Patient-reported Outcomes as a Source of Evidence in Off-Label Prescribing: Analysis of Data From PatientsLikeMe

Abstract

Background Evaluating a new use for an existing drug can be expensive and time consuming. Providers and patients must all too often rely upon their own individual-level experience to inform clinical practice, which generates only anecdotal and unstructured data. While academic-led clinical trials are occasionally conducted to test off-label uses of drugs with expired patents, this is relatively rare. In this work, we explored how a patient-centered online research platform could supplement traditional trials to create a richer understanding of medical products postmarket by efficiently aggregating structured patient-reported data. PatientsLikeMe is a tool for patients, researchers, and caregivers (currently 82,000 members across 11 condition-based communities) that helps users make treatment decisions, manage symptoms, and improve outcomes. Members enter demographic information, longitudinal treatment, symptoms, outcome data, and treatment evaluations. These are reflected back as longitudinal health profiles and aggregated reports. Over the last 3 years, patients have entered treatment histories and evaluations on thousands of medical products. These data may aid in evaluating the effectiveness and safety of some treatments more efficiently and over a longer period of time course than is feasible through traditional trials.Objective The objective of our study was to examine the illustrative cases of amitriptyline and modafinil – drugs commonly used off-label.Methods We analyzed patient-reported treatment histories and drug evaluations for each drug, examining prevalence, treatment purpose, and evaluations of effectiveness, side effects, and burden.Results There were 1948 treatment histories for modafinil and 1394 treatment reports for amitriptyline reported across five PatientsLikeMe communities (multiple sclerosis, Parkinson's disease, mood conditions, fibromyalgia/chronic fatigue syndrome, and amyotrophic lateral sclerosis). In these reports, the majority of members reported taking the drug for off-label uses. Only 34 of the 1755 (1%) reporting purpose used modafinil for an approved purpose (narcolepsy or sleep apnea). Only 104 out of 1197 members (9%) reported taking amitriptyline for its approved indication, depression. Members taking amitriptyline for off-label purposes rated the drug as more effective than those who were taking it for its approved indication. While dry mouth is a commonly reported side effect of amitriptyline for most patients, 88 of 220 (40%) of people with amyotrophic lateral sclerosis on the drug reported taking advantage of this side effect to treat their symptom of excess saliva.Conclusions Patient-reported outcomes, like those entered within PatientsLikeMe, offer a unique real-time approach to understand utilization and performance of treatments across many conditions. These patient-reported data can provide a new source of evidence about secondary uses and potentially identify targets for treatments to be studied systematically in traditional efficacy trials.

Patient- or Participant-Generated Registries

“…12,93 There is a concern that PGR registry participants may be those individuals who feel the strongest -positively or negativelyabout a particular treatment, thus producing a "reporting bias." 94 To the extent that registry participants may participate in more than one registry or study, some individuals may be overrepresented in reports of multiple studies, especially in the context of a rare disease, though this is more difficult to quantify and also occurs in traditional registries and clinical studies.…”

Digital Social Networks and Health

“…31,32 • How can online patient communities be engaged with researchers to evaluate medical interventions and conduct postmarket surveillance studies of medical drugs and devices? 59 Already, Internet communities (such as mailing lists, chat rooms, newsgroups, and discussion boards) are rich sources of qualitative data for health researchers that can help them assess the needs, values, and preferences of consumers and professionals relevant to health and health care. 60 To meet the research objectives outlined above, we also look forward to advanced data sets and tools that respect personal privacy and patient confidentiality rights to be made available by various SNS in collaborative research endeavors.…”

Internet : un facteur de transformation de la relation médecin-patient ?

“…Sur certains sites, comme celui de Patients like me (Frost et al, 2011), les données médicales personnelles fournies par les patients sont agrégées dans le but d'en extraire de nouvelles connaissances. Certaines associations de patients s'impliquent d'ailleurs désormais dans un activisme prenant appui sur les données probantes ain de fonder leur légitimité auprès des pouvoirs de santé publique, de la recherche médicale et des irmes pharmaceutiques (Rabeharisoa, Moreira et Akrich, 2013).…”