Understanding the importance and value of Patient-reported outcomes

by Francesco Patalano

I am a physician, a Pharma R&D executive and, from time to time, a patient – we all are. As a patient, I sometimes find it difficult to describe to the doctor treating me the subjective impact of a disease or the effect of a medication on my overall wellbeing.

The FDA defines Patient-Reported Outcomes (PRO) as reports of a patient’s health condition and health-related quality of life that comes directly from the patient, without anyone else’s interpretation. Because some effects of the disease are known only to the patients, collecting the information directly from the patient who experiences them is absolutely critical to measure the impact of a disease or the efficacy of a drug.

Pain, itch, lack of energy and tiredness are common situations for patients suffering from a number of conditions. However, they are only known to the patients who experience them and cannot be measured by a laboratory test.

Only a patient can describe an episode of migraine affecting the ability to work or the relationship with other family members. These events may be the only manifestation of a disease and will not be measured by any laboratory analysis!

PROs are tools to standardize collection of this information. PROs include information about health-related quality of life, symptoms, functional status, satisfaction with treatments, adherence to treatment, and perceived value of treatment. They are therefore powerful tools to empower patients to engage in their own health care over time. PROs are also important to inform and advance health care decisions based on clear evidences, which will ultimately affect them and many other patients as well.

Using PRO instruments, patients can convey clearly to their physicians their subjective feelings and concerns, including positive or negative effects on quality of life and what they consider as important. Vice-versa, information on what to expect in the course of a disease or following a treatment can be communicated to the patients using the PRO information as starting point and reference. Thus, PROs are very effective in promoting dialogue between physicians and their patients using a language that is understood by both.

Both the patient and medical community should expand the use of PROs to improve overall communication and clinical decision-making. It is, however, important to align on the purpose of data collection, how this data is stored, and the reporting and analyses of the data to avoid any ethical concerns, optimize resources and not overburden patients and implementers.

More efforts are needed to develop PRO instruments in areas of unmet needs. It is, however, undeniably clear that PROs are invaluable to all the stakeholders right from effective outcomes measurement, addressing patient needs, assessing drug efficacy, and discussions concerning the access to medicines. They are the key driver in the patient-centric era of outcomes driving medical decisions and drug development, enabling patients to have a central role in the process.

In considering PRO a critical part of the Health Data – free to flow, but ultimately owned by the patients – the IMI H2O Project will improve communication between individual patients and their physicians and will facilitate value-based healthcare decisions. I am delighted and proud to be part of this exciting journey.