The Precision Medicine Initiative and Access

By Leslie Francis

Persistent differences in participation in clinical trials by race and ethnicity are well known; for example, the 2015 Report of the Working Group on Precision Medicine (PMI) relies on statistics that only 5% of clinical trial participants are African-American and only 1% are Hispanic. A recently-launched website of the FDA, “Drug Trials Snapshots,” confirms this dismal picture.

Designed to “make demographic data more available and transparent,” and to “highlight whether there were any differences in the benefits and side effects among sex, race and age groups,” the website reveals instead an impressive lack of information. Reported on the website are 70 new drug approvals for 78 different indications. These data report only evidence about differences by the census categories for race (White, Black or African-American, Asian, American Indian or Alaska Native, Native Hawaiian or Other Pacific Islander, and Unknown). In nine of the reported trials data were considered sufficient to report detected differences in efficacy or side-effects in all racial categories, in two data were considered sufficient to report these differences for African-Americans and Asians, in seven data were considered sufficient to report these differences for Asians, and in two data were considered sufficient to report these differences only for African-Americans. No data are reported about ethnicity, socioeconomic status, disability, or other categories that might be important to the PMI and the benefits data about the planned cohort might bring.

Reported differences of interest included increased rates of akathisia (urge to constant movement) in African-Americans in a trial of Aristada, used for the treatment of schizophrenia; increased risk of allergic angioedema in African-Americans in a trial of Entresto, used for treatment of heart failure; a possible higher frequency of side effects among Asians treated with Farydak, used for treatment of multiple myeloma (data were insufficient to detect differences in other racial groups); and a higher risk of serious adverse events in whites compared to Asians in a trial of Tagrisso, used for treatment of advanced non-small cell lung cancer with a specific mutation of a receptor T790M. Approved drugs for which data were inadequate to determine any differences by race included Axumin, used for detection of prostate cancer recurrence; Darzalex, used for treatment of multiple myeloma in patients who have received at least three prior treatments; Ibrance, used for treatment of post-menopausal women with ER-positive, HER2-negative breast cancer; Ninlaro, used for treatment of multiple myeloma; Portrazza, used for treatment of squamous non-small cell lung cancer; and Zontivity, used to reduce risks of heart attack or stroke in patients with a prior heart attack or peripheral arterial disease. For Tresiba, recently approved for improving blood sugar control in patients with either type 1 or type 2 diabetes, the majority of clinical trial participants were whites or Asians and data were insufficient to analyze the possibility of differences for other racial groups; Novo Nordisk’s spending on advertising for Tresiba placed it as the fastest growing advertiser among pharmaceutical companies in 2015.

Whatever one’s antecedent judgment about the likely clinical relevance of any of these categories, one of the aims of the PMI is to explore these possibilities. The FDA reports in Drug Trial Snapshots are but another indication of how daunting the challenges to this enterprise may be. Whether in a new administration the PMI remains in roughly its present form, is renamed or reshaped, or abandoned altogether, information deficits may remain to be addressed. On December 1 and 2, the University of Utah S.J. Quinney College of Law and Huntsman Cancer Institute are sponsoring a life-streamed conference about the PMI designed to bring clinical, ethical, and legal experts together to explore these and other challenges of the PMI. Speakers with legal or ethical expertise will include Bernard Chao, Colleen Chien, Shubha Ghosh, Kshitji Kumar Singh, Jessica Roberts (Houston), and Maya Sabatello (Columbia).

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