In his January 2015 State of the Union address, President Obama announced the $215 million Precision Medicine Initiative. Moving beyond a “one-size-fits-all” approach to medical care, the initiative is intended to produce the knowledge that physicians can use to tailor treatments according to each patient’s specific genes, environment, and lifestyle. By sequencing more genomes and linking genomic data to information in electronic health records and to participant-provided lifestyle data, the Precision Medicine Initiative will reveal to researchers and physicians the right treatments for the right patients.
Since the initiative was unveiled, a working group from the National Institutes of Health (NIH) has held a series of public workshops to gather stakeholder input on various aspects of this enormous enterprise. The latest session, held in early July, was devoted to the composition of the national research cohort: the one million Americans who will provide researchers data on their genes, environment, and lifestyles, through such methods as wearing electronic devices, providing bio-samples, and allowing access to their electronic health records. The collected information will be de-identified and analyzed by researchers to achieve the lofty goal of precision medicine for every American.
Ensuring A Representative Research Cohort
The cohort is not only the heart of the entire project; its makeup poses the chief concern for ethicists. For the Precision Medicine Initiative to benefit every American, citizens of all ethnic and racial backgrounds, health statuses, and social and economic circumstances must contribute personal information.
Yet the task of building a just, equitable cohort that reflects the diversity of this country is daunting — so daunting that there is reason to fear that it will not, and perhaps cannot, be achieved. Distrust of the government is strong in many populations. Some groups will require high percentages of participation to garner sufficient information about them. To ensure a representative database, those leading the initiative need to be willing to consider incentivizing participation and other creative solutions to motivate broad participation.
One reason for worry about equity and diversity in recruitment is the initiative’s reliance on electronic health records for data. Those who have been historically underserved by the U.S. health care system and underrepresented in research are more likely to have sparse or incomplete medical records. Many people see different health care providers over the course of their lives. This is especially true of those without reliable access to health care, who instead rely on emergency rooms, mobile medical brigades, free clinics, and the like.
Another cause for concern is that the Precision Medicine Initiative will gather data from existing troves of genetic and health information. There is strong evidence, though, that racial and ethnic minorities are underrepresented in these existing data sets. One study found that of those who’ve participated in modern genetic studies, a full 96 percent were of European descent. More than 80 percent of people who have shared data with the Personal Genome Project, a worldwide open-access database of genetic and personal information, identify as white.
Recent data show that NIH-supported studies also have a poor track record when it comes to enrolling minorities. Between 1993 and 2013, fewer than 5 percent of participants in NIH-supported studies were non-white, and fewer than 5 percent of all studies on respiratory diseases reported the inclusion of members of ethnic and racial minorities. All of this is to say that there is a dearth of existing data on already underrepresented populations in medical research.
Recruiting Participants – Altruism Versus Compensation
As of now, the initiative plans to rely on altruism to recruit participants. The idea is that Americans will be motivated, not by potential benefit to themselves, but because the initiative could produce enormous benefit to others in the future. Yet even if such people comprised an ethnically, racially, socially, and economically diverse lot, there’s not much reason to think they’ll agree to join in, purely to help others.
In a recent study in which whole genome sequencing was done on healthy individuals to obtain genetic and other data, just a third of participants consented to contribute their data to the National Institutes of Health’s Database of Genotypes and Phenotypes. More than half expressed privacy concerns. Distrust is a reality when it comes to government-run databanks.
How, then, can the Precision Medicine Initiative get a diverse enough participant base to guarantee that the cohort is comprehensive, so that the distribution of benefits, short- and long-term, is practicable and just?
One approach would be to compensate subjects for participation. This is common practice in recruiting subjects to research. Money is offered as reimbursement for expenses (child care, transportation), remuneration for time and effort, or as incentives for studies that otherwise might have difficulty enrolling subjects (e.g., challenge studies, toxicity testing, and some Phase 1 trials).
There are a few compelling reasons why the Precision Medicine Initiative could be justified in offering these types of payments to potential cohort participants. First, participants will incur costs—for regular doctor visits, providing bio-samples, procuring the devices that will record and transmit lifestyle data—and so will understandably seek reimbursement.
Second, the initiative will require time and effort from participants over the long course of the project. It is billed as a passive data collection initiative, in which electronic health records will be monitored, and mobile health apps and other technology will track lifestyle in the background of participants’ lives. But of course it’s not that simple. Devices need updating and upkeep; doctor and lab visits require time off from work and child care. New tests and measures will surely be added, requiring more subject time. The initiative will be most successful if cohort members are active participants. The activities expected of them merit remuneration.
Finally, researchers routinely offer incentives to subjects who participate in studies when those studies otherwise would not meet enrollment requirements. Take so-called “challenge studies” for the development of household and beauty products. Healthy volunteers are paid to see if household cleaners induce an allergic reaction or contain toxic levels of chemicals. Few, if any, people do this altruistically. To run these studies, researchers must pay potential participants.
Concerns About Compensating Participants
Of course, the idea of incentivizing participation in the Precision Medicine Initiative raises concerns, chief among them that paying subjects may lead them to discount risks involved in the research. Compensation, then, could exert “undue influence” on subjects, diminishing their ability to weigh a study’s risks and benefits. Yet not all influence is undue; indeed, many studies have shown that compensation does not necessarily undermine subjects’ ability to evaluate risks and rewards.
Another objection is that cash is an inappropriate enticement for participation in a federally funded initiative. Viewing the relationship between participants and researchers as a commercial transaction rather than citizens’ contribution to their country might taint the idea that the Precision Medicine Initiative is creating a national resource. But that need not be the case, because compensating subjects does not mean that the data they provide are a private commodity. Paying scientists at government agencies does not undermine the notion that the data they find are a national resource.
An Initiative That Benefits All Americans
Some will be opposed to compensating research participation because of cost. Providing any financial reward for participation in the Precision Medicine Initiative will require more money for cohort creation, leaving less for the actual research. But without a fairly created cohort, there will be a resource that helps only the few. The initiative can achieve its stated goals in a way that benefits all Americans only through recruiting a representative cohort. To overcome distrust, as well as to oversample minority groups, money may be the answer.
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