There are currently 907 drugs and vaccines targeting more than 100 diseases in development by U.S. biopharmaceutical companies. It is the first step on a path toward bringing much needed treatments to patients. However, with the current focus on the cost of health care, regulatory approval is not the final hurdle. Increasingly, the ability of patients to access new therapies is determined by those who pay for the treatments.
Skyrocketing development costs, concerns about the affordability of treatments, and access to treatments have become the most challenging issues facing the industry today. Our future health care problems will not be solved simply by reducing costs. New models are needed if we are to meet these challenges.
A solution is on the horizon. Scientific advances are increasing our ability to demonstrate the value we create as measured by improved patient outcomes. It starts with a better understanding of the biology behind different diseases. With this, we are able to define subsets of patients sharing a certain biology which may increase their probability of responding to a specific therapy. This is the essence of personalized medicine: developing the right treatments for the right patients, and delivering it at the right time.
To spur advancement in personalized medicine it is important that we continue to invest in not only developing new treatments, but in deepening our understanding of the diseases that we are trying to treat. We must improve our ability to document the value of any therapeutic intervention. For example, we must be able to describe health improvements which translate to direct savings for the health care system. We also need to collect data on the improvements to the quality of life of patients, such as allowing an individual to return to work, and/or lower the burden on caregivers, allowing the caregiver to return to work. These are all effects which create value, not only for the individual, but for society as a whole. Patients’ viewpoints must be central in assessing the value of a new treatment; their role defines the health care outcomes which are most meaningful to them.
Finally, it is essential to engage all stakeholders of the health care ecosystem to ensure that patients benefit from the treatments we develop. A onedimensional focus on costs will stifle innovation.
David Meeker is President and CEO of Genzyme Corporation. He chaired the How Do We Determine and Pay for Value? Does the Patient Have a Voice? session on Monday at the 2013 BIO International Convention.