Palliative care needs and preferences of older adults with advanced or serious chronic illnesses and their families in rural communities of Indiana, USA.

PURPOSE: To explore the palliative care needs and preferences of older adults with advanced or serious chronic illnesses and their families. Also, to propose strategies to promote supportive palliative care in the rural communities of Indiana, USA. METHOD: We conducted qualitative interviews to gather rural caregivers' experiences of palliative care. Recruitment was done in collaboration with community partners using social media, flyers, emails, invitations, and word-of-mouth. A purposive sample of family caregivers was obtained. All the interviews were conducted online. The average interview was 30-45 minutes. Data were analyzed using a thematic analysis approach. FINDINGS: Our findings showed 6 major themes that indicated several palliative care needs and preferences of older patients and their families in rural communities that include: (1) difficulties in pain and symptom burden; (2) perceived discrimination and lack of trust; (3) longer distances to care facilities; (4) difficult conversations; (5) caregiving burden; and (6) use of telehealth in a rural palliative care context. CONCLUSION: Rural family caregivers experience several social inequities and disparities causing a lack of access to and low utilization of palliative care. All these disparities cause several challenges for patients and their families trying to manage serious illnesses and die in place with peace and comfort. Inadequate access and lack of resources cause pain and distress for both patients and their families. Provider education and trainings, initiating early palliative care models, integrating behavioral health in palliative care, and using culturally congruent care delivery approaches in support of community partners can improve palliative care services in rural communities.

Predictors for Initiation of Atypical Long-Acting Injectable Antipsychotic Agents in a Commercial Claims Cohort of Individuals With Early-Phase Schizophrenia.

Objective: Long-acting injectable antipsychotic agents (LAIs) have improved clinical effectiveness and adherence versus oral antipsychotic agents (OAs); however, a minority of individuals with schizophrenia are treated with LAIs compared with OAs. This cohort study aimed to evaluate predictors of initiation of atypical LAIs among patients with newly diagnosed schizophrenia in the United States.Methods: Using claims data from IBM MarketScan Commercial and Medicare Supplemental databases between January 1, 2013, and March 31, 2020, adults with first diagnosis of schizophrenia, ≥ 1 OA claim following diagnosis, and continuous benefits were identified. To evaluate predictors of LAI initiation, a Cox proportional hazard regression model per independent predictors and main outcome (ie, LAI initiation) was performed.Results: Of 3,639 patients with early-phase schizophrenia, 369 (10%) had ≥ 1 LAI claim(s) after ≥ 1 OA claim(s). Several factors present prior to LAI initiation were significantly (P < .0001) predictive of LAI initiation: greater monthly OA switches (hazard ratio [95% CI]: 11.39 [7.01-18.51]), unsuccessful OA implementation (3.09 [2.39-3.98]), greater monthly schizophrenia-related hospitalizations (20.83 [14.22-30.51]), and greater monthly schizophrenia-related emergency department visits (4.13 [2.07-8.22]).Conclusions: In this analysis of pharmacy claims records for patients with early-phase schizophrenia, results suggest that LAIs are used less frequently in the early phase than reported in later stages. Their initiation is often reactive to relapse or disease exacerbation, rather than proactive as a relapse-prevention tool for early-phase schizophrenia. These data highlight the underuse of LAIs, particularly in the early phase when they could make the most difference.

Early versus late administration of long-acting injectable antipsychotic agents among patients with newly diagnosed schizophrenia: an analysis of a commercial claims database.

This study was designed to assess healthcare resource utilization (HCRU) and costs in patients with newly diagnosed schizophrenia based on timing and context of long-acting injectable antipsychotic agent (LAI) initiation. Using claims data, patients (aged 18-40 years) with first schizophrenia diagnosis January 2013-September 2019 (index date), no LAI or oral antipsychotic agent claims during 12-month preindex period, and continuous benefit enrollment from 12 months before index date to 12 months after first LAI administration were identified. Patients were grouped based on timing [early (≤1 year after index date) vs. late] and circumstances [reactive (after schizophrenia-related event) vs. proactive] of LAI initiation. Of 1290 patients with at least one LAI claim, 306 met criteria for early ( n = 204; reactive, n = 107; proactive, n = 97) and late ( n = 102; n = 75; n = 27) initiation. HCRU and costs were numerically lower in early versus late groups, and significantly lower for proactive initiation in both groups. Comparing worst-case (late-reactive) and best-case (early-proactive) scenarios, the average annual cost difference was $7195.13 ( P = 0.0233), with major drivers being emergency department ($171.28; P < 0.05) and other outpatient ($2845.73; P < 0.00001) visits. In addition to the clinical advantages previously described in the literature, the proactive use of LAIs in early-phase schizophrenia is associated with lower healthcare costs.

Incorporating productivity loss in health economic evaluations: a review of guidelines and practices worldwide for research agenda in China.

INTRODUCTION: Productivity loss may contribute to a large proportion of costs of health conditions in an economic evaluation from a societal perspective, but there is currently a lack of methodological consensus on how productivity loss should be measured and valued. Despite the research progress surrounding this issue in other countries, it has been rarely discussed in China. METHODS: We reviewed the official guidelines on economic evaluations in different countries and regions and screened the literature to summarise the extent to which productivity loss was incorporated in economic evaluations and the underlying methodological challenges. RESULTS: A total of 48 guidelines from 46 countries/regions were included. Although 32 (67%) guidelines recommend excluding productivity loss in the base case analysis, 23 (48%) guidelines recommend including productivity loss in the base case or additional analyses. Through a review of systematic reviews and the economic evaluation studies included in these reviews, we found that the average probability of incorporating productivity loss in an economic evaluation was 10.2%. Among the economic evaluations (n=478) that explicitly considered productivity loss, most (n=455) considered losses from paid work, while only a few studies (n=23) considered unpaid work losses. Recognising the existing methodological challenges and the specific context of China, we proposed a practical research agenda and a disease list for progress on this topic, including the development of the disease list comprehensively consisting of health conditions where the productivity loss should be incorporated into economic evaluations. CONCLUSION: An increasing number of guidelines recommend the inclusion of productivity loss in the base case or additional analyses of economic evaluation. We optimistically expect that more Chinese researchers notice the importance of incorporating productivity loss in economic evaluations and anticipate guidelines that may be suitable for Chinese practitioners and decision-makers that facilitate the advancement of research on productivity loss measurement and valuation.

A systematic literature review of revealed preferences of decision-makers for recommendations of cancer drugs in health technology assessment.

OBJECTIVES: This review intends to provide an overview of revealed preferences of decision-makers for recommendations of cancer drugs in health technology assessment (HTA) among the different agencies. METHODS: A systematic literature search was performed in MEDLINE and EMBASE databases from inception to July 2020. The studies were eligible for inclusion if they conducted a quantitative analysis of HTA's previous decisions for cancer drugs. The factors with p-values below the significance level of .05 were considered as the statistically significant factors for HTA decisions. RESULTS: A total of nine studies for six agencies in Australia, Belgium, France, South Korea, the UK, and Canada were eligible to be included. From the univariable analysis, improvements in clinical outcomes and cost-effectiveness were found as significant factors for the agencies in Belgium, South Korea, and Canada. From the multivariable analysis, cost-effectiveness was found as a positive factor for the agencies in the UK, South Korea, and Canada. Few factors related to characteristics of disease and technology were found to be significant among the included agencies. CONCLUSIONS: Despite the different drug reimbursement systems and the socioeconomic situations, cost-effectiveness and/or improvement on clinical outcomes seemed to be the most important factors for recommendations of cancer drugs among the agencies.

Challenges in the market access of regenerative medicines, and implications for manufacturers and decision-makers: a systematic review.

Aim: Regenerative medicines (RMs) are expected to transform the treatment paradigm of rare, life-threatening diseases, while substantial challenges impede its market access. This study aimed to present these challenges. Materials & methods: Publications identified in the Medline and Embase databases until December 2020 were included. Results: Uncertainties around the relative effectiveness and long-term benefits of RMs are most scrutinized. A new reference case for RMs is questionable, but examining impacts of study perspective, time horizon, discount rate and extrapolation methods on estimates is advised. Establishing reasonable prices of RMs requires increased transparency in the development costs and better values measurements. Outcome-based payments require considerable investments and potential legislative adjustments. Conclusion: Greater flexibility for health technology assessment and economic analyses of RMs is necessary. This comprehensive review may prompt more multi-stakeholder conversations to discuss the optimized strategy for value assessment, pricing and payment in order to accelerate the market access of RMs.

Plain language summary Regenerative medicines (RMs) potentially offered new hopes for severe diseases without effective treatments. However, substantial challenges must be overcome to make them available for patients. This systematic review aims to present these challenges. Publications identified in the Medline and Embase databases until December 2020 were included. The limited clinical evidence causes the biggest uncertainties around the relative effectiveness and long-term benefits of RMs. The current methodology for economic analysis of RMs is questionable because broader, societal values related to RMs are not sufficiently captured. The high price of RMs seems unjustified and should be lowered by balancing the development costs and values delivered. Outcome-based payments could be employed to address the long-term financial challenges, but they will require investments to implement it. More flexibility for health technology assessment and economic analysis of RMs is necessary to safeguard the accelerated patient access.

Amortization of gene replacement therapies: A health policy analysis exploring a mechanism for mitigating budget impact of high-cost treatments.

With gene replacement therapies (GRTs) increasingly and rapidly reaching the healthcare marketplace, the vast potential for improving patient health is matched by the potential budgetary impact for healthcare payers. GRTs are highly valuable given their potential life-extending or even curative benefits and may provide significant cost-offsets compared with standard of care. Current healthcare systems are, however, struggling to fund such valuable but costly therapies. Some payers have already implemented specific financing models to account for the new treatment paradigms, but these do not address the budget impact in the year of acquisition or administration of these costly technologies. This health policy analysis aimed to assess the rationale and feasibility of amortization, within the context of financing healthcare technologies, and specifically GRTs. Amortization is an accounting concept applied to intangible assets that allows for spreading the cost an intangible asset over time, allowing for repayment to occur via interest and principal payments sufficient to repay the intangible asset in full by its maturity. Our systematic scoping review on the amortization of healthcare technologies found a very small literature base with even that being unclear and inconsistent in its understanding of the issues. Where amortization was proposed as a solution for funding costly, but highly valuable GRTs, the concept was not fully investigated in detail, nor was the feasibility of the approach fully challenged. However, by providing clear definitions of relevant concepts along with an example of amortization models applied to some example GRTs, we propose that amortization can offer a promising method for funding of extraordinarily high-value healthcare technologies, thereby increasing market and patient access for these technologies. Nonetheless, healthcare accounting principles and financing guidelines must evolve to apply amortization to the rapidly developing GRTs.

Reinforcing Collaboration and Harmonization to Unlock the Potentials of Advanced Therapy Medical Products: Future Efforts Are Awaited From Manufacturers and Decision-Makers.

Some advanced therapy medicinal products (ATMPs) hold great promises for life-threatening diseases with high unmet needs. However, ATMPs are also associated with significant challenges in market access, which necessitates the joint efforts between all relevant stakeholders to navigate. In this review, we will elaborate on the importance of collaborations and harmonization across different stakeholders, to expedite the market access of promising ATMPs. Manufacturers of ATMPs should proactively establish collaborations with other stakeholders throughout the whole lifecycle of ATMPs, from early research to post-market activities. This covered engagements with (1) external developers (i.e., not-for-profit organizations and commercial players) to obtain complementary knowledge, technology, or infrastructures, (2) patient groups and healthcare providers to highlight their roles as active contributors, and (3) decision-makers, such as regulators, health technology assessment (HTA) agencies, and payers, to communicate the uncertainties in evidence package, where parallel consultation will be a powerful strategy. Harmonization between decision-makers is desired at (1) regulatory level, in terms of strengthening the international standardization of regulatory framework to minimize discrepancies in evidence requirements for market authorization, and (2) HTA level, in terms of enhancing alignments between regional and national HTA agencies to narrow inequity in patient access, and cross-border HTA cooperation to improve the quality and efficiency of HTA process. In conclusion, manufacturers and decision-makers shared the common goals to safeguard timely patient access to ATMPs. Collaboration and harmonization will be increasingly leveraged to enable the value delivery of ATMPs to all stakeholders.

Incorporating future unrelated medical costs in cost-effectiveness analysis in China.

The occurrence of future unrelated medical costs is a direct consequence of life-prolonging interventions, but most pharmacoeconomic guidelines recommend the exclusion of these costs. The Chinese guidelines were updated in 2020, taking an exclusion approach for the future unrelated medical cost. We notice the research surrounding this issue continues in other countries and leads to an inclusion recommendation in some guidelines. Meanwhile, this issue has not been discussed in China, reflecting an urgent need for extensive research on its impact. We reviewed the theoretical and practical studies surrounding the inclusion of future unrelated medical costs, summarised the landscape of guidelines in other jurisdictions. We found that the inclusion would increase the internal and external consistency of economic evaluation and the comparability of results between different jurisdictions. However, more research is needed surrounding this issue. We proposed a future research agenda to inform the update of Chinese guidelines. We recommend research on individual-level healthcare reimbursement data and end-of-life costs from hospital administrative data to generate the age-specific, sex-specific and condition-specific costs. We also recommend establishing a formal process to evaluate the ethical and economic impact of including future unrelated medical costs and adjust the threshold accordingly in the guidelines.

The impact of COVID-19 on the cell and gene therapies industry: Disruptions, opportunities, and future prospects.

Coronavirus 2019 (COVID-19) has caused significant disruption to the cell and gene therapy (CGT) industry, which has historically faced substantial complexities in supply of materials, and manufacturing and logistics processes. As decision-makers shifted their priorities to COVID-19-related issues, the challenges in market authorisation, and price and reimbursement of CGTs were amplified. Nevertheless, it is encouraging to see that some CGT developers are adapting their efforts toward the development of promising COVID-19-related therapeutics and vaccines. Manufacturing resilience, digitalisation, telemedicine, value-based pricing, and innovative payment mechanisms will be increasingly harnessed to ensure that market access of CGTs is not severely disrupted.

Which Criteria are Considered and How are They Evaluated in Health Technology Assessments? A Review of Methodological Guidelines Used in Western and Asian Countries.

OBJECTIVES: This study aimed to provide an exhaustive description of criteria and methodological recommendations for evaluating them in health technology assessment (HTA) in Western and Asian countries. METHODS: We conducted a system literature review of HTA-related guidelines by searching the websites of HTA agencies and related data sources. The guidelines, reports, or recommendations introducing the HTA evaluation methods, processes, decision-making frameworks, and criteria for priority setting were eligible to be included. The review was limited to guidelines from countries belonging to the European Network for Health Technology Assessment (EUnetHTA) and HTAsiaLink organisations and other countries with well-established available guidelines. RESULTS: A total of 52 guidelines from 24 countries were identified, including 13 countries from the EUnetHTA organisation, 9 countries from the HTAsiaLink organisation and 2 other countries (Canada and the USA). A strong consensus was observed among the HTA agencies on the core set of criteria including efficacy or effectiveness, cost-effectiveness, safety, and budget impact. More similarities were observed than differences in methodological recommendations for clinical and economic evaluations among the agencies. CONCLUSIONS: Substantial convergence is seen in the criteria included in the HTA process, as well as the methods to evaluate and quantify them. Further efforts are needed to verify whether the criteria identified from the guidelines are incorporated in real HTA decisions, and how they are assessed and weighted in practice.

Quality assessment of randomized controlled trials reporting on knee osteoarthritis treated with warming needle moxibustion.

OBJECTIVE: To evaluate the quality of randomized controlled trials (RCTs) testing the efficacy of warming needle moxibustion on knee osteoarthritis (KOA). METHODS: Nine databases were searched systematically including: the Cochrane Library (1/1993-10/2012), PubMed (1/1980-10/2012), EMBASE (1/1990-10/12012), SCI Expanded (1/1986-10/2012), Science Direct (1/1979-10/2012), Chinese Biomedical Literature Database disc (1/1978-10/2012), China National Knowledge Infrastructure Database (1/1979-10/2012), China Science and Technology Journal Database (a full text issue database of China, 1/1989-10/2012), and Wanfang Database (a full text issue database of China, 1/1990-10/12012). Traditional reference retrieval was also conducted. Language was limited to Chinese and English. We identified 37 RCTs that used warming needle moxibustion as an intervention and they were assessed with the Consolidated Standards for Reporting of Trials Statement 2010 (CONSORT 2010) and Standards for Reporting Interventions Controlled Trials of Acupuncture 2010 (STRICT A 2010). RESULTS: Of the 37 reports, only seventeen (45.95%) mentioned the random allocation of sequence methods, nine used unacceptable methods, and only one (2.70%) gave the description of the mechanism of allocation concealment. One study (2.70%) mentioned blinding, one (2.70%) had a sample size calculation, and twenty-three (62.16%) used reasonable statistical methods. Thirteen (35.14%) described the background and reason for the RCTs, three (8.11%) gave a description of lost or eliminated patients, and two (5.41%) reported accidental situations. Only three (8.11%) gave analysis of the RCT limitations. No report mentioned intentional analysis. CONCLUSION: The quality of the RCTs assessed in this study was from moderate to low. The design of RCTs, the methods of statistical analysis, and the description of reports information needs to be improved. CONSORT 2010 and STRICT A 2010 should be used to standardize the reporting of acupuncture RCTs in the future.