An interview with Daryl Pritchard, PhD, vice president for Science Policy at the Personalized Medicine Coalition, on the FDA's drug approval process, developments in the personalized medicine field, and reimbursement models that integrate diagnostics.
Evidence-Based Oncology™ (EBO™) reached out to Daryl Pritchard, PhD, vice president for Science Policy at the Personalized Medicine Coalition (PMC), an organization created “to promote the understanding and adoption of personalized medicine concepts, services, and products.”1 PMC’s membership boasts a broad spectrum of academic, industrial, patient, and healthcare provider constituencies, and they are very active with educational and advocacy efforts that will draw attention to the advantages of personalized medicine in healthcare.
EBO™: What are your thoughts on the existing FDA drug approval process? Are there ways in which the process can be improved?
PRITCHARD: Scientific progress in personalized medicine is driving an increase in the number of personalized medicine products that are subject to regulatory review. In fact, nearly 1 out of every 4 drugs that were FDA approved between 2014 and 2016 was a personalized medicine. This is a sharp increase in personalized medicine drug approvals. In 2005, personalized medicines accounted for just 5% of new drug approvals. The FDA has responded to the growing demand for regulatory clarity by issuing guidance documents, most notably on principles of co-development of therapeutics with companion diagnostics.
The Center for Drug Evaluation and Research (CDER) within the FDA has indicated strong support for a personalized medicine approach to drug development. In her FDA Voice blog, Janet Woodcock, MD, wrote, “CDER uses a lot of flexibility when reviewing applications for targeted drugs. For example, among the targeted therapies approved in recent years, 90% used 1 or more of FDA’s expedited programs such as Breakthrough, Fast Track, Priority Review and Accelerated Approval.”2
Although regulatory oversight of targeted therapeutics has done a great job keeping up with the science, regulatory oversight for diagnostic tests remains problematic. There are currently 2 separate regulatory pathways for different kinds of diagnostic tests. In vitro diagnostic testing kits are regulated by FDA, while laboratory developed tests are subject to oversight through CMS’ Clinical Laboratory Improvement Amendments, commonly called CLIA. This has caused different stakeholders to raise issues about consistency, transparency, and the creation of an uneven playing field in the development of advanced diagnostics.
EBO™: A companion diagnostic for every drug product on the market would be an ideal situation. But is that feasible, at least in oncology?
PRITCHARD: As the science regarding individual variability in drug response continues to unfold, more and more drugs will be associated with companion diagnostics. It would be ideal if one day doctors and pharmacists had the complete genetic profile for all patients that could be easily used to guide treatment decisions by allowing them to consider all known genetic variants that help predict which drug would be most effective or which drugs would likely cause adverse side effects. However, the scientific process for understanding the complete range of the impact of genetic variability in drug response is not feasible for a very long time. At first, scientists will likely work on understanding genetic variability that has the greatest impact on drug response for diseases with clear molecular mechanisms, and we are seeing a great deal of that research in oncology. The key is to add novel diagnostics, as they are developed, to the expanding toolbox for personalized medicine as quickly as is appropriate.
EBO™: Can you describe PMC’s policy efforts to improve clinical acceptance of genetic testing?
PRITCHARD: PMC is engaged in various policy efforts to accelerate clinical acceptance of genetic testing, including:
EBO™: How can reimbursement for diagnostic tests be improved? What are your thoughts on existing policies by CMS and private health plans?
PRITCHARD: Reimbursement can be improved by establishing payment rates for diagnostic tests that appropriately reflect the value they bring to care.
Many coding and payment policy policies used by both CMS and private payers have led to significant challenges regarding reimbursement for molecular diagnostic tests. CMS’ decision, for example, to use the gap-fill methodology, which allows regional contractors to set prices for laboratory and molecular diagnostic tests, coupled with other payment decisions, has resulted in decreased payment rates for many personalized medicine tests. This, in turn, has placed a consistent downward pressure on physicians and laboratories interested in using novel, high-value molecular diagnostics to inform treatment decisions.
Coverage and payment rate determinations by private payers lack consistency and transparency. Lack of coverage and under-reimbursement threatens access and further exacerbates the downward pressure on utilization of these technologies. Decision-making processes lack mechanisms that capture the value of targeted treatment, and may therefore threaten progress.
EBO™: Oncology trials such as TAPUR and NCI-MATCH have a very different approach to trial design. They have even seen collaboration among pharmaceutical companies. Do you see this as the new standard?
PRITCHARD: In oncology, basket trials, which enroll patients with cancers that originated in all different types of tissue so that researchers can study treatment effects based on molecular pathways, are rapidly becoming more routine. This trial design allows for more rapid examination of a targeted drug’s efficacy across tumor types and could lead to more effective targeted therapies being approved at one time for use in multiple cancer types. It’s a more sensible way to examine drugs that target molecular pathways, and we believe this will become the standard trial approach for these types of drugs.
ABOUT THE EXPERT
Daryl Pritchard, PhD, is vice president for Science Policy at the Personalized Medicine Coalition (PMC).
PMC is very active with educational and advocacy efforts to promote the understanding and adoption of personalized medicine concepts, services, and products.
1. Mission. Personalized Medicine Coalition website. http://www.personalizedmedicinecoalition.org/About_Us/About_PMC. Accessed March 31, 2017.
2. Woodcock J. FDA continues to lead in precision medicine. FDA website. https://blogs.fda. gov/fdavoice/index.php/2015/03/fda-continues-to-lead-in-precision-medicine/. Published March 23, 2015. Accessed March 30, 2017.