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Opinion

In Drug Development, It’s About Time

 

When considering the cost and value of innovative pharmaceuticals, one critical factor often overlooked is the impact of “time” at every stage of biopharmaceutical research. First, consider that biopharmaceutical research most often evolves in three stages. Stage one is when scientists discover and develop a molecule. Companies spend billions of dollars comparing the novel medicine to the standard course of treatment(s).

At times they rely on surrogate clinical end points to demonstrate safety and efficacy. Stage two is the investigation of patient outcomes, surely the holy grail of biopharmaceutical research. Whether through cancer free survival for oncolytic biopharmaceuticals or reduction in cardiovascular mortality or morbidity due to management of hypertension or cholesterol, researchers are asked to confirm that indeed the surrogate clinical end points provide meaningful changes in patients’ lives. Finally, stage three is when researchers are able to observe and appraise long term “value.” Value is not only defined as a medicine having long term positive economic consequences, but also social and human benefits, such as improvement in the quality of life or productivity of a patient.

To execute all of these stages appropriately, biopharmaceutical companies invest a staggering amount of monetary as well as human resources – far greater than the $50 billion plus PhRMA (Pharmaceutical Research and Manufacturers of America) member companies invest annually in pre-approval research and development. Nevertheless, the most daunting barrier to appropriately implementing all three stages of biopharmaceutical research is “time.”

“Time” is a finite commodity in the biopharmaceutical ecosystem. It is defined by patent life and, as such, there is only a limited opportunity for a medicine to realize a financial return that will fund future research opportunities.

Beyond the critical need to re-invest in future cures, another “time” barrier to appropriate evaluation of the value proposition of a biopharmaceutical is the fact that overall societal value for innovative medicines is not captured until years after approval of the medicine. Take for instance the HMG-CoA reductase inhibitor medicines better known as statins. It is only now; years after most statins have lost exclusivity and are now generic, that we truly realize their value.

For example, in a study published in 2012, a year after the most prescribed statin – atorvastatin – lost its exclusivity; researchers concluded that statin therapy reduced low-density lipoprotein levels by 18.8 percent, which translated into roughly 40,000 fewer deaths, 60,000 fewer hospitalizations for heart attacks, and 22,000 fewer hospitalizations for strokes in 2008. In addition, in the same study, the authors estimated that from 1987 to 2008, the aggregate social value of statins was estimated to be $1.252 trillion. The same holds true for oncolytic medicines.

For example, it was only a little more than a decade ago, before the advent of tyrosine kinase inhibitors, that the median survival time for CML (Chronic Myelogenous Leukemia) patients was about 3–5 years from time of diagnosis. Today, according to one study published in 2011, patients on imatinib (one of the first tyrosine kinase inhibitors developed) who achieved a stable cytogenetic response have an overall survival rate of 95.2 percent after 8 years, which is similar to the rate of life expectancy in the general population.

These types of results have tremendous impact on the practice of medicine and society, as healthcare professionals now have the remarkable opportunity to manage the long term health of once morbid patients as they get older and live longer and healthier lives.  Society also benefits from the tremendous value provided by these patients who are more productive and are able to repay the benefits of treatment from innovative biopharmaceuticals many times over as these therapies continue to add value in perpetuity.

Beyond “time,” another barrier that prohibits us from appropriately evaluating value is access to outcomes data and parameters of valuation. For example, the American Cancer Society identifies survival rates from non-small cell lung cancer by stage from data gathered by National Cancer Institute’s SEER database from 1998 – 2000. ACS states that, “[A]lthough they (the data) are based on people diagnosed several years ago, they are the most recent rates published for the current AJCC staging system.” So, in an era of Twitter and Snapchat, where we have real time access to statistics regarding our social media activity, we still rely on information close to two decades old to determine the prognosis of our most precious commodity – our health.

No one disagrees that we have a moral obligation to evaluate the price and cost of innovative biopharmaceuticals in the sphere of overall costs. We all know that a small number of patients are under tremendous financial pressure like never before. However, we need to put the price tag of medicines within the larger context of long term savings as well as economic and social value. We need to consider the barriers of time and data. We need to work together with policy makers and payers on new payment approaches that closely tie the short term cost of medicines to the long term value they deliver years later.

Robert Popovian is the Senior Director of Pfizer US Government Relations.