Designing collaborations involving health technology assessment: discussions and recommendations from the 2024 health technology assessment international global policy forum.

Although collaboration is an intensive way of working together, it is essential for such efforts to achieve shared goals. Health technology assessment (HTA) is transdisciplinary and has an important history of collaboration, with collaboration featuring increasingly in the strategic plans of HTA bodies and stakeholders. Collaboration can be between HTA bodies and between HTA bodies and other stakeholders-most notably regulators but increasingly payers, patient and caregiver organizations, clinicians-clinical societies, and academia.

A network meta-analysis of KarXT and commonly used pharmacological interventions for schizophrenia.

Background: Dopaminergic antipsychotics for schizophrenia have modest effects on symptoms and can cause important side effects. KarXT is an investigational drug for schizophrenia with a novel mechanism targeting muscarinic receptors that may limit these side effects.

Methods: We conducted a systematic review and Bayesian random-effects network meta-analyses of short-term RCTs (3-8 weeks) that enrolled adults with schizophrenia.

Putting meat on the bone: how to fast-track innovative medicines to those who need them and generate data to justify continued use.

Regulatory agencies worldwide have taken significant steps to expedite approval and market authorization of medicines based on their potential to address areas of significant unmet medical need and severe disease burden. However, initial approval of such medicines is often accompanied by limited evidence of benefit, posing a conundrum for payers and health systems who may desire greater certainty of their value. This paper describes a system of "accelerated access" to manage these tensions and coordinate activities across stakeholders, based on discussions held at a multi-stakeholder convening in June 2023.

Informing the United States Medicare Drug Price Negotiation for Apixaban and Rivaroxaban: Methodological Considerations for Value Assessments Many Years After Launch.

Objectives: To demonstrate how health technology assessment methods can be used to support Medicare's price negotiations for apixaban and rivaroxaban.

Methods: Following the statutory outline of evidence that will be considered by Medicare, we conducted a systematic literature review, network meta-analyses, and decision analyses to evaluate the health outcomes and costs associated with apixaban and rivaroxaban compared with warfarin and dabigatran for patients with nonvalvular atrial fibrillation. Our methods inform discussions about the therapeutic impact of apixaban and rivaroxaban and suggest price premiums above their therapeutic alternatives over a range of cost-effectiveness thresholds.

The impact of capping health system cost savings on the projected cost-effectiveness of etranacogene dezaparvovec compared with factor IX prophylaxis for the treatment of hemophilia B.

This viewpoint discusses cost-effectiveness estimates for EtranaDez, a gene therapy for hemophilia B, using the Institute for Clinical and Economic Review's (ICER) framework for single and short-term therapies (SSTs). EtranaDez offers long-term benefits from a single administration, in contrast to the high costs and frequent dosing required by current factor IX prophylaxis. However, the projected gains in health from EtranaDez are small relative to the cost implications of the therapy, and consequently, how the cost offsets associated with EtranaDez are counted has a substantial impact on assessing its cost-effectiveness.

Cost-effectiveness of eculizumab and efgartigimod for the treatment of anti-acetylcholine receptor antibody-positive generalized myasthenia gravis.

Background: Eculizumab and efgartigimod were approved to treat anti-acetylcholine receptor antibody-positive generalized myasthenia gravis (anti-AChR Ab-positive gMG). These relatively new biological treatments provide a more rapid onset of action and improved efficacy compared with conventional immunosuppressive treatments, but at a higher cost.

Objective: To assess the cost-effectiveness of eculizumab and, separately, efgartigimod, each added to conventional therapy vs conventional therapy alone, among patients with refractory anti-AChR Ab-positive gMG and those with anti-AChR Ab-positive gMG, respectively.

How Do the Institute for Clinical and Economic Review's Assessments of Comparative Effectiveness Compare With the German Federal Joint Committee's Assessments of Added Benefit? A Qualitative Study.

Objectives: We compared the Institute for Clinical and Economic Review's (ICER) ratings of comparative clinical effectiveness with the German Federal Joint Committee's (G-BA) added benefit ratings, and explored what factors may explain the disagreement between the 2 organizations.

Methods: We included drugs if they were assessed by ICER under its 2020 to 2023 Value Assessment Framework and had a corresponding assessment by G-BA as of January 2024 for the same indication, patient population, and comparator drug. To compare assessments, we modified ICER's proposed crosswalk between G-BA and ICER benefit ratings to account for G-BA's certainty ratings.

Ethnic Identity Protects and Internalized Racism Harms Health and Coping in Asian Americans Following COVID-19 Discrimination: A Mixed-Methods Study.

Mechanisms underlying the link between COVID-19 anti-Asian racial discrimination and psychological health are underexplored. This mixed-methods study examined the moderating effects of ethnic identity and internalized racism on the relationship between COVID discrimination and behavioral health outcomes among Asian Americans. We hypothesized that individuals with lower ethnic identity and higher internalized racism levels would demonstrate more adverse outcomes, including worsened psychological trauma and identity-avoidant behaviors, post-discrimination.

Economic Evaluations of Chimeric Antigen Receptor T-Cell Therapies for Hematologic and Solid Malignancies: A Systematic Review.

Objectives: This study aimed to systematically review evidence on the cost-effectiveness of chimeric antigen receptor T-cell (CAR-T) therapies for patients with cancer.

Methods: Electronic databases were searched in October 2022 and updated in September 2023. Systematic reviews, health technology assessments, and economic evaluations that compared costs and effects of CAR-T therapy in patients with cancer were included.

HTA community perspectives on the use of patient preference information: lessons learned from a survey with members of HTA bodies.

This research sought to assess whether and how patient preference (PP) data are currently used within health technology assessment (HTA) bodies and affiliated organizations involved in technology/drug appraisals and assessments. An exploratory survey was developed by the PP Project Subcommittee of the HTA International Patient and Citizen Involvement Interest Group to gain insight into the use, impact, and role of PP data in HTA, as well as the perceived barriers to its incorporation. Forty members of HTA bodies and affiliated organizations from twelve countries completed the online survey.

A framework for evaluating the diversity of clinical trials.

Objectives: The topic of diversity in clinical trials is rising to the forefront of many conversations in evidence-based medicine, and efforts are being made to improve the diversity of clinical trials. However, there is little uniformity in the methods used to evaluate these efforts. In this article, we describe our Clinical trial Diversity Rating (CDR) framework and the development process, including the broader considerations for evaluating the demographic diversity of clinical trials and their implications, and demonstrate its use through an illustrative example.

Gene therapies for sickle cell disease: Effectiveness and value.

Drs. Nikitin, McKenna, Rind, Nhan, and Pearson report grants from Arnold Ventures, grants from Blue Cross Blue Shield of MA, grants from California Healthcare Foundation, grants from The Commonwealth Fund, grants from The Patrick and Catherine Weldon Donaghue Medical Research Foundation, during the conduct of the study; other from America's Health Insurance Plans, other from Anthem, other from AbbVie, other from Alnylam, other from AstraZeneca, other from Biogen, other from Blue Shield of CA, other from CVS, other from Editas, other from Express Scripts, other from Genentech/Roche, other from GlaxoSmithKline, other from Harvard Pilgrim, other from Health Care Service Corporation, other from Kaiser Permanente, other from LEO Pharma, other from Mallinckrodt, other from Merck, other from Novartis, other from National Pharmaceutical Council, other from Premera, other from Prime Therapeutics, other from Regeneron, other from Sanofi, other from United Healthcare, other from HealthFirst, other from Pfizer, other from Boehringer-Ingelheim, other from uniQure, other from Envolve Pharmacy Solutions, other from Humana, other from Sunlife, outside the submitted work.