Project description:IntroductionPatient registries, 'organised systems that use observational methods to collect uniform data on a population defined by a particular disease, condition, or exposure, and that is followed over time', are potentially valuable sources of data for supporting regulatory decision-making, especially for products to treat rare diseases. Nevertheless, patient registries are greatly underused in regulatory assessments. Reasons include heterogeneity in registry design and in the data collected, even across registries for the same disease, as well as unreliable data quality and data sharing impediments. The Patient Registries Initiative was established by the European Medicines Agency in 2015 to support registries in collecting data suitable to contribute to regulatory assessments, especially post-authorisation safety and effectiveness studies.MethodsWe conducted a qualitative synthesis of the published observations and recommendations from an initiative-led multi-stakeholder consultation and four disease-specific patient registry workshops. We identified the primary factors facilitating the use of registry data in regulatory assessments. We generated proposals on operational measures needed from stakeholders including registry holders, patients, healthcare professionals, regulators, marketing authorisation applicants and holders, and health technology assessment bodies for implementing these.ResultsTen factors were identified as facilitating registry use for supporting regulatory assessments of medicinal products. Proposals on operational measures needed for implementation were categorised according to three themes: (1) nature of the data collected and registry quality assurance processes; (2) registry governance, informed consent, data protection and sharing; and (3) stakeholder communication and planning of benefit-risk assessments.ConclusionsThese are the first explicit proposals, from a regulatory perspective, on operational methods for increasing the use of patient registries in medicines regulation. They apply to registry holders, patients, regulators, marketing authorisation holders/applicants and healthcare stakeholders broadly, and their implementation would greatly facilitate the use of these valuable data sources in regulatory decision-making.

Project description:BackgroundTotal joint arthroplasty (TJA) registries traditionally have focused on implant longevity and rates of revision surgery. Registries would benefit from the addition of standardized patient-reported outcomes (PROs) such as pain relief and improved physical function. However, PROs have not been routinely adopted, and their incorporation into TJA registries presents challenges.Questions/purposesWe review current PRO use by existing national registries, challenges to integrating PROs in national registries, lessons from national registries that have integrated PROs, and suggestions to guide future adoption of PROs.MethodsWe conducted a literature search of papers addressing PRO use in national knee and hip arthroplasty registries, resulting in 15 articles. These publications were supplemented by discussions with thought leaders from international registries. WHERE ARE WE NOW?: Some national TJA registries are collecting PROs and valuable research is emerging. However, challenges exist, such as selecting suitable PROs, selection bias in countries without government-mandated participation for all hospitals, and challenges with missing data. WHERE DO WE NEED TO GO?: The ideal system will incorporate PROs into TJA registries. In so doing, it will be important to choose suitable PROs and develop innovative methods to collect PROs to ensure complete data and sustainability. HOW DO WE GET THERE?: New methods are required to meet the challenges related to registry design, logistics of PRO collection, and registry cost and sustainability. Modifications to the traditional hospital- and implant-centric design and new procedures to collect complete data from both patients and clinicians may be necessary. For instance, England and Wales, New Zealand, and Sweden developed methods to collect PROs after TJA directly from patients and a US TJA registry collects PROs as the primary outcome. Finally, to assure long-term sustainability, PRO data must be valuable to multiple stakeholders, including patients, clinicians, researchers, and policy makers.

Project description:BackgroundEnrollment in pregnancy registries is challenging despite substantial awareness-raising activities, generally resulting in low recruitment owing to limited safety data. Understanding patient and physician awareness of and attitudes toward pregnancy registries is needed to facilitate enrollment. Crowdsourcing, in which services, ideas, or content are obtained by soliciting contributions from a large group of people using web-based platforms, has shown promise for improving patient engagement and obtaining patient insights.ObjectiveThis study aimed to use web-based crowdsourcing platforms to evaluate Belimumab Pregnancy Registry (BPR) awareness among patients and physicians and to identify potential barriers to pregnancy registry enrollment with the BPR as a case study.MethodsWe conducted 2 surveys using separate web-based crowdsourcing platforms: Amazon Mechanical Turk (a 14-question patient survey) and Sermo RealTime (a 11-question rheumatologist survey). Eligible patients were women, aged 18-55 years; diagnosed with systemic lupus erythematosus (SLE); and pregnant, recently pregnant (within 2 years), or planning pregnancy. Eligible rheumatologists had prescribed belimumab and treated pregnant women. Responses were descriptively analyzed.ResultsOf 151 patient respondents over a 3-month period (n=88, 58.3% aged 26-35 years; n=149, 98.7% with mild or moderate SLE; and n=148, 98% from the United States), 51% (77/151) were currently or recently pregnant. Overall, 169 rheumatologists completed the survey within 48 hours, and 59.2% (100/169) were based in the United States. Belimumab exposure was reported by 41.7% (63/151) patients, whereas 51.7% (75/145) rheumatologists had prescribed belimumab to <5 patients, 25.5% (37/145) had prescribed to 5-10 patients, and 22.8% (33/145) had prescribed to >10 patients who were pregnant or trying to conceive. Of the patients exposed to belimumab, 51% (32/63) were BPR-aware, and 45.5% (77/169) of the rheumatologists were BPR-aware. Overall, 60% (38/63) of patients reported belimumab discontinuation because of pregnancy or planned pregnancy. Among the 77 BPR-aware rheumatologists, 70 (91%) referred patients to the registry. Concerns among rheumatologists who did not prescribe belimumab during pregnancy included unknown pregnancy safety profile (119/169, 70.4%), and 61.5% (104/169) reported their patients' concerns about the unknown pregnancy safety profile. Belimumab exposure during or recently after pregnancy or while trying to conceive was reported in patients with mild (6/64, 9%), moderate (22/85, 26%), or severe (1/2, 50%) SLE. Rheumatologists more commonly recommended belimumab for moderate (84/169, 49.7%) and severe (123/169, 72.8%) SLE than for mild SLE (36/169, 21.3%) for patients trying to conceive recently or currently pregnant. Overall, 81.6% (138/169) of the rheumatologists suggested a belimumab washout period before pregnancy of 0-30 days (44/138, 31.9%), 30-60 days (64/138, 46.4%), or >60 days (30/138, 21.7%).ConclusionsIn this case, crowdsourcing efficiently obtained patient and rheumatologist input, with some patients with SLE continuing to use belimumab during or while planning a pregnancy. There was moderate awareness of the BPR among patients and physicians.

Project description:BackgroundIn the Philippines, one in four pregnancies are unintended and 610 000 unsafe abortions are performed each year. This study explored the association between missed opportunities to provide family planning counseling, quality of counseling and its impact on utilization of effective contraception in the Philippines.MethodsOne-hundred-one nationally representative health facilities were randomly selected from five levels of the health system. Sexually-active women 18-49 years old, wanting to delay or limit childbearing, attending primary care clinics between April 24 and August 8, 2017 were included. Data on contraceptive use, counseling and availability were collected using interviews and facility assessments. Effective contraceptive methods were defined as those with rates of unintended pregnancy of less than 10 per 100 women in first year of typical use.Findings849 women were recruited of whom 51.1% currently used effective contraceptive methods, 20.6% were former effective method users and 28.3% had never used an effective method. Of 1664 cumulative clinic visits reported by women in the previous year, 72.6% had a missed opportunity to receive family planning counseling at any visit regardless of level of facility, with 83.7% having a missed counseling opportunity on the day of the interview. Most women (55.9%) reported health concerns about modern contraception, with 2.9% receiving counseling addressing their concerns. Only 0.6% of former users and 2.1% never-users said they would consider starting a modern contraceptive in the future. Short and long acting reversible contraceptive methods were available in 93% and 68% of facilities respectively.ConclusionsMissed opportunities to provide family planning counseling are widespread in the Philippines. Delivery of effective contraceptive methods requires that wider legal, policy, social, cultural, and structural barriers are addressed, coupled with systems approaches for improving availability and quality of counseling at all primary health care contacts.

Project description:PurposeTo characterize pediatricians' perceived barriers and areas of confidence in assessing patient-reported outcomes (PROs) in the U.S., and to test associations of these factors with implementing PRO assessment.MethodsUsing a random sample from the members of American Medical Association, we recruited general pediatricians and pediatric subspecialists to complete a survey (July 2011 to December 2013). Perceived barriers and confidence in PRO assessment were compared by age, pediatric specialty (general pediatrics, seven subspecialties), practice settings (academic, private), and region of practice. Multivariable logistic regressions tested associations of demographic factors, barriers, and confidence factors with the implementation of PRO assessment.FindingsThe survey was completed by 458 participants (response rate 48.5%); of these, 40.4%, 15.9%, 15.5%, and 8.1% were general pediatricians, cardiology, hematology/oncology, and pulmonary specialists, respectively. PRO assessment was implemented by 29.0% of the pediatricians. The top five barriers for PRO assessment included limited time/manpower (79.0%), limited training (77.4%), lengthy PRO instruments (76%), lack of meaningful cut-offs on PRO scores (75.5%), and unavailable PRO instruments (75.0%). Limited knowledge of PROs (OR 4.10; 95% CI 2.21, 7.60) and unavailability of PRO instruments (OR 1.87; 95% CI 1.01, 3.49) increased the odds of not implementing the assessment, whereas confidence in PRO assessments compatible with norms (OR 0.41; 95% CI 0.23, 0.72) and perceived benefit over clinical judgment alone (OR 0.53; 95% CI 0.31, 0.93) decreased the odds of not implementing the assessment.Interpretationsignificant barriers to PRO assessment in pediatric settings suggest the need for providing training, resources, and practical guidance toward implementation. Patient or Public Contribution: healthcare service users contributed to this study by completing a survey and providing feedback about the barriers and areas of confidence in assessing PROs for pediatric populations.

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Barriers and Opportunities for Use of Patient Registries in Medicines Regulation.

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