8 results
Development of an international template to support patient submissions in Health Technology Assessments
- Nigel Cook, Heidi Livingstone, Jennifer Dickson, Louise Taylor, Kate Morgan, Martin Coombes, Sally Wortley, Elisabeth Oehrlein, María José Vicente-Edo, Franz Waibel, Barry Liden
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- Journal:
- International Journal of Technology Assessment in Health Care / Volume 37 / Issue 1 / 2021
- Published online by Cambridge University Press:
- 01 April 2021, e50
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Objectives
To develop an international template to support patient submissions in Health Technology Assessments (HTAs). This was to be based on the experience and feedback from the implementation and use of the Scottish Medicines Consortium's (SMC) Summary Information for Patient Groups (SIP).
MethodsTo gather feedback on the SMC experience, web-based surveys were conducted with pharmaceutical companies and patient groups familiar with the SMC SIP. Semistructured interviews with representatives from HTA bodies were undertaken, along with patient group discussions with those less familiar with the SIP, to explore issues around the approach. These qualitative data informed the development of an international SIP template.
ResultsSurvey data indicated that 82 percent (18 of 22 respondents) of pharmaceutical company representatives felt that the SIP was worthwhile; 88 percent (15/17) of patient group respondents found the SIP helpful. Both groups highlighted the need for additional support and guidance around plain language summaries. Further suggestions included provision of a glossary of terms and cost-effectiveness information. Patient group interviews supported the survey findings and led to the development of a new template. HTA bodies raised potential challenges around buy-in, timing, and bias connected to the SIP approach.
ConclusionsThe international SIP template is another approach to support deliberative processes in HTA. Although challenges remain around writing summaries for lay audiences, along with feasibility considerations for HTA bodies, the SIP approach should support more meaningful patient involvement in HTAs.
PP177 Health Preference Research In Europe: A Review Of Its Use
- Kevin Marsh, Axel Mühlbacher, Janine van Til, Christin Juhnke, Yookyung Christy Choi, Alejandra Duenas, Wolfgang Greiner, Kara Haas, Natalia Hawken, Mickael Hiligsmann, Kimberley Hockley, Ilya Ivlev, Frank Liu, Elizabeth Molsen-David, Elisabeth Oehrlein, Jan Ostermann, Thomas Poder
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- Journal:
- International Journal of Technology Assessment in Health Care / Volume 35 / Issue S1 / 2019
- Published online by Cambridge University Press:
- 31 December 2019, p. 70
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Introduction
Health Technology Assessment (HTA) and regulatory decisions involve value judgements. As patient groups, industry, and regulatory agencies conduct more preference studies to quantify these judgements, a better understanding of the methods and practices is needed. Currently, there is no systematic mapping of the use of preference data in Europe. This study aimed to identify (i) the use of quantitative preference data by all relevant HTA bodies and regulatory authorities of the European Union (EU) member states, and (ii) key standards and guidelines.
MethodsThis study used a mixed method approach based on a systematic literature review, survey and subsequent interviews with decision makers and experts.
ResultsA total of 62 survey responses were received. Many respondents reported that their agencies were responsible for supporting more than one type of decision, with 69.0 percent supporting approval decisions, 64.3 percent supporting reimbursement decisions, 61.9 percent supporting pricing decisions, and 64.2 percent supporting guideline development. Respondents reported that their agencies supported these decisions in multiple ways: 78.6 percent by assessing health technologies; 54.8 percent by appraising health technologies; 45.2 percent by compiling an HTA report; 7.1 percent by conducting primary research; 9.5 percent by conducting secondary research. More than 40 percent (42.9 percent) of agencies had the final say on one of the decisions of interest – approval, reimbursement, or pricing. Of the 31 countries studied, 71 percent (n = 22) used quantitative preference data in their reimbursement and pricing decisions. Of those, 86 percent (n = 19) used general population preferences to inform the estimation of quality-adjusted life years (QALY) as part of cost utility analysis.
ConclusionsMuch of this use of preference data can be understood within the standard framework of economic analysis adopted by many HTA agencies; either in in the form of: standard ways to estimate QALYs; ways to broaden the impacts of technologies captured in the QALY; or ways to weigh health gain with other decision-making criteria, such as disease severity or innovativeness.
OP143 Conceptualizing Patients’ Experience With Atrial Fibrillation
- Elisabeth Oehrlein, Eleanor Perfetto, Debbe McCall, Jennifer Albrecht, Julia Slejko, Catherine Cooke, Susan dosReis
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- International Journal of Technology Assessment in Health Care / Volume 34 / Issue S1 / 2018
- Published online by Cambridge University Press:
- 03 January 2019, p. 52
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Introduction:
Conceptual models (CMs) are useful tools for researchers and health technology assessment bodies to understand the interplay among environmental characteristics (e.g., health care system), patient characteristics, health behaviors, and patient outcomes. The objective of this pilot study was to elicit perspectives of patients with atrial fibrillation (AF) and health care providers (HCPs) to develop a patient-centered CM of the AF patient experience in a US-based sample.
Methods:We developed two preliminary versions of the Andersen model of healthcare utilization (standard and patient-friendly versions) based on the published literature and the help of a patient advisor. For example, instead of describing “predisposing characteristics,” the patient-friendly CM describes, “what is it about me, or other afib patients that could impact disease or outcomes;” “enabling resources” is swapped for “helpful resources,” and “perceived need” is changed to “what impacts whether I believe I need to be treated”. Five patients from an online patient community and 10 HCPs from the University of Maryland Medical System provided feedback on the preliminary models. Audio recordings of interviews were transcribed verbatim, analyzed, and findings incorporated into a revised CM.
Results:Interviewee additions under “what impacts whether I believe I need to be treated” included: absence of symptoms and fear of experiencing an AF episode; under “helpful resources” suggested additions include resources for navigating insurer formulary/benefits. Suggested additional outcomes of interest include anxiety, bruising, and shortness-of-breath. While patients found the patient-friendly version easy to understand, HCPs required explanation of standard-version headers, for example ‘predisposing characteristics’ and ‘enabling resources’, which had been adapted in the patient-friendly version.
Conclusions:Soliciting input from stakeholders ensures CMs are pragmatic, reflect the real-world experiences of patients and HCPs, and incorporate variables or other considerations not currently described in published literature. Researchers can utilize CMs to aid in selection of variables for observational studies.
OP95 Are Patient-Reported Outcome Measures Meeting Today's Standards?
- Elisabeth Oehrlein, Eleanor Perfetto, T. Rose Love, Yujin Chung, Parima Ghafoori
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- Journal:
- International Journal of Technology Assessment in Health Care / Volume 34 / Issue S1 / 2018
- Published online by Cambridge University Press:
- 03 January 2019, pp. 34-35
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Introduction:
Over the past decade, health technology assessment (HTA) agencies have become interested in improving the patient-centeredness of their assessments. A common approach has been to prioritize patient-reported outcomes (PROs), often describing PROs as patient-relevant or patient-oriented. However, it is often unclear whether and to what degree PRO measures (PROMs) truly reflect what is important to patients. This review examined the pedigree of a sample of measures used as primary or secondary endpoints in trials and discussed in Food and Drug Administration (FDA) approved product labels between 2003 and 2014.
Methods:We examined all 26 PROs included in chapters 1 (Office of Microbial Products) and 2 (Office of Drug Evaluation I) of the FDA's Pilot Clinical Outcome Assessment (COA) Compendium. Three reviewers independently searched PubMed and Google to identify publications or other relevant materials related to method and stage of measure development where patient engagement took place.
Results:Among 26 evaluated PROMs, we were unable to locate any information on development or validation for 12 (patient diary=9; rating scale=3). Among the remaining 14 PROMs, 5 did not include any evidence of patient engagement (questionnaire=1; patient diary=2; rating scale=2); 3 engaged patients during concept elicitation or psychometric validation only (disease-specific questionnaires=3); and 6 engaged patients during both concept elicitation and cognitive interviewing (disease-specific questionnaires=6). PROMs either previously qualified or submitted for qualification by FDA were more likely to include patient engagement.
Conclusions:PROs can provide patient-centered data useful for HTA; however, patient-reported information is not inherently patient-centered. This study found that only a minority of sampled PROMs engaged patients during both concept elicitation and cognitive interviewing. To facilitate patient-centered HTA, manufacturers should ensure that PROMs incorporated into clinical trials measure concepts important to patients. Similarly, HTAs should request data on development and validation of all outcome measures incorporated into trials.
PEER-REVIEWED JOURNAL EDITORS’ VIEWS ON REAL-WORLD EVIDENCE
- Elisabeth M. Oehrlein, Jennifer S. Graff, Eleanor M. Perfetto, C. Daniel Mullins, Robert W. Dubois, Chinenye Anyanwu, Eberechukwu Onukwugha
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- Journal:
- International Journal of Technology Assessment in Health Care / Volume 34 / Issue 1 / 2018
- Published online by Cambridge University Press:
- 08 February 2018, pp. 111-119
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Objectives: Peer-review publication is a critical step to the translation and dissemination of research results into clinical practice guidelines, health technology assessment (HTA) and payment policies, and clinical care. The objective of this study was to examine current views of journal editors regarding: (i) The value of real-world evidence (RWE) and how it compares with other types of studies; (ii) Education and/or resources journal editors provide to their peer reviewers or perceive as needed for authors, reviewers, and editors related to RWE.
Methods: Journal editors’ views on the value of RWE and editorial procedures for RWE manuscripts were obtained through telephone interviews, a survey, and in-person, roundtable discussion.
Results: In total, seventy-nine journals were approached, resulting in fifteen telephone interviews, seventeen survey responses and eight roundtable participants. RWE was considered valuable by all interviewed editors (n = 15). Characteristics of high-quality RWE manuscripts included: novelty/relevance, rigorous methodology, and alignment of data to research question. Editors experience challenges finding peer reviewers; however, these challenges persist across all study designs. Journals generally do not provide guidance, assistance, or training for reviewers, including for RWE studies. Health policy/health services research (HSR) editors were more likely than specialty or general medicine editors to participate in this study, potentially indicating that HSR researchers are more comfortable/interested in RWE.
Conclusions: Editors report favorable views of RWE studies provided studies examine important questions and are methodologically rigorous. Improving peer-review processes across all study designs, has the potential to improve the evidence base for decision making, including HTA.
OP38 Improving The Patient Centricity Of Value Assessments: A Rubric
- Eleanor Perfetto, Elisabeth Oehrlein, Marc Boutin, Sarah Reid, Eric Gascho
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- Journal:
- International Journal of Technology Assessment in Health Care / Volume 33 / Issue S1 / 2017
- Published online by Cambridge University Press:
- 12 January 2018, pp. 17-18
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INTRODUCTION:
Value frameworks, analogous to Health Technology Assessment (HTA) internationally, have emerged in the United States to aid stakeholders in assessing the value of new treatments. Since patient perspectives on value may differ significantly from other stakeholders, formalized procedures to involve patients in their work have been created. Despite these efforts, concerns persist that patient involvement is insufficient or “rhetoric.” To assist in this effort, the National Health Council (NHC) created a rubric to aid decision makers in improving the patient centricity of their value assessments.
METHODS:A convenience sample of twenty-eight organizations was invited to participate in a roundtable discussion. Participants discussed experiences with value frameworks; debated and thematically grouped hallmark patient-centeredness characteristics; and developed illustrative examples of the characteristics. These materials were organized into the rubric, and subsequently vetted via multi-stakeholder peer review.
RESULTS:Participants agreed upon six key domains of patient centeredness: partnership (patients are involved in every step of development/dissemination processes), transparency (assumptions/inputs are disclosed in an understandable, timely way), inclusiveness (perspectives drawn from broad range of stakeholders), diversity (differences in subpopulations, trajectory of disease, and stage of a life should be accounted for), outcomes (includes those that patients have identified as important), and data (variety of credible data sources are used allowing for timely incorporation of new information and account for the diversity of patient populations and patient-centered outcomes). The Rubric describes each domain and includes illustrative examples of how patient engagement/centeredness can be operationalized through direct and indirect pathways.
CONCLUSIONS:The NHC Rubric is a first step toward creating patient-centered value assessments that patients and their families can rely on. It is intended to assist all stakeholders, especially the patient community, in assessing the level of patient centeredness and engagement in a given framework or model. It can be a guide to support developers in conceptualizing plans for meaningfully engaging patients.
VP02 Are Journal Editors A Barrier To Publication Of Real World Evidence?
- Elisabeth Oehrlein, Jennifer Graff, Eleanor Perfetto, C. Daniel Mullins, Robert Dubois, Chinenye Anyanwu, Ebere Onukwugha
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- International Journal of Technology Assessment in Health Care / Volume 33 / Issue S1 / 2017
- Published online by Cambridge University Press:
- 12 January 2018, pp. 147-148
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INTRODUCTION:
Health technology assessment (HTA) bodies often rely heavily upon evidence from peer-review publications to inform their recommendations. Since journal editors attitudes likely influence the types of study designs that are accepted through the peer-review process and on to publication, journal editors serve as gatekeepers to translation of evidence, including real word evidence (RWE), into practice. Given past skepticism of RWE study designs, journal editors perceptions of and possible biases toward RWE are important to understand as they may impact dissemination and, therefore, uptake of research findings. The objective of this study was to examine current views of journal editors regarding the: (i) value of RWE studies and (ii) challenges editors face in managing, reviewing, and publishing RWE manuscripts.
METHODS:Journal editors representing general medicine (GM), specialty medicine (SM), health policy/services research (HSR) were invited to participate in a telephone interview, a survey, and an in-person, roundtable discussion.
RESULTS:In total, seventy-nine journals were approached, resulting in: 15 interviews (GM = 2; SM = 5; HSR = 8), 17 survey responses (GM = 2; SM = 6; HSR = 9) and 8 roundtable participants. RWE was viewed favorably by interviewed editors (n = 15). Characteristics of high-quality RWE manuscripts included: research question novelty/relevance, rigorous methodology, alignment of data with question, and the extent data-source advantages are optimized. Similar manuscript review processes and challenges were voiced for RWE and other study designs. HSR editors were more likely than SM or GM editors to participate, potentially indicating these researchers are more comfortable or interested in RWE. A possible study limitation was that editors favorable toward RWE may have been more likley to participate.
CONCLUSIONS:Peer-review journal editors appear to have favorable views regarding RWE studies and can be accelerators to dissemination of RWE findings. However, they do report that studies and processes could be improved. One suggested improvement included a checklist for editors to speed rejections and improve communications with authors.
VP165 Landscape Assessment: Patient Engagement In Health Technology Assessment
- Elisabeth Oehrlein, Jason Harris, Nicole Labkoff, Eleanor Perfetto, Manal Ziadeh, Sarah El-Gendi, Bahareh Ghorashi, Marc Boutin
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- Journal:
- International Journal of Technology Assessment in Health Care / Volume 33 / Issue S1 / 2017
- Published online by Cambridge University Press:
- 12 January 2018, pp. 224-225
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INTRODUCTION:
Understanding the current landscape of patient engagement across value decision-making bodies internationally is a critical first step toward improving the patient centricity of Health Technology Assessment (HTA). This study assessed: (i) Terms and definitions used; (ii) Patient engagement opportunities; (iii) Evidence of patient engagement.
METHODS:A sample of country-specific HTA's (HTA; n = 6), professional organizations (PO; n = 4), and collaborations/independent organizations (CO; n = 3) was selected for representativeness. Information was gathered through: (i) targeted web search and (ii) emailing organizations directly. Definitions, HTA methods documents, and the three most recent evaluations were identified, abstracted, and compared. Data were collected between September-October 2016.
RESULTS:Numerous terms are used to describe patient engagement: patient input (HTA = 1, PO = 1), patient-group submitted information (HTA = 1), cooperation with patients/users (HTA = 1), public consultation (HTA = 1), patient perspectives (HTA = 1, PO = 1), involvement of people affected (HTA = 1), patient involvement (HTA = 2), patient and public involvement (HTA = 1), lay involvement (HTA = 1), inclusion of patient representative (PO = 3), patient reports (PO = 1), patient preference (PO = 2), public consultation (CO = 1), stakeholder consultation (CO = 1), open input (CO = 1), stakeholder engagement (CO = 1), and patient participation (CO = 1). Opportunities for patient engagement were described as: patient questionnaire (HTA = 2); comment period (HTA = 1; CO = 1); committee participation (HTA = 3; PO = 3); propose topics (HTA = 1); draft guidance (HTA = 1); general stakeholder forum (CO = 1). While organizations outline opportunities for patient engagement, not all organizations have clear evidence the practices are used or have impact. Recent evaluations demonstrate clear evidence of engagement (HTA = 2); Unclear or mixed evidence (HTA = 1; PO = 1; CO = 2); No evidence (HTA = 3; PO = 3; CO = 1).
CONCLUSIONS:There is substantial heterogeneity in the terms used to describe patient engagement activities across organizations. While a variety of opportunities for patient engagement are described, lack of clear evidence to how patient engagement practices are consistently used may contribute to the perception that engagement by HTAs.