Personalized medicine is the customization of healthcare. Already, a suite of molecular approaches is being used to tailor certain medical decisions, practice and products to the individual patient. For example, melanoma, leukemia and metastatic lung, breast and brain cancers can be routinely “molecularly diagnosed” and treated with medicines that target the specific genetic mutations. These represent a remarkable improvement over trial-and-error medicine.
Other diagnostic tests use the patient’s DNA sequence to determine drug-metabolizing capabilities. This helps improve dosing of drugs for conditions as wide-ranging as depression, coronary artery disease, inflammatory bowel disease and cancer.
And the scope of personalized medicine has rapidly broadened to encompass all sorts of personalization measures. Among these are tools that will help transform medicine from the treatment of illness to the maintenance of wellness. This not just some future-world vision: personalized medicine is already beginning to happen, and happen quickly. In 2006, there were just 13 prominent examples of personalized drugs, treatments and diagnostics on the market. By 2011, this number had risen to 72, and today there are 113. That’s a 57 percent increase in the last three years. But the introduction of personalized medicine can’t happen quickly enough.
The great opportunity for personalized medicine is its potential to introduce new scientific, business, and medical models. Segmenting populations into groups of patients who have a greater likelihood of responding to a particular treatment or avoiding side effects not only can change the dynamic of drug development but also the practice of medicine. Patients can benefit from better drugs, as well as new diagnostic and prognostic tools.
The way forward
As part of the effort to maintain momentum, yesterday a new report was published, titled The Case for Personalized Medicine. This is the fourth version – the first version was published in 2006 – and it is required because of the speed of progress in personalized medicine since the third edition was published in 2011. The Case is an examination of the opportunities – and the challenges – that the continued development and adoption of personalized medicine is meeting, set against a backdrop of faster and cheaper genetic sequencing, increase commitment from the pharmaceutical industry and evolution in the public policy landscape.
The Case for Personalized Medicine is published by the Personalized Medicine Coalition (PMC), which represents innovators, scientists, patients, providers and payers. “With progress comes a greater responsibility to move personalized medicine forward,” says Edward Abrahams, President of PMC. “In a time of unprecedented scientific breakthroughs and technological advancements, personalized health care has the capacity to detect the onset of disease at its earliest stages, preempt the progression of disease, and, at the same time, increase the efficiency of the health care system by improving quality, accessibility, and affordability. We’ve come a long way, but we have a lot to do, especially in education and advocacy.”
The report identifies seven major benefits of personalized medicine. These are to:
- Shift the emphasis in medicine from reaction to prevention: Personalized medicine introduces the ability to use molecular markers that signal disease risk or presence before clinical signs and symptoms appear, and it offers the opportunity to focus on prevention and early intervention rather than on reaction at advanced stages of disease. In many areas, the clinical interventions can be life-saving.
- Direct the selection of optimal therapy and reduce trial-and-error prescribing: Many patients do not benefit from the first drug they are offered in treatment. The majority of patients, for example, have at least one DNA-based variation in the enzymes that metabolize half of the most commonly prescribed medicines. The use of genetic and other forms of molecular screening allows the physician to select an optimal therapy the first time, thus avoiding the frustrating and costly practice of trial-and-error prescribing.
- Help avoid adverse drug reactions: According to several studies, about 5.3 percent of all hospital admissions are associated with adverse drug reactions. Gene variants can cause drugs to be metabolized either faster or slower than normal. As a result, some individuals have trouble eliminating it from their bodies, leading to “overdose toxicity”; others eliminate the drug too rapidly before it has had a chance to work. If these genetic variations are not considered when dosing, the consequences can range from unpleasant to fatal.
- Increase patient adherence to treatment: Patient non-compliance with treatment leads to adverse health effects and increased overall health care costs. When personalized therapies prove more effective or present fewer side effects, patients may be more likely to comply with their treatments.
- Improve quality of life: A molecular diagnostic test that simply requires a blood sample can replace invasive and uncomfortable tissue biopsies.
- Reveal additional or alternative uses for medicines and drug candidates: A medicine that may show weaker efficacy in a more generalized patient population may also show greater benefits when its use is limited to genetically defined patient populations.
- Help control the overall cost of health care: Personalized medical care has the potential to reduce health care costs worldwide. By concentrating therapeutic interventions on those who will benefit, we spare expense and side effects for those who will not.
Team effort required
To achieve the above benefits, the report argues, we need intelligent coverage and payment policies, new regulatory guidelines, and modernized professional education to prepare the next generation of doctors and other health care professionals for personalized medicine.
The report pays special attention to the laws and regulations that govern personalized medicine products and services used in clinical practice which are, necessarily, complex. FDA policies pertaining to personalized medicine tests, pharmaceuticals, and companion diagnostics are of particular importance.
Likewise, coverage and payment policies—whether in government programs like Medicare or those of private payers—play an essential role. Payers recognize the benefits of personalized medicine products in patient care management, but they increasingly seek additional evidence of their clinical, if not economic, value. In addition, payers are expanding new models for health care payment and delivery that could have a significant impact on the ability of patients to gain access to personalized medicine products and services. Understanding the changes and potential consequences these policies will have on personalized medicine tests, pharmaceuticals, and companion diagnostics is essential to ensure continued progress in the improvements to patient care.
The opportunity offered is summed up in the report by noted biotech entrepreneur Brook Byers: “Personalized medicine is our chance to revolutionize health care, but it will require a team effort by innovators, entrepreneurs, regulators, payers, and policymakers.”