Regulatory Challenges of Precision Medicine
Researchers from many countries say that responding to President Barack Obama’s call to modernize the current regulatory landscape for precision medicine will be no easy task. The worldwide regulatory challenges that arise in response to calls for precision medicine were explored in a 2014 workshop in Hobart, Australia. Information from that workshop and other sources is in a lengthy article, “Precision Medicine: Drowning in a Regulatory Soup?”, in the Journal of Law and the Biosciences.
“In this era of rapid technological change,” the article says, “the need for appropriate regulatory oversight is acute to ensure that available technologies are safe and that people receive the best-indicated treatment. There is, however, more to regulation than the (at times) blunt instrument of the law. Softer regulatory and educative tools also need to be brought into the mix.”
The authors note that in some ways the regulatory challenges presented by precision medicine are formulations of old tensions such as community welfare versus individual liberty, risk versus benefit, and autonomy versus paternalism. “The potential for precision medicine to exacerbate existing disparities in healthcare, both within countries and internationally, should also be at the forefront of our concerns,” they write.
They quote statements from FDA officials indicating that the first challenge that must be overcome for precision medicine to advance is scientific. Essentially, the article says, proof of analytic and clinical validity and clinical utility is required. But FDA also is said to realize that regulatory policy and management challenges are posed by the technological advances.
“Some commentators,” the authors say, “argue that current uncertainties in regulatory requirements are deterring investment in this field. This, they argue, is leading to the conclusion that there will need to be significant changes in the way that FDA and equivalent agencies in other countries operate, along with unprecedented cooperation across multiple centers and departments. Such significant changes will likely require corresponding legislative reform.”
The article says it is not yet clear whether effective implementation of precision medicine will require a different approach to how we apply and use regulation than what is currently the case. “Who and what we regulate,” it says, “clearly needs to be in scope — is it tests and healthcare products or is it professional and research practice, or all of these? This affects what is regulated and who the regulator will be. The question is not how to regulate, but how to regulate well.”
The key regulatory challenge, the article concludes, is how to effectively facilitate the practice of precision medicine so that we maximize the potential benefits while avoiding excessive and inappropriate utilization that could harm patients, drive up healthcare costs, and draw funding away from other needs. “Whether regulation, either existing or tailor-made for precision medicine, can be effective remains to be seen,” the authors say. They identify five recurring elements that they say must be taken into account in developing any precision medicine regulatory framework: appropriate consideration of safety, efficacy, and patient need; cost-effectiveness; consistency/equivalency across geographical, technological, and institutional borders; respect for cultural differences; and genuine engagement with all relevant stakeholders.