Priorities to Promote Participant Engagement in the Participant Engagement and Cancer Genome Sequencing (PE-CGS) Network.

Background: Engaging diverse populations in cancer genomics research is of critical importance and is a fundamental goal of the NCI Participant Engagement and Cancer Genome Sequencing (PE-CGS) Network. Established as part of the Cancer Moonshot, PE-CGS is a consortium of stakeholders including clinicians, scientists, genetic counselors, and representatives of potential study participants and their communities. Participant engagement is an ongoing, bidirectional, and mutually beneficial interaction between study participants and researchers.

The evolution of patient-focused drug development and Duchenne muscular dystrophy.

: There is a groundswell of interest from patient, industry, and regulatory groups to rigorously and transparently integrate patient-voice into regulatory decision-making. Patient-focused drug development (PFDD) is an approach established by the US Food and Drug Administration to systematically incorporate patient experiences into drug development and evaluation. It has created a demand for scientific advancement to measure and integrate patient-voice into decision-making.

Neoadjuvant therapy for resectable pancreatic ductal adenocarcinoma: The need for patient-centered research.

Pancreatic ductal adenocarcinoma is an aggressive cancer with high recurrence rates following surgical resection. While adjuvant chemotherapy improves survival, a significant proportion of patients are unable to initiate or complete all intended therapy following pancreatectomy due to postoperative complications or poor performance status. The administration of chemotherapy prior to surgical resection is an alternative strategy that ensures its early and near universal delivery as well as improves margin-negative resection rates and potentially improves long-term survival outcomes.

Patient preferences for attributes of tyrosine kinase inhibitor treatments for EGFR mutation-positive non-small-cell lung cancer.

EGFR-tyrosine kinase inhibitors (TKIs) vary in efficacy, side effects (SEs) and dosing regimen. We explored EGFR-TKI treatment attribute preferences in EGFR mutation-positive metastatic non-small-cell lung cancer. Patients completed a survey utilizing preference elicitation methods: direct elicitation of four EGFR-TKI profiles describing progression-free survival (PFS), severe SE risk, administration; discrete choice experiment involving 12 choice tasks.

Collecting patient preference information using a Clinical Data Research Network: demonstrating feasibility with idiopathic pulmonary fibrosis.

Rare diseases present challenges for accessing patient populations to conduct surveys. Clinical Data Research Networks (CDRNs) offer an opportunity to overcome those challenges by providing infrastructure for accessing patients and sharing data. This study aims to demonstrate the feasibility of collecting patient preference information for a rare disease in a CDRN, using idiopathic pulmonary fibrosis as proof of concept.

Japanese patient preferences regarding intermediate to advanced hepatocellular carcinoma treatments.

This study aimed to evaluate Japanese patient preferences regarding features of intermediate or advanced (Progressed) hepatocellular carcinoma (HCC) treatments: transarterial chemoembolization (TACE), hepatic arterial infusion chemotherapy (HAIC), and oral anti-cancer therapy. Patients with HCC, recruited from clinical sites and a patient panel in Japan, completed a cross-sectional web-based survey. Preferences were quantified using best-worst scaling, where patients identified the best and worst among 13 treatment features.

Research as an event: a novel approach to promote patient-focused drug development.

Patient groups are increasingly engaging in research to understand patients' preferences and incorporate their perspectives into drug development and regulation. Several models of patient engagement have emerged, but there is little guidance on how to partner with patient groups to engage the disease community. Our group has been using an approach to engage patient groups that we call research as an event.

Developing and piloting an instrument to prioritize the worries of patients with acute myeloid leukemia.

Background: Acute myeloid leukemia (AML) is a rapidly progressing blood cancer for which new treatments are needed. We sought to promote patient-focused drug development (PFDD) for AML by developing and piloting an instrument to prioritize the worries of patients with AML.

Patients And Methods: An innovative community-centered approach was used to engage expert and community stakeholders in the development, pretesting, pilot testing, and dissemination of a novel best-worst scaling instrument.

Identifying and prioritizing concerns associated with prosthetic devices for use in a benefit-risk assessment: a mixed-methods approach.

Objective: We identified and prioritized concerns reported by stakeholders associated with novel upper-limb prostheses.

Methods: An evidence review and key-informant engagement, identified 62 concerns with upper-limb prostheses with implantable components. We selected 16 concerns for inclusion in a best-worst scaling (BWS) prioritization survey.

A patient and community-centered approach selecting endpoints for a randomized trial of a novel advance care planning tool.

Background: Despite a movement toward patient-centered outcomes, best practices on how to gather and refine patients' perspectives on research endpoints are limited. Advanced care planning (ACP) is inherently patient centered and would benefit from patient prioritization of endpoints for ACP-related tools and studies.

Objective: This investigation sought to prioritize patient-centered endpoints for the content and evaluation of an ACP video being developed for patients undergoing major surgery.

Quantifying the treatment goals of people recently diagnosed with schizophrenia using best-worst scaling.

Objective: This study seeks to quantify the treatment goals of people recently diagnosed with schizophrenia and explore their impact on treatment plan.

Methods: People aged 18-35 years with a confirmed diagnosis of schizophrenia within the past 5 years were surveyed in the UK, Germany, and Italy. Treatment goals were assessed via a validated best-worst scaling instrument, where participants evaluated subsets of 13 possible treatment goals identified using a balanced incomplete block design.

Live, Learn, Pass It on: A Patient Advocacy Engagement Project on the Lived Experience of Lung Cancer Survivors.

Introduction: The objective of this project was to engage survivor-advocates by describing their experiences living with lung cancer in an era of increasing treatment options.

Methods: This was a qualitative engagement project with participants from a lung cancer advocacy organization's survivor advisory board. Interviews were conducted, transcribed, and analyzed for stages and associated experiences using interpretive phenomenological analysis and elements of narrative analysis, in partnership with the patient advocacy organization.

Education and patient preferences for treating type 2 diabetes: a stratified discrete-choice experiment.

Purpose: Diabetes is a chronic condition that is more prevalent among people with lower educational attainment. This study assessed the treatment preferences of patients with type 2 diabetes by educational attainment.

Methods: Patients with type 2 diabetes were recruited from a national online panel in the US.

Applying stated-preference methods to improve health systems in sub-Saharan Africa: a systematic review.

Sub-Saharan African health systems must balance shifting disease burdens with desires for robust institutions. Stated-preference methods have been applied extensively to elicit health care workers' preferences and priorities for rural practice. This systematic review characterizes the range of their applications to African health systems problems.

Using clinical data to predict high-cost performance coding issues associated with pressure ulcers: a multilevel cohort model.

Objective: Hospital-acquired pressure ulcers (HAPUs) have a mortality rate of 11.6%, are costly to treat, and result in Medicare reimbursement penalties. Medicare codes HAPUs according to Agency for Healthcare Research and Quality Patient-Safety Indicator 3 (PSI-03), but they are sometimes inappropriately coded.

Factors that influence patient preferences for prostate cancer management options: A systematic review.

Purpose: We performed a systematic review to evaluate evidence regarding factors that influence patient preferences for management options for localized prostate cancer.

Methods: We followed a prespecified search protocol (PROSPERO identifier CRD42014009173) to identify studies that evaluated patient preferences for prostate cancer management options for localized prostate cancer. We queried PubMed, the Cochrane Database of Systematic Reviews, Embase, Cumulative Index to Nursing & Allied Health Literature (CINAHL) Plus, and Econ-Lit databases.

Creating an advance-care-planning decision aid for high-risk surgery: a qualitative study.

Background: High-risk surgery patients may lose decision-making capacity as a result of surgical complications. Advance care planning prior to surgery may be beneficial, but remains controversial and is hindered by a lack of appropriate decision aids. This study sought to examine stakeholders' views on the appropriateness of using decision aids, in general, to support advance care planning among high-risk surgery populations and the design of such a decision aid.

The changing role of economic evaluation in valuing medical technologies.

Economic evaluation is established within health-technology assessment but is challenged by those wanting to use economic evaluation to inform pricing and/or incorporate nontraditional sources of value and the views of diverse stakeholders. The changing role of economic evaluation in (formally or informally) assessing prices/values in four jurisdictions (UK, Australia, Germany and USA) is detailed and the authors propose a taxonomy of factors impacting the value of medical technology spanning clinical utility (effectiveness, safety/tolerability and quality of evidence), consumer demand (consumer preferences, process utility and unmet need), economic incentives (innovation, option value and market competition) and the societal perspective (social justice, social values and national interest). The authors suggest that multicriteria decision analysis methods grounded in hedonic-pricing theory can facilitate the valuing/pricing of medical technologies.

Controlling liver cancer internationally: A qualitative study of clinicians' perceptions of current public policy needs.

Background: Liver cancer is the fifth most common cancer in men and the seventh for women. Usually because of late diagnosis, the prognosis for liver cancer remains poor, resulting in liver cancer being the third most common cause of death from cancer. While some countries have treatment guidelines, little is known or understood about the strategies needed for liver cancer control internationally.

Can pharmacoeconomics and outcomes research contribute to the empowerment of women affected by breast cancer?

Breakthroughs in genetic testing have informed patients and physicians in the treatment of breast cancer; however, they have also added to the complexity of decision-making. Genetic testing for breast cancer susceptibility not only changes treatment and screening options, but also challenges the way in which interventions are evaluated. While comparative effectiveness and cost-effective analysis methods are now standard for evaluation at the societal level, technologies such as genetic testing require us to consider the role of patient preference, especially as we move towards more personalized approaches to medicine.

Promoting patient empowerment in the healthcare system: highlighting the need for patient-centered drug policy.

In recent years the term 'patient empowerment' has increasingly been used among healthcare policy makers; however, discussions have focused on narrow activities that as stand-alone activities would be unlikely to give patients power. This paper presents findings from a systematic review of the medical literature. After offering a new definition of patient empowerment, we review the literature behind the concept, aided by a conceptual model.

Globalization and healthcare: understanding health and medical tourism.

Faced with long waiting lists, the high cost of elective treatment and fewer barriers to travel, the idea of availing healthcare in another country is gaining greater appeal to many. The objective of this review is to perform a literature review of health and medical tourism, to define health and medical tourism based on the medical literature and to estimate the size of trade in healthcare. The Medline database was used for our literature review.

What can economics add to health technology assessment? Please not just another cost-effectiveness analysis!

Evidence based medicine is not only important for clinical practice, but national governments have embraced it through health technology assessment (HTA). HTA combines data from randomized controlled trials (RCT) and observational studies with an economic component (among other issues). HTA, however, is not taking full advantage of economics.