RESUMEN
The US Food and Drug Administration (FDA) understands the value of patient input in the regulatory decision-making process and has worked to enhance meaningful engagement. In recent years, there has been an increased scientific demand for more systematic and quantitative approaches to incorporate patient input throughout the medical product lifecycle, including to inform regulatory benefit-risk assessments. The use of patient preference information (PPI), elicited using established scientific methods, is a promising strategy for accomplishing this. Although much of the science behind PPI is not new, its application in a regulatory setting will require adapting and advancing the science of identifying, collecting, and evaluating patient input for informing regulatory decision making. Patient input and empowerment are foundational to a learning healthcare system. A learning healthcare system paradigm can also help us better understand and continuously improve the incorporation of the patient perspective in regulatory decision making. In this article, we highlight the Food and Drug Administration's Center for Biologics Evaluation and Research experience and current initiatives on advancing the science of patient input in a regulatory setting, in particular, PPI. We provide a use case that explores how the principles and benefits of PPI applied in shared clinical decision making can be realized and leveraged to enhance regulatory evaluation of innovative therapies. To further advance the application of the science of patient input in our regulatory framework, we compiled a list of example resources that support stakeholders in designing and conducting PPI studies. More collaborative research among stakeholders is needed to establish best practice approaches, ensure scientific validity, and continuously learn and improve the systematic incorporation of scientific patient input throughout the regulatory decision-making process.
RESUMEN
PURPOSE: To identify factors associated with early treatment discontinuation of three agents commonly prescribed for women with low bone density. METHODS: A telephone survey was conducted in 2000 to 2001 in a random sample of women aged 45 years or older who had bone density T-scores -1.0 or lower and who had initiated treatment with hormone replacement therapy, raloxifene, oral endronate. Logistic regression was used to estimate adjusted odds ratios for early treatment discontinuation. RESULTS: Among 956 women who were interviewed an average of 7 months after treatment initiation, 334 were taking hormone therapy, and 88 (26%) had discontinued; 256 were taking raloxifene, and 48 (19%) had discontinued (P = 0.03 vs. hormone therapy); and 366 were taking alendronate, and 70(19%) had discontinued (P = 0.02 vs. hormone therapy). Women with bothersome side effects (somewhat bothered: odds ratio [OR] = 4.0; 95% confidence interval [CI]: 2.5 to 6.5; very or extremely bothered: OR = 25; 95% CI: 16 to 39) or who thought that their bone density test results did not show osteoporosis (OR = 1.6; 95% CI: 1.0 to 2.5) were more likely to discontinue therapy, as compared with women reporting regular exercise (OR = 0.7; 95% CI: 0.4 to 1.0) or a willingness to take prescribed medications (OR = 0.6; 95% CI: 0.4 to 0.9). After adjustment for side effects and patient characteristics, the odds of early treatment discontinuation did not differ significantly among treatments. CONCLUSION: Improved adherence to osteoporosis treatment requires that treatment side effects be minimized and women be educated regarding their bone density test results.