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More Drugs, Fewer Patients: Limits of Precision Medicine

Posted on November 9th, 2015 by in Chemistry


A number of targeted medications that are highly effective for a subgroup of patients with a particular disease have emerged in the last decade. These are often needed alternatives to debilitating therapies with uncertain outcomes. The small number of patients that benefit from such therapies, however, may pose a serious challenge looking into the future.The evolution of drug development models, from “blockbuster” to precision medicine and into the future.

Read this excerpt from our white paper, ‘No Drug Designed For Me’ and find out if precision medicine can evolve to overcome the challenge.

Not All Patients Are Alike

The idea that a drug is more effective in patients with a particular gene or gene mutation was not new in 2004. Already in the 1970s, the correlation between treatment response to the cancer drug tamoxifen (Novaldex®, AstraZeneca) and high expression of the estrogen receptor gene led to the suggestion to select patients for treatment on the basis of this overexpression (12). Furthermore, the first precision medicine to be approved by the FDA with a companion diagnostic for patient selection came in 1998: the breast cancer drug trastuzumab (Herceptin®, Genentech) and HER2 assay HercepTest® (Dako).

In the last decade, precision medicines have afforded new treatment options and renewed hope for thousands of non-small cell lung cancer patients.

What was new was the realization that low efficacies of cancer drugs might be attributed to the heterogeneity of the tested patient population, which essentially dilutes the strong therapeutic effect that a drug might have on a specific patient subgroup (14). Prior to this realization, drug development assumed that one drug works for all patients diagnosed with a particular disease. This “blockbuster” model has been, with few exceptions, notoriously unsuccessful in oncology. An FDA publication highlights a 2001 study reporting an average efficacy of cancer drugs — a measure of the capacity for beneficial change of a given intervention — of only 25% (15). Another study in 2004 reported that only 5% of available drugs are effective in patients whose cancer has metastasized (16).

This realization marks a milestone in the clinical development of drugs. The blockbuster approach, with its assumption “one drug fits all,” began to crumble and a new approach aiming to deliver “the right drug to the right group of patients” emerged as a more meaningful and potentially more successful way of bringing new drugs on to the market  (Figure 3). Information about patient traits that impact response to a drug presented an opportunity to dodge the high attrition rate that has plagued the pharmaceutical industry for decades. The benefit of focusing the clinical development of a drug is epitomized by the approval history of the highly effective precision medicine for NSCLC, crizotinib  (Xalkori®, Pfizer).

Read the full white paper, No Drug Designed For Me.

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