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PMC Report Outlines Strong Growth, Future Challenges for Personalized Medicine Industry

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NEW YORK – The Personalized Medicine Coalition said this week that the number of personalized medicines on the market in the US has more than doubled over the past four years.

According to the sixth edition of the organization's Personalized Medicine Report, the number of individualized medicines on the US market grew from 132 in 2016 to 286 in 2020, a 116 percent spike and the largest four-year increase since it began tracking this figure in 2008. In contrast, between 2012 and 2016 precision therapies grew only about 62 percent.

According to the coalition, there are now more than 75,000 genetic tests being marketed. And in oncology, which has been and continues to be a driver for this new medical paradigm, the PMC reported that 61 percent of clinical trials now incorporate biomarkers compared to just 18 percent in 2000.

The PMC defines personalized medicine, also called precision or individualized medicine, as "the use of diagnostic tests to determine which medical treatments will work best for each patient" coupled with "the use of medical interventions to alter molecular mechanisms, often genetic, that cause disease or influence a patient's response to certain treatments."

According to the coalition, the acceleration in this field over the last four years is expected to translate to significant improvements in the quality and cost efficiency of medical care. However, authors also stressed that the rising tide has brought with it new challenges related to regulatory oversight, payor coverage and reimbursement, and clinical adoption of these transformational technologies.

To ensure that the most possible patients can benefit, health systems are now challenged to double down on addressing these obstacles.

"Because our increasing understanding of human heterogeneity demands it, healthcare is in the midst of a transformation away from one-size- fits-all, trial-and-error medicine and toward this new, targeted approach in which, as is often said, the right patient will get the right treatment at the right time. Completing that transformation, however, will require a collaborative effort based on shared values across stakeholder groups to keep up with the pace of progress in science and technology," authors of the report wrote.

In cancer, the PMC characterized the past four years as a period in which drug development has begun to shift more decidedly from a focus on tumor tissue of origin to the genetic basis of the disease.

This is reflected in the progress of multi-cohort programs like the National Cancer Institute's NCI-MATCH trial, the most recent data from which has begun to demonstrate the value of such trials for the population of refractory cancer patients with rare genomic tumor alterations that would not be explored in a traditional drug trial let alone available on-label.

It is also evident, according to PMC, in the field's first tumor-agnostic drug approvals, in which a therapy is indicated based on the presence of a specific cancer biomarker regardless of a tumor's location in the body.

These include the first such approval for pembrolizumab (Merck's Keytruda) in advanced solid tumors that are microsatellite instability-high (MSI-H) or mismatch repair deficiency (dMMR) in 2017. This was followed by a second approval for the same drug and patient population, this time based on tumor mutational burden, a calculation of the overall degree of mutation occurring across the genome of cancer cells.

PMC also cited the recent tissue-agnostic approvals of larotrectinib (Vitrakvi) and entrectinib (Rozlytrek), for patients with NTRK gene fusions, which have invigorated similar efforts around other biomarkers like FGFR, ERBB2, and others.

The coalition also highlighted growing efforts to develop personalized strategies for the use of cancer immunotherapies, whether through predictive biomarkers, or in the advance of treatments that are themselves personalized, such as adapted cell-based therapies.

Authors also noted the growth in non-invasive, blood-based genomic tests for cancer, with the first US Food and Drug Administration approval for a liquid biopsy test in 2016, and the first two approvals for blood-based comprehensive sequencing tests this summer.

One other area of advancement that has emerged during this latest four-year period in oncology is a focus shift from the analysis and personalized treatment of advanced cancers to the precision diagnosis and screening of early-stage tumors, with the goal of catching and curing disease before it progresses to terminal stages.

Christopher Wells, PMC's VP of public affairs, said in an email this week that the report makes particular note of new blood-based biomarker tests being advanced to detect multiple types of cancers at their earliest stages, "when they can often be treated more successfully and with less costs to the healthcare system."

Considering the intense investment in this area in recent years, for example, in companies like Grail and Thrive Earlier Detection, which were both recently acquired in multi-billion dollar deals, the next four years could see a significant change in how precision medicine practices are distributed across the cancer care continuum.

"Regarding the growth of personalized medicine in areas other than cancer, it is clear from this report that [the field] is expanding its footprint across various rare diseases. Recently approved drugs are delivering unprecedented benefits for patients with cystic fibrosis, for example. The emergence of gene therapies promises even more impact in the rare disease space," Wells added.

According to PMC, of the 286 currently available personalized drugs collated by the coalition and listed in an appendix to the report, only 92 are oncology therapies, about 32 percent of the total.

"This is a testament to the indications in pharmacogenomics, rare diseases and many other areas that continue to play a major role in the evolution of personalized medicine," Wells said.

The report authors also cited recent research by Concert Genetics, which estimates that the number of clinical exome tests offered by US labs has grown from fewer than 80 in 2016 to more than 120 as of 2020.

While the PMC's takeaway in its report is one of accelerated growth patterns for personalized medicine as a whole, some of the data collected also suggests that there could be slowing or perhaps saturation occurring in certain areas. The coalition's analysis of cancer clinical trial trends, based on an analysis conducted by LEK Consulting, for example, shows that biomarker use in clinical trials for cancer treatments rose from 57 percent to 61 percent in 2017, but has since remained at about that same level through the end of 2019.

A significant portion of the coalition's report is dedicated to describing challenges and outlining best practices and principles that may be necessary to see the promise of new advances made real.

"Despite rapid scientific and technological advancement, the healthcare system has been relatively slow to integrate personalized medicine into clinical practice," the authors wrote, citing recent survey data from the PMC and others that suggest most healthcare organizations have only somewhat integrated personalized medicine practices and that only a small percentage of American patients said their doctor has discussed or recommended personalized medicine treatment options for them.

"Every corner of the healthcare spectrum must come together to advance science-driven, value-based solutions," the authors said. Among other recommendations, the report contends that regulatory authorities must clarify and standardize their oversight policies, translational researchers must demonstrate real benefit, and policymakers should begin to consider real-world evidence from clinical practice in their evaluation of novel personalized therapies.

On top of this, the field will need better-established pathways for evaluating clinical and economic utility of personalized medicine practices to facilitate coverage and reimbursement. Healthcare delivery organizations will have to get better at integrating precision medicine into clinical practice. Patients should take a more active role, and health information systems are going to have to continue to adapt, according to the report.

"Scientific discovery in personalized medicine will continue to accelerate, offering tremendous opportunities to both researchers and the patients who are looking to the next generation of medical advances. Personalizing care, however, requires the combined resources of multiple stakeholders — all of whom must be willing to invest in a paradigm change that can preserve innovation, improve outcomes, and reduce the overall costs of healthcare," the PMC authors wrote.