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Personalized Medicine’s Quality and Regulatory Issues

Genomic medicine has long offered the promise of gene-based disease prevention and treatment—allowing medical providers to successfully predict, prevent, and treat disease based upon an individual’s genetic or biomarker information. And while we are a long way from the fountain of youth, personalized medicine, also known as precision medicine, is no longer a future-situated hypothetical; many physicians even offer genetic testing at routine wellness visits. But personalized medicine triggers a host of questions for life sciences companies to sort through, not the least of which pertain to quality and regulatory concerns.

How Do Companies Establish Quality Standards for Personalized Medicine?

While the practice of medicine has always been personalized—individual patients treated based on their health profiles and disease markers—the evolving field of personalized and precision medicine involves more refined diagnostic testing than traditional medicine. And treatment is tailored to the patient after accounting for the patient’s unique biological, physiological, and environmental profiles. As defined by the International Society for Pharmacoeconomics and Outcomes Research (ISPOR), personalized medicine is the “use of genetic or other molecular biomarker information to improve the safety, effectiveness, and health outcomes of patients via more efficiently targeted risk stratification, prevention, and tailored medication and treatment management approaches.”

But given the departure from one-size-fits-all or trial and error diagnostic and treatment models, unique quality assurance challenges arise. For instance, how can adverse event tracking models apply to highly tailored treatments specific to each patient’s unique biology? How can clinicians and pathologists ensure that specimens remain valid and reliable throughout processing? And how will companies absolutely ensure the safety of protected health information that will increasingly include patients’ individual genetic code? These questions are generating a lot of buzz in the world of personalized and precision medicine and will continue to do so in the coming years.

Adverse Event Tracking in Personalized Medicine

Ironically, though the prevalence of adverse events theoretically decreases with the targeted provision of personalized medicine, a new problem of tracking adverse events emerges. While adverse event tracking in traditional medicine involves large sets of variable data, in personalized medicine, the number of variables is vastly increased.

Sophisticated big data analytics will continue to play an important role in adverse event detection, developing efficient flows of information and complex algorithmic solutions to assist pharmaceutical and biotech companies as well as regulators in successfully interacting with adverse event data.

Specimen Processing: A Lack of Standardization

A celebrated tenet of personalized medicine is that only treatments likely to benefit the patient are administered. But it was discovered that unreliable specimens led to inaccurate HER2 tests in up to 20 percent of cases, leading to inappropriate breast cancer treatment decisions.  As a result, guidelines were established to ensure proper specimen-handling. However, such guidelines are not the norm vis-à-vis specimen processing and there is generally a lack of standardization as specimens travel from patient to pathologist.

As this issue becomes more visible, watch for further guidance from the following authorities: the American Society of Clinical Oncology (ASCO) as well as other professional organizations; the World Health Organization (WHO); the FDA; and the Centers for Medicare and Medicaid Services (CMS).

No Clear Regulatory Framework for Personalized Medicine

Another challenge for pharmaceutical and other life sciences companies is anticipating the regulations related to personalized healthcare, including gene and cell therapy. As human cell and gene therapy-related research and development rapidly expands, all stakeholders must watch for equally rapid shifts in regulations.

For the third year in a row, more than 25% of all new molecular entities (NMEs) approved by the FDA’s Center for Drug Evaluation and Research (CDER) in 2016 were personalized medicines. And July 2017 saw the FDA’s approval of Kymriah, Novartis’ CAR-T gene therapy, indicating the FDA’s readiness to support personalized medicine. However, with novel biologic products comes minimal long-term evidence of safety and efficacy as well as a dearth of treatment experience. This doesn’t help in alleviating uncertainty around the future of regulatory action and market viability of such personalized treatments.

Lately, many international workshops have convened to address the emerging regulatory issues pertaining to cell therapy while regulatory bodies have published guidance for cell therapy products—including several guidance documents added by the FDA in late 2017.

The well-known provision for real world evidence (RWE) in the recent 21st Century Cures Act (the “Cures Act”) has the potential for a major impact on the approvals of personalized medicine products. Given the non-traditional treatment profile of a gene therapy, RWE is especially helpful in assessing personalized medicines’ long-term benefits and risks in clinical practice. Under the sixth installment of the Prescription Drug User Fee Act (PDUFA VI), the FDA has committed to speeding the drug approval process by hiring more highly qualified experts as well as facilitating a “more well-established use of real-world evidence to support post market drug safety surveillance” by instating a new safety review system.

The Quality and Regulatory Landscapes Will Evolve with Personalized Medicine

As with many emerging technologies, the enthusiasm surrounding personalized medicine is tempered by uncertainties in quality and regulatory schemas. As the shift to personalized medicine is younger than the laws that otherwise regulate the medical and research fields, there are frustrating gaps between technology and oversight. But these gaps should narrow while companies continue to make reasonable assumptions while they wait. As always, all stakeholders must remain vigilantly abreast of quality and regulatory developments as personalized medicine shows promise as the future of healthcare. Vigilance is of particular import to the many smaller developers of personalized medicines that may have limited resources or regulatory support.

Stakeholders must remember that regulatory views differ on a global basis, including between the FDA and the EMA. We expect a common set of principles to emerge as personalized medicine continues to secure its foothold as an accepted field of medical treatment.

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