Personalized Medicine is the natural progression of medicine and science. If we are interested in treating or curing illnesses, then we must work to ensure that the best medicine is provided to the correct patient.
No one knew this better than Arno Motulsky, MD, dubbed the “founder of modern pharmacogenetics,” who passed away in January 2018 at the age of 94. Dr. Motulsky dedicated his career to investigating the genetics of medical therapies, essentially personalized medicine. Six decades later, computers and big data similarly allow us to design personalized medicine as well. While scientific and medical advances have alleviated tremendous suffering and saved countless lives over the last century, these treatments are useless if patients do not receive them. Marketers must continuously adapt to the changing needs of the healthcare landscape to improve disease state awareness, diagnostic efficiency, and appropriate, quality care.
Payers Change Marketing Equation with Patient Outcomes Push
Spark Therapeutics’ Luxturna, the first gene therapy for a genetic disease, the first and only pharmacologic therapy for an inherited retinal disease, and the first adeno associated viral vector gene therapy approved in the United States, will now be available to patients with biallelic RPE65 gene mutations and can be purchased for $850,000 to treat both eyes. While this was modest in comparison to analysts’ expectations of $1 million, payers still want assurances, so Spark is tying reimbursement to outcomes. In other words, if certain patient outcomes are not met by the drug, Spark will offer a partial refund to the insurer. Spark is not alone; Gilead and Novartis have tied reimbursement of CAR-T drugs (priced around $370,000 to $475,000, respectively) to pay-for-performance.
Outcomes-based reimbursement is not limited to technically complicated therapies or drugs with six-figure price tags either. The PCSK9 class of lipid-lowering agents—Repatha, developed by Amgen, and Praluent, a Sanofi-Regeneron partnership—are reimbursed according to performance. So too is Novartis’ heart failure drug Entresto, a relatively less complicated oral medication. As payers see it, if they are going to pay for a treatment, it better work. They want less risk. Perhaps the old-fashioned prospective clinical trial won’t cut it anymore. The 21st Century Cures Act helps alleviate this challenge by permitting smaller trials and real-world evidence into the drug review process.
Rethinking the Modern Medical Practice
The U.S. healthcare landscape is evolving; large systems are buying out or merging with smaller healthcare networks and fee-for-service reimbursement is being replaced with a system based on quality. Data, while having an impact on this changing landscape, is as yet insufficient to drive a conversation.
Although the era of big data is in its infancy, it is already fostering an environment in which 88% of U.S. physicians and 83% of European physicians now view real-world evidence as the top criteria for prescribing—placing increasing pressure on marketers and drug companies to provide meaningful messaging and information to stakeholders. According to Bain & Company research, half of all drug launches underperform. One explanation offered is that information channels are changing, and companies are experiencing difficulty communicating science effectively with the growing array of stakeholders that influence purchasing decisions.
However, the situation is not so nuanced. Customer-facing relationships cannot be expected to result in prescriptions; the issue is value in the context of quality care. Data without tangible or practical use will not result in changing medical practice. In other words, we must be able to demonstrate real value at both the system-wide and local levels of patient care to make an impact. Information must have tangible benefit to all stakeholders.
Quality Improvement and Reimbursement
Pay-for-performance is now impacting physician practices as well. However, tracking personalized data in a paper-based healthcare record prior to the advent of electronic health records (EHR) was labor intensive, impractical, and not reimbursable. Thanks to Medicare Access and the Children’s Health Insurance Program Reauthorization Act of 2015 (MACRA), physicians can now seek reimbursement for quality improvement activities. An effective method to improving a practice’s quality outcomes is the use of registry data at the local level. Practices can now efficiently query EHRs and assess their own outcomes—and be rewarded for such efforts.
Replacing the Sustainable Growth Rate (SGR), MACRA contains a new provision that changes healthcare reimbursement from a model that emphasizes volume to one that emphasizes quality of care. Under MACRA, physicians will have the opportunity to receive bonuses for delivering high-quality, low-cost care. While physicians will incur penalties for care that is deemed low on quality and high on cost, MACRA shifts the healthcare focus from volume of care to value in care.
Real-world evidence in healthcare can be generated from a variety of sources, including but not limited to patients (vital signs, behavioral data, patient-reported outcomes) and providers (EHRs, clinical notes, medical imaging). In context, more than 13 million EHRs exist for cancer patients in the United States alone. What drug makers, payers, healthcare networks, and other stakeholders do with that data matters. In order for pay-for-performance to be meaningful, drug makers, payers, and physicians must be aligned, analyzing data points that really matter to patient care, such as hospitalizations, mortality, and comorbidities—not surrogate endpoints. Real-world evidence that supports the use of a product based on surrogate endpoints or biomarkers without tangible improvement in outcomes, will do little to change patterns of care, nor should it.
Physicians Take on Rx Risk
Physicians are now responsible for their outcomes and are being asked by drug makers to take on the risks of prescribing new therapies while in some cases, standards of care have been proven in the real world for decades. Thus, physicians and healthcare networks now have an incentive to track their own outcomes and decide what is best for their patients and practices based on their own data. In an era of responsible medicine, marketers must approach the healthcare industry differently than before. No longer are practices separated from data, passively awaiting an industry interaction to inform them. Essentially physicians and healthcare networks, not drug makers, will be the data generators. Predictive modeling based on real-world data means that physicians will eventually be able to tailor therapies to individual patients based on their own data.
What to Expect
The focus on quality and rewarding physicians for their outcomes presents a challenge to drug companies. As practices collect data and query their own records, there is a plausible scenario where physicians will confront drug companies and their respective customer-facing personnel with conflicting outcomes data. Outcomes data can also be shared across healthcare networks, resulting in increased reimbursement for practices, but more importantly, improving patient outcomes.
Real-world evidence generated by quality improvement measures at the practice or system level will be evaluated against national averages. The data sets generated will reflect system-wide outcomes, but they are the outcomes of individualized care. Analyzing these data will permit practices to make informed individualized treatment decisions, personalizing medicine. This in turn, should at least in part, result in better outcomes aligning with the shared pay-for-performance expectations of drug makers and payers, thus reducing failed treatments.
As a result of quality metrics, new positions likely called “quality control officers” or “quality committees” will develop across healthcare practices and systems. These individuals or groups will become the primary (and possibly sole) nexus through which industry representatives will have accessibility to discuss data. The data discussed will not be strictly trial data, but rather reflect the real-world evidence, both clinical and economic, of a company’s product portfolio. These data will be compared against the regional outcomes collected by these systems.
Thus, healthcare marketing will evolve from a messaging- or branding-focused enterprise, towards a relationship-management and healthcare-solutions focused industry. Marketers will find new ways to improve communications between healthcare providers and payers, resulting in better patient outcomes. They will develop platforms to improve data collection and analysis, partnering between drug makers and healthcare providers to improve patient care. The dialogue must change wherein the goal of the Industry-Physician relationship results not in a prescription or a sale, per se, but rather quality care for the right patient, serving both drug makers and physicians, mitigating financial risks under the new pay-for-performance models.
Bringing It Together
The drug industry paradigm is evolving from launching a handful of blockbuster drugs towards a focus on more numerous but smaller products with more intense competition. Drug manufacturers are looking to efficiently identify appropriate patients to expedite and improve care, while also reducing costs. Personalized medicine—tailoring treatments to the individual patient—is part of the solution. Advances in personalized medicine, such as companion diagnostics, direct the correct therapy to the correct patient at the correct time, or in some cases, prevent the wrong medicine being prescribed to a patient, thus minimizing harm, improving outcomes, and reducing costs. Likewise, recent developments in artificial intelligence offer promising opportunities for analyzing large data sets, identifying appropriate patients based on historical outcomes, and improving outcomes.
To deliver effective personalized care, we will have to determine how sub-populations perform at the local or regional level compared to national means. This will permit physicians to tailor medical practice patterns according to the needs of—or rather, in response to—the outcomes of their local patient populations. In addition to searching outcomes data based on an admitting diagnosis, such as heart failure or pneumonia, physicians can now segment their populations by genetic profiles if they have such data on record. They can determine for themselves or their affiliated organizations what the real-world evidence is in their practices and healthcare networks.
Physicians and drug makers are entering an era of pay-for-performance. Rather than directing messaging at physicians, drug makers must partner with physicians with the help of marketers, focusing on diagnosis and treatment outcomes as a priority—rather than sales numbers. That means sharing the risks of reimbursement. Marketers will remain relevant by shaping campaigns and tools that help physicians improve patient care. Rather than selling, healthcare marketing of the future will surround partnering to meet patient needs.