Personalized Medicine: Where Is Specialty Pharmacy Heading?

A personal look at personalized medicine, its many ramifications, and a new era of pharmaceutical care.

Personalized medicine—the tailored treatments that a few patients now receive based on their own DNA—is finally headed for the greater public. Here are a few examples of how personalized medicine is making an impact on health care today.

We have witnessed the introduction of Provenge from Dendreon in the past 6 months. This product is indicated for metastatic prostate cancer. The product is made by using the cells of the afflicted patient in the manufacturing process.

At least 2 million Americans with an abnormal, clot-triggering heart rhythm take the drug warfarin, sold as Coumadin (Bristol-Myers Squibb) among others. Getting too little of this medication can lead to a stroke, and getting too much can cause life-threatening bleeding. To find the right dose for each patient, doctors use trial and error—and the errors lead to tens of thousands of hospitalizations and deaths every year.

About 1000 patients who have a condition known as atrial fibrillation will take part in a project that will match their warfarin dose to their specific genetic needs. This genetic fingerprinting should single out the many people whose bodies break down warfarin faster or slower than normal—and their doctors can immediately adjust their dosage to prevent dangerous complications.

The Evolving Future

Personalized health care is a rapidly evolving science that is guiding the development of so-called “designer drugs” that will be individually tailored, administered, and dosed based on a patient’s specific genetic composition. The goal is to have fewer side effects and holds the promise of treating diseases years before the symptoms would become apparent.

The evolving future of personalized medicine as it affects the pharmacy community is a positive one. The entire health care delivery system must respond with changes in medical education and medical practice. Thirdparty payers, health care facilities, and regulatory agencies must all embrace the promise and benefits of personalized medicine and adapt their processes accordingly. Pharmacy will play an important role in a number of situations, including monitoring patients and their response to therapy. We will discuss later how pharmacy can play a role in the management of the Risk Evaluation and Mitigation Strategy (REMS) required by the FDA to allow a product to come to market.

In oncology, for example, a therapeutic arena in which medications may cost a single patient hundreds of thousands of dollars a year, understanding the complex genetic pathways that will identify whether a patient will respond to a particular therapy is becoming an essential component of care. This aspect of medical practice is truly personalized to the individual.

The FDA provides a table of Valid Genomic Biomarkers (www.fda.gov) that can assist physicians in therapeutic decision making as well as provide pharmacists with risk identification. In light of the accelerating science, it is recommended that pharmacists be aware of the developments in genomic science and technologies of prescription medicine. This will put pharmacy in a good position to assist the medical community in ensuring that the patient has gone through proper testing before being placed on a particular therapy.

Policymakers and federal agencies are 2 other groups whose efforts must be engaged in the goals of personalized medicine. The recently signed HR 3590 Health Reform bill includes a section that will establish a Patient-Centered Outcomes Research Institute responsible for “developing and improving the science and methods of comparative clinical effectiveness,” also known as comparative effectiveness research.

The inclusion of this policy was greeted as another important step toward bringing personalized medicine officially into mainstream health care delivery. The process by which industry develops new drugs through a personalized genomic approach versus the traditional “one size fits all” approach to blockbuster drug research is also facing an overhaul. In order to facilitate this, however, changes must include a meaningful amendment of the FDA regulatory process affecting the way new drugs are developed and how quickly they can be brought to market.

The pharmacy community encourages the FDA to build on Prescription Drug User Fee Act provisions related to personalized medicine by continuing to direct resources for staff and outreach activities. This is a fast-growing field that will continue to impact the drug approval and labeling process. Much education and outreach is needed on this evolving practice and on pharmacogenomics. We encourage the FDA to work with partner organizations— such as various pharmacy organizations and advocacy groups—to help increase awareness and educate practitioners on how pharmacogenomics and personalized medicine can improve patient safety and outcomes.

Patient Safety and REMS

The pharmacy community has been actively involved in many REMS discussions with the FDA and other stakeholders throughout the past few years. As mentioned, the pharmacy community supports the FDA’s efforts to assure patient safety and the appropriate and safe use of medications. We recognize that the FDA has dedicated a great deal of staff to work on REMS issues. We also appreciate that the FDA has acknowledged the important role of pharmacists and pharmacies in implementing REMS programs, and the administrative and workflow challenges that must be addressed for both prescribers and pharmacists.

Unfortunately, REMS programs at this point have been inconsistent in their methodology and implementation. Pharmacy must play a role in helping the FDA to ensure that:

• Manufacturers and the FDA receive input from front-line pharmacists, physicians, and other prescribers early in the development of any REMS.
• Manufacturers are made aware early in the drug approval process if a REMS is going to be required.
• Standardized processes and components are used to address administrative, logistical, and workflow implementation challenges.
• Components are proved to be effective in mitigating the defined risks, and are workable for all stakeholders (patients, prescribers, pharmacists, manufacturers, wholesalers, and system vendors).
• Outcome metrics capture the reason for patient failure and/or success, rather than just documenting the occurrence.
• The potential impact of pharmacistprovided clinical care, such as in improving patient safety as an element of a REMS, is recognized in addition to the need for a business model for implementing such requirements.

A New Era of Pharmaceutical Care

We are entering a new era of pharmaceutical care. The pharmacist is now considered an integral part of the health care team. We must embrace this role and focus our efforts on proper counseling for the patient. We need to partner with the manufacturer community to deliver effective methods of managing a REMS process.

We need to align our profession to serve a role in the identification of the correct patient population as it relates to personalized medicines that are now entering our space and to monitor the successes and failures of the patient.

Pharmacy must have the ability to report these outcomes back to the provider so adjustments can be made quickly for the patient’s safety and benefit. Only then will our profession truly be viewed as an important component of the overall health care community. SPT

Nick Calla, RPh, is vice president, trade relations, Walgreens Specialty Pharmacy. He is an editorial board member of Specialty Pharmacy Times.