Screening policy for cystic fibrosis: the role of evidence.

Setting priorities about medical services, including genetic testing services, often occurs in an extemporaneous fashion. Normative assumptions may not be examined critically, although doing so is a necessary component of making health policy decisions about clinical practice. The normative dimension to health policy questions suggests a need for greater public participation in the development of clinical practice guidelines. The experiences of newborn screening and carrier screening for cystic fibrosis in the United States can be examined within the framework of two models of health policy development that help explain the role in health policy development of normative assumptions and public participation.[1] Specifically, this paper focuses on assumptions about what counts as sufficient empirical data to make health policy decisions. Models of Health Policy Development The Extemporaneous Model. There are many groups that may have an interest in the introduction of new technologies into clinical practice, Patients and patients' advocacy groups may be enthusiastic about a potentially beneficial treatment or test. Industry representatives, with obligations to stockholders and interests in their own jobs can be expected to promote potentially beneficial technologies. On the other hand, private and public insurers may be more skeptical of technological advances, given concerns about limited fiscal resources. Physicians may be supportive of new tests and treatments for their patients and may worry about the legal implications of not providing new technologies that are available and that patients request. State health departments may also have an interest in developing new genetics services programs that strengthen their position in the bureaucracy. The extemporaneous model assumes that these various forces will determine the utilization of and reimbursement for the new service. Utilization and reimbursement will then define the standard of care. Thus, decisions about new services are based on the interactions of the various groups with vested interests in either facilitating or impeding the development of new technologies. The Evidentiary Model. While the extemporaneous model describes the sociopolitical experience, the evidentiary model uses empirical evidence of benefits and harms as the basis for policy decisions. Although political mechanisms to employ this model still need further elucidation, the model describes some significant features for policymakers to consider. As the examples of screening for cystic fibrosis illustrate, the importance of collecting empirical evidence as a precondition for using new technologies, obvious as it may seem, is not always conceded. In fact, the debate has focused on what kind and amount of empirical evidence are sufficient for making policy decisions. The thrust of the evidentiary model is that while good empirical evidence is necessary, it is insufficient for making health policy decisions; the underlying normative dimensions of such decisions must also be explicitly discussed. For example, before it can be decided empirically whether a screening program is successful in educating the participants, decisions must be made about what counts as successful education. This in turn is based on what information is considered important and what amount of misunderstanding is considered acceptable. A second distinguishing feature of the evidentiary model is that once the implicit normative components of health policies are laid bare, the subsequent decisions about setting priorities for health care resources must directly involve the public. Clinical practice guidelines would thus be fashioned not only by professionals but with the help of the public. This nudges the debate toward what counts as public participation and how it can he determined that any social policy decisions that are made are just. These important questions are not addressed here. …