Executive Summary (Chapter 1)
In this article, we illustrate how personalized medicine will change the pharmaceutical industry. When one contemplates the complexity of the future environment, it becomes quite clear that existing discovery, development, regulatory, commercialization, and reimbursement frameworks are nowhere near prepared for the changes coming with personalized medicine.
It is imperative that pharmaceutical company executives develop a common understanding of the changes ahead, so a cohesive strategy and set of actions can be implemented across the entire enterprise. If not, pharmaceutical companies run the risk that each function and therapeutic business unit will develop its own, unaligned set of strategies and actions to address personalized medicine.
Personalized Medicine – Background and Definition
Drugs work for some patients but not others; the same is true for drug side effects. For example, when receiving a chemotherapeutic like paclitaxel, some patients benefit and others do not – some patients have nausea or neuropathy while others do not. Thanks to a better understanding of cell signaling pathways, molecular biology, genetics, and genomics, we are starting to understand why some drugs are safe and effective for certain people but useless or harmful for others.
Although several definitions exist, “personalized medicine” or “targeted therapy” refers to the tailoring of medical care to the individual characteristics of each patient, often by classifying individuals into subpopulations based on their genetic makeup, susceptibility to a particular disease, or response to a specific treatment. Preventative or therapeutic interventions can then be concentrated on those who will benefit, sparing expense and side effects for those who will not. Some personalized medicines or targeted therapies have added years of life expectancy to patients with life-threatening conditions such as chronic myeloid leukemia (CML), HIV/AIDS, lung cancer and malignant melanoma.
Based on the definition above, there are at least two ways to develop personalized medicines:
- Leverage molecular biology to identify targets that are differentially expressed in the medical condition and then create drugs to “hit” the target. For example, both Herceptin® (Genentech) and Tykerb® (GSK) target the HER2 receptor, which is overexpressed in many breast cancer tumors.
- Leverage genomics and diagnostics to identify patients with a particular genomic profile who are most likely to respond to therapy or most likely to suffer toxicity. For example, Zelboraf®(Genentech) is approved for patients whose metastatic melanoma tissue has a specific mutation in the BRAF gene.
What’s Different about Personalized Medicine?
To bring personalized medicines to market, scientists analyze genomic profiles from hundreds or thousands of patients with a particular disease and compare them to genomic profiles of healthy patients. The goal is to understand how genetic differences contribute to disease. If a certain gene is missing or is not producing a needed protein, that protein can be synthesized and administered. If a certain gene is causing the body to create a “harmful” protein, that protein can be neutralized therapeutically and, if necessary, replaced with a “healthy” protein.
Because of how these drugs are developed, they are only likely to work for the subset of patients for whom they were designed. For example, the drug Herceptin® is only effective for breast cancer patients with HER2 positive tumors.
Similar to how the Internet has transformed the retail, music, and publishing industries, personalized medicine (or genomics or targeted therapy) is going to change to the entire healthcare ecosystem. Pharmaceutical companies will experience the greatest amount of change because personalized medicine changes how drugs are discovered, developed, submitted, commercialized, and reimbursed.
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