Not long ago, the challenges of bringing pharmaceutical products to market ended with regulatory approval. The picture has completely changed now, with companies facing greater challenges post approval. There is now a greater awareness about the benefits and risk of products, not only among health care professionals (HCPs), but also among consumers and payers. In addition, payers are not laying more emphasis on patient perspective. There is increasing demand from all interested parties for more evidence than mere safety and efficacy data from clinical trials.
It has become more important to measure the effectiveness of treatments and comparative effectiveness relative to other therapies. To do so requires real-world data, such as Patient Reported Outcomes (PROs) that are based on longer follow-up and are a more representative of the patient population than is typically present in clinical trials.
The primary source of this data comes from claims databases, disease registries and electronic health records (EHRs). However, there is genuine concern regarding the quality of readily available real-world data due to missing data and inadequate validation. To rectify this, more outcomes data has to be generated in a planned manner to provide comprehensive evidence of the value of the drug.
PROs reported directly by the patient like symptoms, quality of life (QoL), or patient-perceived health status are highly effective tools for providing relevant data to patients, clinicians, and policy-makers, especially about morbidity and ‘patient suffering’ in case of chronic diseases. Patient-reported outcomes allow for a more holistic approach to clinical decision-making, which can improve the overall quality of patient care.
Data collected via PROs and other similar tools is more compelling than survival or even progression-free survival data, when capturing Quality of Life (QoL) data. QoL data rose into prominence a number of years ago with the rise in incidence of oncology indications, high drug prices and little or incremental gains in survival rates offered by new drugs. The fundamental premise now is that health-related QoL and well-being of patients is a core co-primary endpoint in clinical research and clinical care.
Outcomes research (OR) studies provide end results of medical treatment and care, in terms of the impact on health and well-being of patients and general populations. The scope of OR encompasses studies that evaluate effectiveness of treatments, development and use of tools to measure health status and analysis and dissemination of the results. OR uses effectiveness research rather than efficacy studies, to evaluate the results of the healthcare process in the real world and assesses which treatments for specific problems work best for whom. It also factors in patient preferences and patient satisfaction. Governments, insurance companies, employers and consumers now progressively seek OR data for better decision-making.
Furthermore, regulators are making approval decisions based on outcomes data. For example, when NICE (National Institute for Health and Care Excellence) reviewed Novartis’ asthma drug Xolair, it considered data from the Asthma QoL questionnaire in observational studies and overturned an earlier decision to reject Xolair . PRO health surveys have also been used for label claims (e.g., Humira, Allegra, Lyrica) . PRO data can help influence pharmacy benefit managers and insurers to include a drug in their formularies. Health surveys can also be used to answer any questions regarding comparative effectiveness in order to build an economic basis for formulary inclusion, thus helping to lower claim costs over time. Companies successfully use PROs to prove the positive impact of a product on patient health and ultimately health expenses.
Due to its potential in addressing the interrelated issues of cost and quality of healthcare, public and private sector interest in outcomes has burgeoned the past few years. Outcomes research touches all aspects of health care delivery, from the clinical encounter itself to aspects of the organization, financing and regulation of the health care system. Each of these factors plays a role in the outcome of care, or the ultimate health status of the patient. Understanding how the different factors interact requires collaboration among a broad range of health services researchers, such as physicians and nurses, economists, sociologists, political scientists, operations researchers, biostatisticians and epidemiologists.
- Nick Taylor, 2014. Analyzing real-world data for Lifecycle Management. (Internet) http://www.fiercebiotech.com/offer/analyzingdata?source=listing [Accessed 12/01/2015]
- Gus Gardner, 2009. The Trial Never Ends. (Internet) https://www.optum.com/content/dam/optum/resources/articles/TheTrialNeverEnds08.11.09.pdf [Accessed 15/12/2014]