Three Things You Need To Know About Personalized Medicine

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By Caitlin Rothermel

I couldn’t decide whether I knew enough about personalized medicine. So I took it on myself to catch up on the topic. Based on what I learned, I think there are three things it’s important to keep in mind.

1. Personalized medicine is great for patients

Let’s quickly define “personalized medicine.” It’s the tailoring of health care delivery to individual patients based on their responses to specific treatments or susceptibility to particular diseases. In most cases, these targeted treatments are based on genetic information.

That’s great for patients. The biggest personalized medicine successes so far have probably been in the field of oncology, with new drugs that are particularly effective in tumors with specific mutations. In a recent study, treatment with the mutated-BRAF inhibitor dabrafenib in patients with BRAF mutation-positive advanced melanoma resulted in a doubling of progression-free survival vs. conventional therapy. Companion diagnostic testing (where testing for genetic susceptibility is required before treatment is initiated) is also key to personalized medicine. Patients with non-small cell lung cancer can take a diagnostic test to find out if they are among the 4%-5% likely to respond very well to crizotinib, an ALK inhibitor.

Personalized medicine also exists beyond genetic profiling. IndiGO, a decision support tool developed by Archimedes takes patients’ individualized health information and calculates their unique risk for negative health outcomes, as well as the likely impact if guideline-based interventions are implemented (eg, lifestyle changes or addition of a blood pressure-lowering medication).

2. Personalized medicine is progressing more slowly than you might think, but it is also everywhere

Some experts think that the term “personalized medicine” is too confusing, and this makes sense. Think of patients or think of yourself – don’t you expect personalized attention from your doctor? Because of issues like this an alternate term, “precision medicine,” is gaining traction.

It has been suggested that personalized medicine should refer to the individual genomic information that can be mined to learn about hereditary tendencies for specific diseases. Precision medicine, on the other hand, uses genetic information obtained from tumors to develop more precise therapies. This distinction helps to explain why change is coming slowly. The most heralded personalized treatment advances have been for cancers where researchers have had access to a large, longstanding repository of genetic samples. On the other hand, although a huge amount of genomic profiling information has been collected (with a lot more likely to come), it’s really a big-data dump at this point, with very few usable, causal associations identified.

There are implementation barriers, too. A recent report from the ISPOR Personalized Medicine Special Interest Group has an excellent discussion of how development incentives differ for pharmaceutical and diagnostic companies, making partnerships difficult to establish. Other research has shown that primary care physicians experience barriers to effectively communicating personalized medicine concepts.

3. The financial model for personalized medicine is not clear

According to ISPOR, more than 50% of biopharmaceutical companies now have personalized medicine divisions, with high hopes for the future. Broadly speaking, the economic model for personalized medicine is as follows: targeted approaches will replace the blockbuster drug model, meaning that fewer patients will be able to use specific treatments, but they will use them more efficiently. Therefore a higher price point can be applied, correspondent with the associated benefit.

But there’s reason to suspect that the “sky is the limit” approach to medication reimbursement may be over. Risk-sharing agreements are becoming increasingly common in the U.S. and overseas, and pricing for orphan drugs, traditionally higher than for other treatments, is currently under pushback in Europe. In the U.S. one month ago, Memorial Sloan Kettering hospital announced that it would not use the colon cancer drug aflibercept because of its hefty price tag (in this case, the drug was no more effective than other existing treatments). The manufacturer backed down and lowered the cost.

One thing for certain is that there will be plenty of work for health economists when it comes to assessing the value of personalized medicine. Since each personalized treatment creates its own niche market, cost-effectiveness analyses will be needed earlier in the process.

What do you think? Is personalized medicine a 21st Century game-changer? What are the practical applications of widespread genomic sampling? And what should we be watching out for – how will we know that personalized medicine has really arrived?

Caitlin Rothermel, MA, MPH is a medical and health economics writer. She lives in Seattle, WA with her family. Thanks to genetic sequencing, her husband now knows that he is of Irish extraction (this made him very happy). You can learn more about Caitlin by visiting