Personalized Medicine vs. Comparative Effectiveness Mailbag

Better late than never. Readers of IN VIVO Blog responded to a previous post on whether proponents of personalized medicine can also be supporters of comparative effectiveness research. We asked readers to weigh in on that question. Their emails initiated a lively discussion on the future of healthcare so we’ve picked excerpts from the most noteworthy responses to share. Here they are:

“Here’s an example to answer your query: Drug A is generic, Drug B is not. Compare their effectiveness and B works “better” so we all pay more for the newer drug. However, Biomarker Q indicates that some people do not respond to A at all. Remove those people from the analysis and now A&B work the same except for in the people with Biomarker Q, who have to get Drug B, although most would benefit from the less expensive drug A.” -Amy Miller, PhD, public policy director, Personalized Medicine Coalition

“Sure they go together. Right now, we characterize the groups in whom we compare therapies for ‘effectiveness’ based on their clinical diagnosis. For example, in cancer research we might compare a new treatment with an older one in ‘breast cancer’ as diagnosed by a pathologist reading a biopsy slide of a breast mass.

In the future, the ‘groups’ will be characterized not by their tumor biopsy’s look under a microscope, but by the unique genes expressed by the tumor. It’s the grouping parameters that will change and that’s how personalized (or genomic) medicine assists comparative effectiveness research. Personalized medicine just means we have better tools.” -Leonard Zwelling, MD, University of Texas M. D. Anderson Cancer Center

“The end-goal of personalized medicine and pharmacogenomics (the right drug in the right dose for the right person at the right time – comprising multiple, different ‘right’ answers) is very, very different from so-called ‘evidence-based medicine’ or comparative effectiveness, which looks to find a single best answer across an entire population. And it’s surprising how often people mix up the concepts—after all, each is an approach to improving medical care, but the underlying unit of analysis (individual versus population) is completely different.” -Bryan Walser, CEO Perlegen Sciences Inc.

“I hardly see the two options as diametrically opposed. The key concern for any legislator is getting the most value for every healthcare dollar spent. Comparative effectiveness attempts to divine the best option among multiple treatment options. Personalized medicine does the same thing, albeit for a narrower subset of patients. If one concedes that personalized medicine will always focus on smaller and smaller subsets of patients, it is not hard to imagine that comparative effectiveness will always be one subset above.” -Howard Hechler, director of business development, XL TechGroup

“Consider that personalized medicine can never be unique for each individual patient. Rather, it will be various medicines aimed at specific subsets within a disease state. For instance, multiple genetic defects can be responsible for inhibiting the production of a specific vital protein. One drug can treat one specific defect, while another drug could be used when the defect is at another location within the same gene. We could end up with dozens of drugs to treat the same disease, yet each treats very different specific defects.” -Steve Evans

“Think of personalized medicine as The Promised Land. Even its biggest boosters concede that it’s some way out on the horizon. There’s still a lot of progress to be made on the biomedical, informatics, education and training, and economic fronts. So, while we’re still wandering around in the wilderness treating patients with one-size-fits-all drugs, comparative effectiveness trials seem like a good idea.” -Michael Goodman, VP research products, AVOS Life Sciences LLP

“Personalized medicine and comparative effectiveness may indeed be strange bedfellows as you say in your post. In some ways, though, I think they both represent the triumph of science over marketing, and that appeals to a certain segment of the health policy community. With both of those principles fully in place, drug firms couldn’t use promotional muscle to get scripts written; they’d have to use data. And while it’s true the generalizations of comparative effectiveness are the opposite of the specifics of personalized medicine, I don’t think CE advocates would really object to a step therapy formulary that would eventually bring patients to the treatment that’s best for them personally.” -Anonymous

“My understanding is that the concepts are consistent because both personalized medicine and comparative effectiveness are based on making the best use of available information. In some cases we have enough information to craft therapies to particular patient situations (e.g. we might know that someone with a certain gene will respond in a certain way). Whereas comparative effectiveness deals with those cases where we simply do not have enough specific information but we do have statistical averages suggesting what the best practice would be given no other information about the specific case. Sometimes that’s all the information we have.” -Jason Bradfield

“Perhaps the similarity is that both personalized medicine and comparative effectiveness medicine use comparative measures. The difference is that one is in an individual population, while one is in a “smeared” population. The lowest standard for the measure of the safety and effectiveness of personalized medicine is on a comparative scale to other therapies used within the broader population. A higher, and probably unachievable standard, is for personalized therapy to be measured on an absolute scale for safety and effectiveness within a single person. Because this former standard is the more achievable standard, the two proponents can align, at least for now.” -Anonymous