By Mark Crawford
Pharmaceutical priorities shift as diseases strengthen, evolve, spread, and make headlines. Drug developers must balance social need with R&D funding and maintaining an income stream. Also, with the passage of the Affordable Care Act, reimbursements are now tied directly to quality of care, instead of fee for service. Therefore health care organizations are eager (and willing to pay for) pharmaceutical products that can dramatically improve the care they provide—for example, drugs that can knock out superbugs like MRSA.
When it takes 10 years and over $1 billion to get a new drug into the marketplace, pharmaceutical companies want to provide solutions that positively impact large numbers of people around the globe—not just to achieve the greatest possible good, but also to generate a healthy return on those investment dollars. As a result, serious and often deadly diseases that affect only small numbers of the population are often overlooked as targets—which is why many orphan diseases are still orphans.
This, however, creates a disparity in the drug pipeline. According to an article by Kenneth Kaitin, Ph.D., director of the Tufts Center for the Study of Drug Development in Boston, and Christopher-Paul Milne, D.V.M., the center’s director of research, that was recently published on PharmExec.com, 30 percent of the drug pipeline is dominated by oncology—which they say is both economically and medically out of balance.
“While cancer is certainly a major health problem, it is neither the nation’s number one health concern, nor the most urgent in terms of unmet medical needs,” they write. In contrast, the number of new molecular entities (NMEs) for infection, a top therapeutic area that represents notable unmet need, dropped significantly, even though “infection through drug-resistant pathogens and newly emergent infections such as SARS and Ebola is the No. 1 threat with the potential to impact a sizeable portion of the U.S. population,” they add. New NMEs for central nervous system disorders have remained flat—part of “a concerning trend,” they note.
This skewed focus means fewer research dollars are available for understanding and researching other serious diseases, which are far less understood than cancer. “For example, with ALS [amyotrophic lateral sclerosis] you are dealing with complex biology with lots of unknowns, a heterogenous patient population, challenges related to therapeutic delivery to the central nervous system, and finally challenges related to patient population size and major challenges around the design of clinical trials,” says Fernando Veira, M.D., director of research at the ALS Therapy Development Institute in Cambridge, Massachusetts. With no diagnostic or prognostic biomarkers, this heterogeneity makes clinical trials difficult to design and interpret—something pharmaceutical companies prefer to avoid.
The cover article in the June AAPS Newsmagazine reviews four key areas of unmet medical need: severe neurodegenerative diseases/ALS, antibiotic resistance, viral diseases, and chronic pain. What are the challenges? What have been the achievements? And what will the future bring? Read Unmet Medical Needs Continue to Challenge Pharmaceutical Science and then participate in the discussion point below.
In view of these and other unmet medical needs, what should our R&D priorities be?