least $158 billion, a 27% increase over 2010, which is 2 to 3 times the rate of increase of other medical costs. This requires payers to assess genomic biomarker testing for their accuracy, clinical relevance, and economic value (which, in practical terms, constitutes an assessment of cost-savings and/or cost-effectiveness). The latter issue was highlighted in an example he provided of nivolumab therapy in lung cancer, where in the absence of a molecular biomarker to drive therapy for the most appropriate patients, nearly $20 billion could be spent on futile and potentially harmful therapy in patients who would not benefit from this drug and may be harmed by its adverse effects. The solutions he offered to this conundrum included collecting more data on clinical utility, optimizing patient outcomes and experience, and focusing providers on cost and value. “Personalized, precision medicine may offer a solution.” Following this session, Congress Co-chair Dr. Jorge Cortes provided closing remarks on another stimulating and informative meeting that had fostered debate and discussion, with interaction among basic scientists, clinicians, and other healthcare providers from various disciplines. Participants were then afforded a final networking opportunity to engage faculty in a “Meet the Experts” reception, to discuss issues on molecular biomarkers and personalized medicine in oncology and to visit the exhibit booths from Congress supporters.