A just released report from the American Cancer Society (ACS) has triggered for me, yet again, a concern: The American clinical research system is not designed to maximize the potential for the best possible outcomes of health services that are delivered to all American health care consumers.
According to the ACS Cancer Statistics 2012, cancer related deaths in the United States have declined considerably over the past two decades. Specifically, mortality rates have dropped 23% for males and 15% for females since 1990. This means that approximately 1 million lives have been saved.
Clearly, this trend is to be celebrated, and indicates that sound research and effective innovation are alive and well in America. Yet despite overall improvements in survivorship rates for the general population, the African American community continues to be disproportionately affected by cancer. Specifically, the ACS report reveals that, "For all cancer sites combined, African American men have a 15% higher incidence rate and a 33% higher death rate than white men, whereas African American women have a 6% lower incidence rate but a 16% higher death rate than white women." The report states further that, "Compared with whites, African American men and women have poorer survival once cancer is diagnosed. The 5-year relative survival is lower in African Americans than in whites for every stage of diagnosis for nearly every type of cancer."
Yes, the reasons may vary. There may be issues of financial or geographic access to diagnostic and treatment services. There may be challenges associated with compliance with treatment plans. There are also, is a very real and documented sense, differences in the way non-whites are diagnosed and treated once they present for care. Even more problematic, is the persistent gap that is due in part to lack of inclusiveness in clinical trials and other research that form the basis of diagnoses, of treatment guidelines, and the related prognoses.
My point is this. We will not learn what is possible in terms of treatment outcomes if we don't include all populations in a statistically valid way in all research. The continued norming of research, treatment and payment to traditionally insured populations is the disparity that must be eliminated if we are to improve the health of the American general population -- in other words, all of us. As we move into the implementation phases of health care reform, diversity in clinical trials must be given priority.
Why? Well, consider this. By 2020, 40% of the U.S. population will consist of racial and ethnic groups currently classified as "minorities." It is projected that this percentage will increase to more than 50% no later than 2050. Advances in genetics and biomedical technologies have created unprecedented opportunities to develop effective therapies that reduce the percentage of avoidable mortality and morbidity for larger numbers of American families and children. A major, but not insurmountable, barrier is the lack of reliable, cost-effective processes to facilitate the recruitment of diverse clinical trial cohorts. This infrastructure weakness has the potential to compromise the ability of the health care research, delivery and financing system to innovate -- to create new science, to translate existing science into effective therapies, or to identify new areas of inquiry.
Just as cancer is an example of a disease process that must be addressed through changes in diagnosis and treatment protocols, biologics and biosimilars are examples of treatment modalities for which efficacy and safety must be considered for all Americans.
Biologics are highly complex new medicines that effectively treat debilitating ailments such as cancer, arthritis, and multiple sclerosis. Because these medicines are made from living cells, they are highly sensitive to differences in the manufacturing process -- so sensitive in fact that they are impossible to copy exactly. However, copied, they are. These replications, because they cannot be exact copies, are called "biosimilars." Due to small yet significant differences in the composition of an innovator drug and its biosimilar, the use of biosimilars can lead to unexpected or even harmful effects, even in patients who have reacted positively to the innovator product. In short, biosimilars may sometimes work as well as the innovator drugs, but doctors and patients alike should be aware of the risks associated with substituting biosimilars for the innovator biologics.
The Affordable Care Act's authors, in recognition of the need to expedite the approval process for biosimilars, directed the Food and Drug Administration to create a pathway for the introduction of biosimilars into the market. Nearly two years after the passage of this landmark legislation, the FDA has indicated that it is nearing completion of its proposed pathway.
I hope that the FDA uses this opportunity to enact strong patient protections by requiring rigorous clinical trials for new biologics and for their biosimilars. Only if all consumer groups are represented in all phases of development of innovator, generic and biosimilar drugs will FDA and the American health care system overcome what appear to be intractable health status and health care challenges.