Psychometric validation of the Cystic Fibrosis Impact Questionnaire (CF-IQ): A patient-reported outcome assessing impacts of cystic fibrosis.

The Cystic Fibrosis (CF) Impact Questionnaire (CF-IQ) was qualitatively developed to assess the impact of CF in the context of treatment advancements and increased longevity. This study reports the CF-IQ validation. In this noninterventional validation study, people with CF completed the 40-item CF-IQ and validating patient-reported outcome measures (PROMs) via electronic diaries at enrollment (baseline) and at the 4-week follow-up.

A Retrospective, Longitudinal Registry Study on the Long-Term Durability of Ivacaftor Treatment in People with Cystic Fibrosis.

Introduction: Ivacaftor (IVA) has been shown to change the trajectory of cystic fibrosis (CF) disease progression by slowing the rate of lung function decline in clinical studies. Long-term real-world data help to confirm the durability of this response.

Methods: This non-interventional, longitudinal study used data from the US CF Foundation Patient Registry to describe the annualized rate of change in lung function in people with CF receiving IVA.

Real-world impact of ivacaftor in people with cystic fibrosis and select ivacaftor-responsive mutations.

Background: Ivacaftor approval was extended to people with cystic fibrosis (CF) with ≥1 of 28 additional ivacaftor-responsive mutations in the USA in 2017 based on preclinical in vitro data. This retrospective, observational study assessed real-world clinical response to ivacaftor in people with CF with ≥1 of these mutations, using data from the US Cystic Fibrosis Foundation Patient Registry.

Methods: Participants aged ≥2 years with ≥1 of 28 eligible mutations initiating ivacaftor between May 2017 and December 2018 were included.

Long-term impact of ivacaftor on mortality rate and health outcomes in people with cystic fibrosis.

Background: Ivacaftor (IVA) has been shown to improve lung function and other clinical outcomes in people with cystic fibrosis (CF). A decade of real-world IVA availability has enabled the examination of long-term outcomes with this treatment. This retrospective, longitudinal cohort study investigated the impact of IVA on mortality rate and health outcomes among people with CF in the US.

Impact of age at ivacaftor initiation on pulmonary outcomes among people with cystic fibrosis.

Background: Ivacaftor (IVA) improves lung function and other extrapulmonary outcomes in people with cystic fibrosis (CF). However, the effect of initiating IVA at earlier versus later ages has not been studied.

Methods: We conducted an observational cohort study of people in the US CF Foundation Patient Registry aged ≥6 years with ≥1 CF transmembrane conductance regulator-gating mutation to compare the effects of initiating IVA at earlier ages on per cent predicted forced expiratory volume in 1 s (ppFEV) and pulmonary exacerbation (PEx) outcomes.

VOCAL: An observational study of ivacaftor for people with cystic fibrosis and selected non-G551D-CFTR gating mutations.

Background: VOCAL was an observational study of the effect of long-term ivacaftor on real-world clinical outcomes and healthcare resource utilization (HCRU) in people with cystic fibrosis (pwCF) in Italy, the Netherlands, and the UK.

Methods: pwCF aged ≥6 years with non-G551D-CFTR gating mutations were eligible. Prospective data were collected up to 48 months after enrollment; retrospective data were collected to ensure that 12 months of pre-ivacaftor data were available.

Healthcare resource utilization and costs among children with cystic fibrosis in the United States.

Background: Adverse health impacts of cystic fibrosis (CF) can be present in children before respiratory complications are observed. Children with CF show progressive health decline, with increasing lung function decline in adolescence. This study aims to quantify the healthcare resource utilization (HCRU) and costs attributable to CF by comparing children with CF with the general pediatric population.

Real-World Outcomes of Ivacaftor Treatment in People with Cystic Fibrosis: A Systematic Review.

Cystic fibrosis (CF) is a rare, progressive, multi-organ genetic disease. Ivacaftor, a small-molecule CF transmembrane conductance regulator modulator, was the first medication to treat the underlying cause of CF. Since its approval, real-world clinical experience on the use of ivacaftor has been documented in large registries and smaller studies.

Long-Term Impact of Ivacaftor on Healthcare Resource Utilization Among People with Cystic Fibrosis in the United States.

Introduction: Ivacaftor was first approved in 2012 for the treatment of a select population of individuals with cystic fibrosis (CF), a rare, life-shortening genetic disease. Reductions in healthcare resource utilization (HCRU) associated with ivacaftor have been observed during limited follow-up and for selected outcomes in real-world studies. This study aimed to further describe the long-term impact of ivacaftor treatment on multiple measures of HCRU among people with CF (pwCF).

Caregiver Burden Due to Pulmonary Exacerbations in Patients with Cystic Fibrosis.

Objective: To describe the poorly understood burden of pulmonary exacerbations experienced by primary caregivers of children (aged 2-17 years) with cystic fibrosis (CF), who frequently require prolonged hospitalizations for treatment of pulmonary exacerbations with intravenous (IV) antibiotics.

Study Design: In this prospective observational study, 88 caregivers in Germany, Ireland, the United Kingdom, and the US completed a survey during pulmonary exacerbation-related hospitalizations (T1) and after return to a "well state" of health (T2). The impact of pulmonary exacerbations on caregiver-reported productivity, mental/physical health, and social/family/emotional functioning was quantified.

Hemophilia Burden of Disease: A Systematic Review of the Cost-Utility Literature for Hemophilia.

Background: Prophylaxis with clotting factor replacement products is recommended by the Medical and Scientific Advisory Council of the National Hemophilia Foundation as the optimal therapy for the prevention of bleeding episodes in individuals with severe hemophilia A or B (< 1 IU per dL endogenous factor VIII or factor IX activity, respectively). Prophylaxis is associated with an improved health-related quality of life and has been shown to be cost-effective compared with on-demand therapy. However, the overall cost of treatment remains high, particularly among patients with a greater propensity to bleed.

Patient Variability Seldom Assessed in Cost-effectiveness Studies.

Background: Cost-effectiveness analysis (CEA) estimates can vary substantially across patient subgroups when patient characteristics influence preferences, outcome risks, treatment effectiveness, life expectancy, or associated costs. However, no systematic review has reported the frequency of subgroup analysis in CEA, what type of heterogeneity they address, and how often heterogeneity influences whether cost-effectiveness ratios exceed or fall below conventional thresholds.

Methods: We reviewed the CEA literature cataloged in the Tufts Medical Center CEA Registry, a repository describing cost-utility analyses published through 2016.

When cost-effective interventions are unaffordable: Integrating cost-effectiveness and budget impact in priority setting for global health programs.

Potential cost-effective barriers in cost-effectiveness studies mean that budgetary impact analyses should also be included in post-2015 Sustainable Development Goal projects says Joshua Salomon and colleagues.

Drugs Cleared Through The FDA's Expedited Review Offer Greater Gains Than Drugs Approved By Conventional Process.

We investigated whether drugs approved by the Food and Drug Administration (FDA) through expedited review have offered larger health gains, compared to drugs approved through conventional review processes. We identified published estimates of additional health gains (measured in quality-adjusted life-years, or QALYs) associated with drugs approved in the period 1999-2012 through expedited (seventy-six drugs) versus conventional (fifty-nine) review processes. We found that drugs in at least one expedited review program offered greater gains than drugs reviewed through conventional processes (0.

A Systematic Review of Cost-Effectiveness Studies Reporting Cost-per-DALY Averted.

Introduction: Calculating the cost per disability-adjusted life years (DALYs) averted associated with interventions is an increasing popular means of assessing the cost-effectiveness of strategies to improve population health. However, there has been no systematic attempt to characterize the literature and its evolution.

Methods: We conducted a systematic review of cost-effectiveness studies reporting cost-per-DALY averted from 2000 through 2015.

Estimating Population Health Benefits Associated with Specialty and Traditional Drugs in the Year Following Product Approval.

Objective: Compared to traditional drugs, specialty drugs tend to be indicated for lower prevalence diseases. Our objective was to compare the potential population health benefits associated with specialty and traditional drugs in the year following product approval.

Methods: First, we created a dataset of estimates of incremental quality-adjusted life-year (QALY) gains and incremental life-year (LY) gains for US FDA-approved drugs (1999-2011) compared to standard of care at the time of approval identified from a literature search.

Private payers disagree with Medicare over medical device coverage about half the time.

There are multiple payers in the US health care system, each making its own coverage determinations for medical technologies. For each of the forty-seven medical devices considered in national coverage determinations (NCDs) of the Centers for Medicare and Medicaid Services (CMS) issued between February 1999 and August 2013, we compared CMS's coverage policy with the policies issued by the largest sixteen private payers that made their decisions publicly available. Overall, we found that NCDs were equivalent to the corresponding private payer policies roughly half of the time, more restrictive approximately a quarter of the time, and less restrictive about a quarter of the time.

The State of Cost-Utility Analyses in Asia: A Systematic Review.

Objective: To review and evaluate published cost-utility analyses (CUAs) targeting populations in Asia.

Methods: We examined data from the Tufts Medical Center Cost-Effectiveness Analysis Registry, which contains detailed information on more than 3700 English-language CUAs in peer-reviewed journals through 2012. We focused on CUAs pertaining to Asian countries (Asian CUAs), summarized study features and methodological practices, and compared them with CUAs focusing on non-Asian countries (non-Asian CUAs) from 2000 to 2012.

The changing face of the cost-utility literature, 1990-2012.

Objectives: Cost-utility analyses (CUAs) have been published widely over the years to measure the value of health care interventions. We investigated the growth and characteristics of CUAs in the peer-reviewed English-language literature through 2012.

Methods: We analyzed data from the Tufts Medical Center Cost-Effectiveness Analysis (CEA) Registry, a database containing more than 3700 English-language CUAs published through 2012.

The lag from FDA approval to published cost-utility evidence.

The lag between FDA approval and publication of cost-utility evidence can hamper payers from accounting for value for money in coverage and reimbursement decisions. We examine this gap, and whether it has changed over time. For drugs approved from 2000 to 2010 (n = 274), we searched the Tufts Medical Center Cost-Effectiveness Analysis Registry to identify relevant cost-utility analyses (CUAs).

Despite high costs, specialty drugs may offer value for money comparable to that of traditional drugs.

Specialty drugs are often many times more expensive than traditional drugs, which raises questions of affordability and value. We compared the value of specialty and traditional drugs approved by the Food and Drug Administration (FDA) in the period 1999-2011. To do this, we identified published estimates of additional health gains (measured in quality-adjusted life-years, or QALYs) and increased costs of drug and health care resource use that were associated with fifty-eight specialty drugs and forty-four traditional drugs, compared to preexisting care.