- A new five-year study, published in the Journal of Clinical Oncology, found that many newly approved oncology drugs don't include information on patient experiences in their labeling. Only three of the 40 cancer drugs approved by the FDA between January 2010 and December 2014 came with patient-reported outcomes in their package inserts.
- Patient-reported outcomes (PRO) data is derived from questionnaires or patient logs used during clinical trials and can be helpful to others making treatment decisions. While the information doesn't have to be included on a label, the FDA has encouraged companies to include it.
- The researchers, led by Carla Demuro, also analyzed the labels of all drugs approved during that time period for comparison: 24% of the 160 new drugs included PRO data.
Oncology is a therapeutic area fraught with fear and uncertainty for patients, which is why inclusion of PRO as part of labeling could help patients and physicians make more informed treatment decisions.
As DeMuro told Reuters, “It [PRO] provides insight into the patient experience and allows us to understand how a drug impacts how a patient feels and functions."
DeMuro and her colleagues did find, however, that roughly one-third of oncology drugs in their data set had PRO data available which wasn't included in the product insert.
One possible reason behind the less-frequent use of PRO information in cancer drug labeling could be due to the smaller trial sizes common with cancer drug research. Additionally, most of the cancer drugs included in DeMuro's research were either designated fast-track, priority, or accelerated by the FDA, speeding up the approval process. This could inadvertently make it more difficult to fully build out patient experience information.
Robert Goldberg, PhD, vice president and co-founder of the Center for Medicine in the Public Interest, said he is not surprised by the lack of PRO in oncology drug labeling.
"That is to be expected since the population receiving the drugs are often different than those in clinical trials and have wide variations in response unique to small groups of patients. That is why doctors, not payers or armchair oncologists, should make decisions," said Goldberg.
Goldberg believes enhancing quality of life for patients goes beyond using PRO. "Unfortunately, only a small group of patients can get genomic testing that might help mitigate side effects or optimize treatment," he explained.
"The FDA can collect and help share data but we need to guide treatment at point of care and based on what patients need and respond to. Direct patient reporting to the FDA is one more hassle that every day oncologists don't have the time or luxury to participate in."
DeMuro and co. drew a different conclusion to their study's findings. "PROs should be included in development programs to capture a comprehensive evaluation of the study participants’ experience, which can provide useful information on the impact of the new therapy for patients, regulators, health care providers, caregivers, and payers who need to choose between competing therapies," the report said.