The Limitations on Learning from Clinical Trials

One of the many benefits from RWE can be realized when you look at the limitations on learning from clinical trials themselves.

A randomized clinical trial (RCT) is still the current standard to measure the safety and efficacy of a new drug. Unfortunately, the results obtained from a RCT do not always indicate how the drug will perform in the real world. In some cases, drugs that show promising results in the trial phase fail to deliver on expected outcomes, underperforming or triggering a side effect when introduced to a broader group of patients.

There are many reasons for the differences that can occur between a trial and the real world.

Patients who are recruited to participate in a RCT must often meet precise medical criteria and are monitored closely throughout the study to see that they adhere to the trial protocol. In the real world, however, patients are more complex. In many cases, they will have multiple medical conditions that make them tougher to treat or that limit their treatment options. Often patients are on other medications for chronic conditions like high blood pressure, diabetes or arthritis. In an environment where the average person has 12 prescriptions filled annually, and the average senior fills 28 prescriptions each year, drug to drug interactions become a real concern.

Furthermore, patients do not always follow their prescribed treatment regimen. There are many reasons why they do not take their medications as directed: forgetting, avoiding unpleasant side effects, and reducing the expense of prescription drug use are just a few. During a clinical trial, the medication is provided to the participant at no cost. Once a drug is on the market, however, financial issues are a significant consideration. Patients use many strategies to reduce their prescription drug costs, including skipping doses or taking less than the prescribed dose. Results from the National Health Interview Survey showed that 8% of adults admitted to not taking their medication as prescribed in order to save money.

Since the specifics of care in a clinical trial can differ markedly from what happens in the real world, the benefits found in a trial’s results can be difficult to ascertain more broadly. With the bill for prescription drugs rising year over year, however, payers are putting pressure on drug companies to prove the long-term benefits of their products. By analyzing data sets from other areas of healthcare, like EMRs and hospital data sets, it is possible for clinical study sponsors to augment the results of a clinical trial with knowledge of what happens in a larger population over a longer time frame. Not only is this useful to show a drug’s effectiveness over time but, through pharmacovigilance, insights may be revealed that can improve patient care and clinical treatment pathways.

Often it is only after a drug sees widespread use that patient subgroups are found who are unresponsive to the treatment protocol or who experience a previously unseen adverse drug reaction (ADR). The discovery of an ADR, in particular, can prompt a call for labeling changes from the Food and Drug Administration (FDA), a process that can be costly and laborious.

Suspected ADRs cannot be taken lightly though and must be thoroughly examined. Companies that have a robust RWE practice in place will have access to a rich source of de-identified patient data that can be used to investigate cases of suspected ADRs. This can be used to demonstrate a product’s safety or weigh the risks versus the benefits. The ability to provide this analysis may be integral to the product’s continued inclusion on a payer’s drug formulary or to maintain its preferred status for treatment.


This is the second installment in our RWE series. Next week: Using RWE Across the Drug Product Lifecycle.

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