Personalized medicine has attracted much interest within the healthcare community during recent years. More physicians are striving to customize their medical treatments to their patients’ unique genetic makeup and circumstances.
However, personalized medicine poses a number of regulatory challenges. As such, pharmaceutical executives should understand these challenges and how they affect the industry.
What Is Personalized Medicine?
Personalized medicine describes the tailoring of medical treatment to suit a patient's unique medical needs. This includes customizing a treatment according to a patient's genetic makeup or using a three-dimensional printer to create a device tailored to an individual patient. In personalized medicine, healthcare providers generally use a diagnostic device or test and a therapeutic product to treat conditions.
For instance, an oncologist may order a laboratory-developed in vitro diagnostic test that determines the biomarkers on a patient’s tumor. After the oncologist determines what biomarkers are involved, he or she may prescribe a chemotherapy treatment that targets a tumor with those biomarkers. As such, personalized medicine is designed to reduce guesswork, streamline the diagnosis and treatment of diseases, increase the efficacy of treatments, and decrease adverse events experienced by patients.
Issues Associated With Personalized Medicine
Because personalized medication has become increasingly complex and is constantly evolving, lawmakers have questioned the Food and Drug Administration's ability to regulate such treatments. Some issues concern whether patients should be able to purchase and use unapproved or unregulated tests. Other issues consist of whether manufacturers can be trusted to market tests that conform to standards for safety and efficacy if the process of regulating these tests is too slow and expensive.
An approval process that is not flexible enough to accommodate personalized medicine is another concern. Because a given population in which a personal medication product is tested may be small, some critics have argued that the Food and Drug's Administration’s requirement for large-scale trials and postmarketing surveillance is too unwieldy and expensive. They maintain that smaller more-focused trials may be necessary.
However, because clinical trials are expected to be more focused, less of the population is likely to be exposed to a drug, biologic, or device before the Food and Drug Administration approves it. As such, corporations and the Food and Drug Administration are less likely to understand the adverse event profile of a product.
Additional challenges include collaboration issues between government organizations that conduct research and the Food and Drug Administration and challenges regarding transparency among the Food and Drug Administration, companies, and patients regarding personalized medicine treatments as they are approved and marketed.
How the Food and Drug Administration Has Been Working to Resolve These Issues
The Food and Drug Administration has been working with pharmaceutical, device, and biologic companies and with researchers to make personalized medicine possible.
As such, they have been revising their processes to allow for more flexibility and to encompass the entire lifespan of a pharmaceutical, biologic, or device. Some areas that the agency is looking at include restructuring their organization to accommodate personalized medicine; changing regulatory mechanisms and clarifying policies; and collaborating with other organizations.
Changes to the Infrastructure of the Food and Drug Administration to Accommodate Personalized Medicine
The Food and Drug Administration has changed their infrastructure to address issues associated with personalized medicine. This has included restructuring the offices responsible for the approval of drugs, devices, and biologics and creating programs to accommodate research associated with personalized medicine.
One such example is their creation of Office of in Vitro Diagnostics and Radiological Health, a department within the Center for Drug Evaluation and Research that evaluates the efficacy of in vitro devices designed to diagnose conditions and devices related to radiological health. The Center for Biologics Evaluation and Research has also launched initiatives to create a review process that accommodates genetics and other innovative technologies.
Programs such as the Voluntary Exploratory Data Submission program enable pharmaceutical companies to discuss with the Food and Drug Administration issues associated with personalized medicine outside of the standard approval process. These changes define the responsibilities of different departments so that the reviews of personalized medical products can be timely and consistent.
Regulations and Guidances Designed to Address Personalized Medicine
Although no regulations address personalized medicine as a whole, a number of laws address specific treatments categorized as personalized medicine. For instance, laboratory-developed tests are regulated in conformance with the Clinical Laboratory Improvement Amendment regulations.
The Food and Drug Administration has also issued guidances to address issues associated with regulating personalized medicine. These guidances address the collection and use of genetic information in clinical studies and labeling requirements for personalized medication products.
For early-phase clinical research, the Food and Drug Administration has been working to develop standards and mechanisms that integrate the use of genetic information into the development of pharmaceuticals, biologics, and devices.
This includes issuing guidances on the design of early-phase clinical trials of cellular and gene therapy products and on the evaluation of genetic information in early-phase clinical studies. These guidances clarify to pharmaceutical corporations how to design early-phase trials that collect genetic information and how to evaluate that information once it has been collected.
Guidances that make regulatory requirements clearer and coordinate reviews of pharmaceuticals, devices, and biologics before they are approved have been issued. These guidances include, but are not limited to, the Pharmacogenomic Data Submissions, Pharmacogenomic Tests and Genetic Tests for Heritable Markers, Applying Human Factors and Usability Engineering to Optimize Medical Device Design, and Mobile Medical Applications. These provide clarification on research that integrates genetic information into the development of pharmaceuticals, devices, and biologics.
Food and Drug Administration has also developed guidances to address issues of labeling in the context of personalized medicine. In addition to being balanced and free of bias, the labels of medications that are more toxic, have a different effect, or must be given in different doses in certain populations must include such information. These labels may also be required to contain information on genetics and other biomarkers.
Changes to Monitoring Requirements
The agency has been developing mechanisms to monitor personalized drugs, biologics, and devices throughout their entire lifecycle. Expected to gain importance as a result of a lesser-known adverse event profile of a pharmaceutical, device, or biologic at approval, the Food and Drug Administration may require pharmaceutical corporations to submit postmarketing research protocols as part of postmarketing surveillance.
These enhanced methods will enable corporations and the Food and Drug Administration to monitor for adverse events that are less obvious or rarer in smaller populations.
Collaboration With Other Organizations
The Food and Drug Administration has also started collaborating with other organizations, such as the National Institutes of Health, the Agency for Healthcare Research and Quality, and the Centers for Medicare and Medicaid Services. These collaborations help the agency to gather information on the use of drugs, devices, and biologics in a real-world context. These collaborations can also benefit pharmaceutical companies because the companies can determine whether a product is economically feasible more easily.
Conclusion
Although regulating personalized medicine has been challenging, the Food and Drug Administration has been taking steps to address the issue. By understanding how these measures change the regulatory processes and expectations, pharmaceutical and medical device executives can better respond to these changes and develop products that accommodate this growing trend in medicine.
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