The integration of patient perspectives and experiences is becoming increasingly important in today’s clinical research, especially during the development of health products or services. Indeed, regulatory authorities such as FDA or EMA are increasingly demanding this type of data coming directly from patients in order to approve products that meet a real need for patients.
This type of data coming directly from patients can be generated thanks to patient-reported outcome (PROs). PROs are defined by the FDA as “any report of the status of a patient’s health condition that originates directly from the patient, without interpretation by a clinician or any other party.” PRO instruments are validated questionnaires or scales that ask patients to report their symptoms, physical functioning, emotional well-being, preferences and overall quality of life.
Progressively, longitudinal observational and experimental studies have incorporated more and more data coming directly from patient’s experience, especially thanks to PROs.
PROs complete traditional clinical and biological data in assessing not only the safety or effectiveness of a product but also the real-life experiences of the patients. Traditional clinical endpoints such as survival or tumor shrinkage are still essential, but it is also important to consider the impact of the disease and treatment on patients’ health-related quality of life. Using PROs in healthcare is pivotal to support shared decision-making, compare interventions, improve patient care, influence regulatory decisions and optimize health economic evaluation, amongst others.
How to choose the right PRO?
Because of the growing need for data coming directly from the patient for the development of healthcare products and services development, choosing the right PRO has become a critical step in the process and it raises the question of “How to choose the right PRO?”
Patients provide insights in a variety of domains like symptoms (such as pain, fatigue), functional status (like sexual, bowel, or urinary functioning), wellness (physical, mental, social), quality of life, or satisfaction with care or treatment. To improve decision-making in patient-centered outcomes research (PCOR), it is essential to measure these PROs in a standardized manner but always adapted to the context (pathology, product or service concerned, patient population, profile) using validated questionnaires that demonstrate specific measurement properties.
Due to the wide variety of instruments available, it can be complex to identify the most appropriate and suitable PROs to use in a specific context. There is no consensus on a standardized method for selecting PROs, considering the scientific background and the regulatory recommendations.
How Carenity can help you?
According to Carenity, the selection of the most effective PROs should take into account patients’ needs in addition to scientific and regulatory expectations. This is why Carenity has developed a method named Patient-Driven Outcome Selection (PDOS): A patient-focused approach for selecting outcomes using patient-generated data that are representing meaningful domains for patients as best as possible.
This patient-centric, structured, replicable and generalizable method for PROs selection and development using patient-generated data was developed and tested as pilots in 3 therapeutic areas: lung cancer, breast cancer and diabetes.
PDOS methods
The PDOS method is a four-step methodology:
Firstly, it is important to start with a literature review of patient-centered researches and clinical trials to determine which PROs are frequently used in a specific therapeutic area.
Secondly, outcomes and sub-domains identified are put into perspective with medical experts’ perceptions available health guidelines, and HTA bodies’ expectations.
Then, in the third stage, an extensive direct-to-patient survey aims to rank the domains by order of importance from the patient perspective and highlight potential additional domains to be assessed.
Finally, a gap analysis is performed to identify the most suitable PROs to use.
Application example of PDOS method in breast cancer
Regarding breast cancer, the literature review showed that some outcomes were well covered by existing PROs: Quality of life (QoL), survival vs QoL considerations, satisfaction with breast(s) and cancer healthcare pathway. On the other hand, some outcomes were partially or poorly covered (copying strategies, satisfaction and confidence in decision making). Moreover, experts’ groups recommended to consider several profile attributes QoL in breast cancer context (cancer stage, treatment strategy, complementary care, etc.).
The direct-to-patient online survey launched in several countries among the breast cancer Carenity patient communities contained socio-demographic and medical questions, as well as questions aiming to rank the previous outcomes and identifying new ones. Needs and expectations appeared to be slightly different between domains covered by PROs and domains really valued by patients with breast cancer.
Indeed, for instance coping strategies (attitudes and behaviors used to maintain emotional well-being and to adjust to the stresses caused by cancer) and burden of the disease (lack of energy, ability to feel like a woman, pain, weight loss) were only considered as intermediate domains for current PROs whereas it was a major domain for patients. And on the opposite, satisfaction with cancer healthcare pathway was classify as a major domain in current PROs whereas it is just a minor domain from patients’ perspectives.
This first example highlights the gap between existing PROs and real patients ‘perspectives bringing to light the fact that always having in mind patient centricity is key to answer patients needs.
Application example of PDOS method in lung cancer
Regarding lung cancer, the literature review showed that in current PROs major domains are daily life, physical functioning and well-being, emotional well-being, medical care and treatment care. Medical care and Physical functioning and well-being were also considered as major domains for patients and caregivers in the online questionnaire, but end-of-life care and caregivers’ perspectives were major domains for patients.
This second example with a different disease highlights the same point as the first example.
Testing this methodology in multiple therapeutic areas showed that this approach allows us to highlight differences according to patients’ profile and disease, and to identify domains that are under-assessed in frequently used PROMs.
Conclusion
The increasing importance of Health-Related Quality of Life (HR-QoL) in healthcare decision-making reinforces the need to choose relevant PRO measures.
The PDOS method developed by Carenity is a patient-centric and structured approach to identify the most appropriate instrument according to patients, physicians and HTA bodies. This method can support Evidence Generation Plans, optimize the selection of study endpoints, facilitate HTA discussions by backing-up the measured outcomes and encourage shared decision-making when used accros the care pathway.
Figure 1: Key steps of the PDOS method (Patient-Driven Outcome Selection)
