The Importance of Patients Perspectives:  Patient Reported Outcome Measures

What are late-phase clinical studies and why are they important?

Late-stage clinical studies are postmarketing trials classified as Phase IIIb and Phase IV that are conducted to establish a drug’s long-term safety and efficacy following regulatory approval, and to provide product differentiation and positioning in the competitive drug market place.

As with Phase II and Phase III studies, patients represent valuable data, therefore retention of patients, so that they are not lost to follow-up (LTFU) is equally important in later phase studies to assure appropriate data collection and dissemination of results.

Is there a difference between Phase IIIb and Phase IV studies?

Yes.  Phase IIIb are typically conducted to provide head-to head comparison data, confirmation of earlier results prior to product approval and/or to gain approval of the drug for an additional therapeutic indication.  Phase IV studies are conducted for several postmarketing objectives such as pharmacovigilance, patient registries, disease and risk management, and patient reported outcome.

What are the implications of patient reported outcomes (PRO)?

Studies have shown that attention to patient feedback on healthcare outcomes and patient behavior change can lead to improved treatment adherence and outcomes.¹ Applying such results to all early and late phase clinical studies, can help retain patients so that they are not LTFU.

What do PRO measure?

PRO measures are used to assess several factors such as health-related quality of life (HRQOL), treatment satisfaction, and disease symptoms and include and use endpoint derived from patient reports collected from several sources such as investigator sites, patient diaries, questionnaires, etc.²

What are some examples of PRO outcomes?

PRO endpoints in late-phase trial are used to gain patients’ perspectives in several areas such as support of secondary indications for an approved product label; revised guideline treatment recommendations and addition of an agent or tier advancement in manage care formulary.³

Notably, regardless of the clinical development and postmarketing programs designed for the lifecycle of a drug, valuable patient efficacy and safety data along with their treatment perspectives are critical for the long-term longevity of a product. Therefore patient retention efforts including a process to find those LTFU are essential to insure such success.

What is OmniTrace and how can it assist drug manufacturers in finding early and late stage clinical trial patients LTFU?

Omnitrace is a people locate company that has been in operation for 8 years.  Omnitrace has a stellar record of success in locating thousands of people worldwide, servicing several sectors including the pharmaceutical/biotechnological industry in finding clinical trial patient dropouts and those LTFU.  As mentioned, as every participant in clinical research is a valuable source of safety information, locating patients Lost To Follow-Up (LTFU) is critical to maintaining a robust risk management system.

References

1. Institute of Medicine, Crossing the Quality Chasm-A New Health System for the 21^st Century, 1^st Edition National Academies Press, Washington, DC, 2001

2. R.J. Willke, L.B. Burke, P. Erickson, “Measuring Treatment Impact: A Review of Patient-Reported Outcomes and Other Efficacy Endpoints in Approved Product Labels,” Controlled Clinical Trials, 25 (6) 535-552 (2004).

3. E.P. Rock, D.L. Kennedy, M.H. Furness et al., “Patient-Reported Outcomes Supporting Anticancer Product Approvals,” Journal of Clinical Oncology, 25 (32) 5094-5099 (2007).