by Patrick Chassaigne
These days, pharmaceutical and biotechnology companies need to demonstrate innovation and differentiation as they launch new products to market. Some of the critical questions asked by regulatory agencies, insurance payers, government health authorities, physicians and patients are:
- What is the value of this medication?
- How does it compare to other treatments?
- Is it worth the cost?
- For which patient population is it best used?
- How can we optimize outcomes?
Patients are at the center of this debate since there is a growing desire on their part to be more empowered in their search for quality care, to be informed of alternatives for potential treatment and to access affordable health care and health benefits. Patients are understandably driven to improve their own quality of life, and are motivated to know more about their particular disease and how to manage it.
The creation of the U.S.-based Patient-Centered Outcomes Research Institute (PCORI) embodies this new role of the patient. As an independent, nonprofit organization, PCORI conducts research to assist health care decisions by providing evidence on the effectiveness, benefits and harms of different treatment options for patients. This research recognizes that the patient’s voice should be heard, and that their experiences should be taken into consideration as part of the health care decision process.
One of the principle duties of PCORI is to identify priorities for research. This work is guided by the following four questions asked from a patient’s perspective:
- Given my personal characteristics, conditions and preferences, what should I expect will happen to me?
- What are my options and what are the benefits and harms of these options?
- What can I do to improve the outcomes that are most important to me?
- How can the health care system improve my chances of achieving the outcomes I prefer?
To provide answers to these questions, PCORI will conduct outcomes research studies that will collect data on patient experiences as well as other important factors affecting their health. Considering the significant momentum behind this new initiative, Phase IV observational studies and patients registries will likely become increasingly important, not only in the U.S. but in all major countries in Europe and Asia.
The Benefits of Implementing Phase IV Studies and Registries
Pharmaceutical companies recognize that implementing real-world, observational studies will complement data collected in pre-approval trials. Through properly designed Phase IV studies and registries, additional information about the long-term safety and side effects of new products will be obtained as well as insight into product effectiveness and utilization in a variety of patient populations.
Since companies need to know how products are affecting patients in their own environment, they capture patient reported outcomes (PROs) using web-based technologies to document quality of life, adverse events and real-time patient health status directly from the patient, caregiver, parent, spouse or observer.
In addition to providing basic health assessment data, patients may offer information that can help the drug manufacturer better understand product utilization, medication compliance and treatment satisfaction. Patients can also identify any occurrence of major symptoms, any events linked to disease progression and any potential safety concerns or adverse events issues.
Patient questionnaires or surveys can be taken in the comfort of the patient’s home or during a routine visit to their health care provider, if appropriate. Depending on the scope of the study, a health care provider or a pharmacist may invite the patient to answer questionnaires or take part in a study. It is also feasible to have an electronic data capture (EDC) system auto-enroll patients into a web-based patient reported outcomes solution following a certain set of predefined criteria. As a third scenario, the patient may take the opportunity to enroll directly into a study following a recruitment campaign that provides all the necessary information such as the scope of the study, procedures regarding patient consent, benefits for participating in the study and the type of data requested to be entered by the patient.
In addition, patients need to be able to use familiar devices such as a home computer or mobile phone and enter data via an Internet browser of their choice. In most cases, the interaction with patients needs to be in the local language for ease of use and to keep them interested in continuing with the study.
Interactive disease management portals represent another key opportunity to increase effectiveness of Phase IV studies and patient registries, especially if many sites are involved or if a well-defined patient population is targeted by the study. Rare diseases registries are good examples of studies that can benefit from implementing a disease management platform. This approach will allow physicians, patients and pharmaceutical companies to stay connected and share information quickly and easily. While contributing data to the study, patients can also retrieve up-to-date information on their disease, communicate with other patients and feel part of a community.
With an ever-increasing percentage of the population having access to the Internet, the global use of clinical technology to capture patient outcomes or facilitate participation in a disease management portal platform can lead to a better experience and increased convenience for patients. This translates to higher patient retention and improved data collection and quality for pharmaceuticals companies when implementing Phase IV studies and registries, a win/win for patients and manufacturers.
With the constant influx of new technologies and process optimization techniques, pharmaceuticals companies are faced with many opportunities to innovate and change the way they interact with patients as they design new studies to capture real-world data.
Patrick Chassaigne is director of late phase solutions at Medidata Solutions.