Current clinical research has seen an increased focus on patient-reported outcomes (PROs) as they provide unique information on the physical, functional and psychological effects of treatments from the perspective of patients themselves. Recent scientific research suggests that data from PRO studies can be successfully used for shared decision-making, to inform pharmaceutical labeling claims, and to influence the shaping of health policy and practice.
At the same time, the integration of patient-centered approaches into the health care system is taking place as an effort to make the healthcare system more efficient. The focus is shifting from traditional medicine to incorporating patients’ preferences and values to create a more personalized and effective approach to care. In this context, patient-reported outcomes are becoming an integral part of the strategy to improve the quality of care and achieve better patient outcomes.
The aim of this article is to identify the impact of patient-reported outcomes (PROs) on the progress of clinical medicine research as well as on Health Economics and Outcomes Research (HEOR) in Pharma.
The importance of patient-reported outcomes for medicine and society
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Patient-reported outcomes are data about the impact of a medical intervention on a patient obtained directly from the patient. These data include:
- physical condition;
- quality of life;
- psychological aspects;
- functional parameters.
In medicine and medical research, a major trend in the last decade has been a shift in focus from traditional clinical metrics to more meaningful patient-centered outcomes. Regulatory agencies are therefore increasingly looking to patients to understand how they describe their health status, given that patients are the experts in adapting to their disease or condition. Patient-reported outcomes allow the measurement of subjective aspects of a patient’s health status, such as pain, quality of life, or satisfaction with treatment.
Thus, patient self-reported outcomes play an important role in modern medicine by providing a unique perspective on the impact of treatment. They help regulators make decisions that best meet the needs of patients. Integrating PRO results into research and healthcare practice helps achieve a more personalized and targeted approach to treatment.
Integrating PROs into clinical trials
Clinical trial data helps regulators determine whether a new drug or treatment protocol is effective and safe. This data is collected based on objective metrics such as tumor size reduction and progression-free survival. However, these metrics do not always reflect patients’ subjective experiences, such as pain, quality of life, or satisfaction with treatment.
Clinical trials help determine how effective and safe a new drug or treatment protocol is. However, they often do not take into account how patients feel and manage their disease while participating in the trial. This is because clinical trials most regularly rely on outcomes reported by observers rather than patient-reported outcomes.
Once a drug is approved by a regulatory agency, patient-reported outcomes become an important tool for treatment decisions. For example, in oncology, where patients may have multiple treatment options, patient-reported outcomes can help select the treatment that is most effective and safest. In other words, if two treatments provide equivalent survival rates, patient-reported outcomes can help select the treatment that causes fewer side effects.
The results reported by the participants in clinical trials themselves not only reflect their experiences, but can also confirm the information on the drug’s packaging. This makes PROs especially valuable to drug manufacturers.
Therefore, today, patient-reported outcomes (PROs) can be included in clinical trials as primary or supplemental indicators. They are increasingly recognized by regulators, clinicians, and patients as a valuable source of information about how treatments affect patients. To ensure the high quality of PRO data, regulators and international organizations provide guidance on their collection, analysis, and interpretation.
Impact of PROs on drug development
Patient-reported outcomes have a significant impact on the drug development process. By taking PROs into account, drug developers gain a deeper understanding of how new drugs impact patients’ quality of life, physical condition and overall well-being.
PROs can become critical indicators of drug efficacy, contributing to the evaluation of not only clinical but also patient-centered aspects. This is particularly important in drug development for chronic diseases, where quality of life plays a significant role.
In addition, PRO results become an essential element of evidence when new drugs are submitted to regulatory authorities. Taking them into account provides a more objective view of the positive and negative aspects of treatment, and allows attention to be paid to those parameters that are truly significant to patients.
Thus, incorporating PROs into the drug development process expands the understanding of the efficacy and impact of medicines, making the process more focused on the real needs and expectations of patients.
PRO Measures
Patient-reported outcome measures are tools and methods for collecting information about patients’ perceptions and experiences regarding their health, disease states, and treatments. These measures are designed to measure aspects that can only be assessed by patients themselves, such as symptoms, quality of life, satisfaction with treatment, and other indicators important from the patient’s perspective.
Patient-reported outcome (PRO) measures can be categorized according to various criteria
Disease-specific:
- disease-specific — focus on specific characteristics and symptoms of a particular disease;
- generic — assess general aspects of quality of life independent of a specific disease.
By purpose of measurement:
- symptomatic — focused on measuring the patient’s symptoms and physical condition;
- functional — measures the patient’s ability to perform certain functions and activities;
- psychological — assesses psychological aspects such as emotional well-being and stress levels;
- social — measures the impact of the disease on social relationships and interactions with the world around them.
By type of questionnaires:
- general quality of life questionnaires — designed for a wide range of diseases;
- disease-specific questionnaires — designed for and tailored to specific diseases.
By time aspect:
- periodic — measure changes over a period of time;
- instantaneous — record the patient’s condition at a specific moment in time.
By the method of filling out:
- self-filled — questionnaires and patient diaries;
- interviewer-administered — open-ended interviews.
The choice of the type of PRO measure depends on the specific research objectives, the characteristics of the patient population, and the characteristics of the disease.
Challenges of including PROs in clinical trials
Despite the proven utility of including PROs to assess the efficacy, safety, and quality of life of treatments, there are several challenges to its implementation in clinical trials.
One major difficulty is that PROs are subjective measurements. This means that they can be influenced by factors such as mood, memory and patient understanding. To ensure the reliability and validity of PRO data, instruments for collecting and analyzing them must be carefully developed.
Another challenge is that collecting PRO data can be time-consuming and expensive. Patients may need to complete questionnaires or diaries on a regular basis. This can be particularly challenging for patients with severe medical conditions or disabilities.
Despite the challenges, the inclusion of PROs in clinical trials is becoming increasingly common. Researchers, regulators and international organizations are working to address the challenges associated with the use of PROs.
To cope with the fact that patient-reported outcomes may be too subjective, validated and approved instruments can be used. These have undergone special checks to make sure that the information they provide is indeed accurate and reliable.
Modern technologies, such as electronic data collection and mobile device surveys, are helping to reduce the labor and cost of PRO data collection.
Future directions in PRO data collection
Researchers and technology developers are working on new methods of PRO data collection that will solve existing problems and open up new opportunities.
One promising direction is the use of big data and artificial intelligence to analyze PROs. This can help researchers identify patterns that cannot be detected using traditional analysis methods. In addition, AI can help researchers identify patterns in data that may not be obvious to humans.
Another promising trend is the use of wearable devices to collect PRO data. Wearable devices such as fitness trackers and smartwatches can collect data on physical activity, sleep, heart rate, and other health metrics. This data can be used to evaluate the effectiveness of treatments and improve patients’ quality of life.
Work is also underway to develop new tools for collecting PRO data that will be more convenient and accessible to patients. For example, questionnaires are being developed that can be completed using voice control or gestures. Use of virtual reality (VR) and augmented reality (AR) for PRO data collection. VR and AR can be used to create a more engaging experience for patients, which can improve the accuracy and completeness of PRO data.
Conclusion
In modern medicine, it is not only traditional numbers and metrics that have become important, but also how patients themselves evaluate their health. Patient-reported outcomes (PROs) now play a key role in how we assess how well treatments are working. They help us better understand how medical procedures affect people’s usual lives and preferences. Using PROs helps make healthcare more adaptive and personalized. It also improves transparency in the healthcare system and provides the information needed to make informed decisions.
In sum, with the constant changes in the healthcare environment, attention to PROs becomes key to building efficient and sustainable healthcare systems that can combine quality and accessibility with economic viability.
