CLINICAL RESEARCH METHODOLOGY
Year : 2010 | Volume
: 1 | Issue : 3 | Page : 104--105
Outcomes research and drug development
Head, Market Access & Pricing Emerging Markets Business Unit Pfizer Inc
Head, Market Access & Pricing Emerging Markets Business Unit Pfizer Inc
With increasing health care cost, focus needs to be given towards value-for-money, especially in the context of innovative drugs. A multi-disciplinary approach towards drug development is important in order to demonstrate the value of innovation to physicians and patients. Input into the drug development process at various stages of clinical trials must incorporate patient-focused endpoints and analyses. Demonstrating value of drugs will help ensure that innovative therapies should be seen as health care investment and not expense.
|How to cite this article:|
Duttagupta S. Outcomes research and drug development.Perspect Clin Res 2010;1:104-105
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Duttagupta S. Outcomes research and drug development. Perspect Clin Res [serial online] 2010 [cited 2020 Sep 30 ];1:104-105
Available from: http://www.picronline.org/text.asp?2010/1/3/104/71866
Outcomes Research (OR) is an interdisciplinary science combining principles of epidemiology, clinical research, health economics, quality of life assessment, and health policy. Clinical research answers the question that leads to Regulatory approval - "Is the product safe and efficacious?" Outcomes research answers the question that leads to Reimbursement approval - "Is the product worth paying for?" In other words, it helps address the question, "Given how much value I can already buy with low priced agents, how much additional cost am I paying per additional unit of benefit gained?"
Comparisons are based, at least in part, on costs and value (cost per unit of benefit). Pharmacoeconomic analysis is increasingly being used to assist decision makers in getting the biggest "bang for the buck" within cost-constrained healthcare budgets. An interesting aspect is that drug cost frequently is a small proportion of overall cost of managing the illness and in fact often helps in reducing overall cost or burden of illness. Such burden of illness data is crucial in order to identify the biggest driver of the cost of any disease, within a health care system.
Outcomes Research and Drug Development
During the drug development process, when a compound is identified in the research laboratory for further development, OR initiatives would usually include understanding the implication of the disease and the existing treatments that are currently available. Also, if there is any need to develop specific quality of life (QOL) or patientreported outcomes (PRO) questionnaires for further use to measure patient-reported outcomes, in subsequent clinical trials, those 'instruments' are also started getting developed with input from patients and physicians.
Next, a comprehensive burden of illness studies are developed in order to identify the biggest cost drivers, and the clinical and humanistic impact of the disease at a population level. This is either based on literature, in case of existing disease conditions or through a prospective, crosssectional study incase the disease is under-represented or under-diagnosed in any population. Using a comparison group of non-diseases patients, such burden of illness study helps to identify the various components of the disease from a health system, as well as from patient's perspective. From a health system's perspective, the results amplify how patients with specific physiological symptoms enter the system, how are they diagnosed, and treated. This also shows the various types of health services used (such as diagnostic tests, pharmacological and non-pharmacological interventions, etc.) for such patients, which in turn provides a cost estimate of treating these patients within the health system.
In the next phase, the PROs and/or QOL questionnaires are validated within clinical trial setting, and their clinical meaningfulness (i.e. inter-relationships between quality if life and clinical improvement) is ascertained, after consultation with physicians. At the same time, burden of illness studies are also evaluated and developed in various countries, to identify how the disease impacts at a population level.
In the critical Phase III stage, OR endpoints are incorporated into pivotal registration trials and subsequent analyses are incorporated into economic models to provide the value proposition of the compound. Communications about the OR and economic analyses are done via peerreviewed abstracts and publications in various medical and OR journals. All such information is combined to provide justification for access and reimbursement from various health care payers around the world.
Once the drug is approved by various regulatory agencies, attention is focused towards the evaluation of real-world effectiveness and health economic implications. Collaborative research projects are explored with various payers and providers to evaluate such data, which helps develop the value proposition of the drug outside the clinical trial environment.
Burden of Illness Information as a Critical Tool
Alleviating the clinical and humanistic burden of the disease is one of the principal reasons for which new treatments are developed by the bio-medical research community. BOI information is extremely important to demonstrate the population impact of the disease and highlight the principal cost drivers of any disease condition. As widely described in medical and health economic literature, cost of the pharmaceuticals is almost always the smallest component of the cost of illness of any disease. Majority of the cost drivers in any disease comes from other health care service utilizations and in a large number of disease areas, from the cost of hospitalizations. Therefore, treatment modalities that can alter the long-term clinical impact that can either lead to less institutionalization of patients or reduce any disease-related co-morbid conditions, or both, will always be preferred over any other. Since drug costs tend to be the smallest component of the total cost of the disease, investment should be made in innovative treatments that can lead to reduction of long-term cost and burden for the patients, to the health care systems, and to the government.
Once the patients manifest the disease, focus should be given to preventing the long-term health care-related cost and burden that may arise if these patients are not properly treated with innovative medicines. Prevention is always more cost-effective than treatment during the lifetime of the patient. As economies grow, and productivity of its population flourishes, more emphasis should be given to reduce the longterm burden of diseases to the society. Identifying the burden and cost of major illness should be the right direction in this endeavor.
Innovative Medicines are Investments, not Expenses
Attention should also encompass the quality of life of the individuals, and identify opportunities to improve the healthrelated quality of life by providing the most innovative treatment options available. As such, innovative medicines should be viewed as an investment to improve patient's health, and not as an expense. In stead of focusing only on shortterm cost, emphasis should be given towards evaluating the long-term impact of interventions that will prove worthwhile not only for patients, but also for improving the general health status of populations and the country. Especially, in the area of chronic diseases, by preventing costlier health situations (such as extended hospitalizations or additional morbidities) in the future, the health care system can certainly benefit from by taking a long-term view of the health care cost.
Outcomes Research is a growing discipline in the health care area, and collaborative research with academic institutions and key opinion leaders is extremely essential to provide arguments for effective health care resource allocation.
This article reflects the speaker's personal views only, and may not represent the views of Pfizer Inc.
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