Inflation reduction act impact on pharmaceutical investment: insights from investor interviews.

Introduction: There is limited direct measurement of whether the Inflation Reduction Act (IRA) is beginning to influence investment strategy and decisions.

Methods: Using a standardized guide, we interviewed life science investors from a range of stages, investment sizes, and fund types to explore how incentives under the IRA have impacted investment decisions.

Results: We interviewed 31 active investors.

Early impact of the Inflation Reduction Act on small molecule vs biologic post-approval oncology trials.

Introduction: Under the Inflation Reduction Act (IRA), small molecule drugs are subject to a shorter timeline toward eligibility for selection to the Drug Price Negotiation Program (DPNP) than biologics (7 vs 11 years post-approval), raising concerns about incentives for post-approval clinical development.

Methods: Using Citeline's Trialtrove database (7/2014-8/2024), this longitudinal study explored the impact of IRA's passage on industry-sponsored, post-approval phase I-III clinical trials in small molecule vs biologic oncology drugs, excluding vaccine-related trials. We used a difference-in-difference design to explore the impact of the IRA's differential DPNP timeline on small molecule trials in oncology by comparing changes in the number of newly initiated post-approval trials in small molecule drugs after the IRA (first difference) with changes in biological trials (second difference).

Prescription Drug Utilization and Spending by Race, Ethnicity, Payer, Health Condition, and US State.

Importance: Achieving equitable access to medicines requires understanding of how pharmaceutical use and spending vary by race and ethnicity across the US.

Objective: To quantify variation in prescription drug utilization and spending per capita and per prevalent case by race, ethnicity, health condition, payer, and US state.

Design, Setting, And Participants: In this cross-sectional study, the US Disease Expenditure project was extended to incorporate disaggregation by race and ethnicity for state-level retail prescription drug utilization and spending-in addition to 143 health conditions, 38 age and sex groups, and 4 payers (Medicare, Medicaid, private insurance, and out of pocket)-across the 2019 population in all 50 states and Washington, DC.

Challenges with judging and interpreting a drug's launch price.

This commentary explains why comparing a launch price with a value-based price from a cost-effectiveness analysis requires further examination.

Drug Coverage Policies And Clinical Guidelines Alignment: Most Coverage Decisions Include Additional Restrictions.

Utilization management criteria influence patients' access to specialty drugs, yet the processes used by health plans to establish these criteria are not well understood. This study examined the alignment between clinical practice guidelines and plans' utilization management criteria. Using the Tufts Medicine Specialty Drug Evidence and Coverage Database (December 2023), we reviewed US-based guidelines for 389 drug-indication pairs, excluding oncology and biosimilar treatments.

The Inflation Reduction Act and Drug Development: Potential Early Signals of Impact on Post-Approval Clinical Trials.

Background: The Inflation Reduction Act's (IRA) Drug Price Negotiation Program (DPNP) may reduce incentives for industry investments in post-approval clinical development. We aimed to explore the IRA's impact on the initiation of industry-sponsored, post-approval clinical trials.

Methods: Using Citeline's Trialtrove database (7/2014-8/2024), we conducted an interrupted time series analysis (ITSA) to estimate the IRA's impact on the initiation of industry-sponsored Phase I-III trials in previously approved drugs, excluding all vaccines and COVID-19 treatments.

Identifying the Influential Dynamic Inputs in Cost-Effectiveness Analyses.

Objectives: Compare cost-effectiveness estimates calculated using a static approach to pricing over time with an approach that accounts for price changes over time (a dynamic approach) and identify which dynamic inputs have the greatest influence on cost-effectiveness.

Methods: A panel of economic modeling experts identified cost-effectiveness analysis model inputs that change over time, possible values for each dynamic input, and approaches for incorporating dynamic inputs into an economic model. Informed by the advisory panel, static and dynamic cost-effectiveness estimates were calculated for 4 cases: (1) a chronically administered treatment for a chronic condition, (2) a chronically administered treatment for a catastrophic condition, (3) a 1-time treatment for a chronic condition, and (4) a 1-time treatment for a catastrophic condition.

US Drug Pricing Patterns Before Loss of Exclusivity.

Objectives: Most cost-effectiveness analyses assume that a drug's price remains unchanged after launch. Because prices typically change, this assumption can distort cost-effectiveness projections. Limited data on how prices change over a drug's life cycle have limited the inclusion of dynamic pricing assumptions.

Improving access to gene therapies: a holistic view of current challenges and future policy solutions in the United States.

Gene therapies hold the promise of delivering groundbreaking improvements in health outcomes for previously intractable diseases. These therapies possess unique characteristics that differ from traditional small molecule or biologic treatments, posing new challenges for healthcare stakeholders. While previous analyses have predominantly focused on the payment challenges associated with emerging gene therapies, a more holistic examination reveals numerous obstacles that currently hinder optimal patient access.

CMS's Drug Price Negotiation Program 2023 Patient-Focused Listening Sessions: A Descriptive Analysis of Speaker Remarks.

Background: The US Centers for Medicare and Medicaid Services (CMS) held patient-focused listening sessions in Fall 2023 for each of the first ten drugs selected for the Inflation Reduction Act's (IRA) Drug Price Negotiation Program (DPNP). This study aimed to quantitatively describe speaker input at the sessions, including the absolute and relative time allocated to key areas of interest for the DPNP.

Methods: In this descriptive analysis of speaker remarks from ten CMS-hosted patient-focused listening sessions, speaker demographics were examined using video streams, time-stamped transcripts, public data, contextual clues, validated tools, and visual assessment when necessary.

Subsequent Indications in Oncology Drugs: Pathways, Timelines, and the Inflation Reduction Act.

Introduction: Recent research has raised questions about potential unintended consequences of the Inflation Reduction Act's Drug Price Negotiation Program (DPNP), suggesting that the timelines introduced by the law may reduce manufacturer incentives to invest in post-approval research towards additional indications. Given the role of multiple indications in expanding treatment options in patients with cancer, IRA-related changes to development incentives are especially relevant in oncology. This study aimed to describe heterogeneous drug-level trajectories and timelines of subsequent indications in a cohort of recently approved, multi-indication oncology drugs, including overall, across subgroups of drugs characterized by the timing and pace of additional indications, and by drug type (i.

The accelerated approval program for oncology drugs: celebrating more than 250,000 life-years gained and counting.

This commentary explores how 2 recently published studies evaluating the clinical benefit of the FDA's accelerated approval program for oncology drugs came to different conclusions.

Cost-Effectiveness of HIV Screening in Emergency Departments: Results From the Pragmatic Randomized HIV Testing Using Enhanced Screening Techniques in Emergency Departments Trial.

Study Objective: Identification of HIV remains a critical health priority for which emergency departments (EDs) are a central focus. The comparative cost-effectiveness of various HIV screening strategies in EDs remains largely unknown. The goal of this study was to compare programmatic costs and cost-effectiveness of nontargeted and 2 forms of targeted opt-out HIV screening in EDs using results from a multicenter, pragmatic randomized clinical trial.

An Alternative Measure of Health for Value Assessment: The Equal Value Life-Year.

The quality-adjusted life-year (QALY) is an international standard in cost-effectiveness analysis. A known concern arises from the relatively lower QALY gains attributed to treatments that extend the life of individuals with chronic disability. We analyze here the advantages and disadvantages of the equal value life-year (evLY) as an alternative or a complementary measure to the QALY, and share learned experiences from using this measure in health technology assessments.

Cost-Effectiveness Analysis of New Beta-Lactam Beta-Lactamase Inhibitor Antibiotics Versus Colistin for the Treatment of Carbapenem-Resistant Infections.

Carbapenem-resistant organisms (CROs) present a serious public health problem. Limited treatment options has led to increased use of colistin and polymyxin. Since 2014, the US Food and Drug Administration approved 4 new beta-lactam beta-lactamase inhibitor (BLBLI) combination antibiotics with activity against CROs.

The Cost-Effectiveness of Remdesivir for Hospitalized Patients With COVID-19.

Objectives: This study aimed to estimate the cost-effectiveness of remdesivir, the first novel therapeutic to receive Emergency Use Authorization for the treatment of hospitalized patients with COVID-19, and identify key drivers of value to guide future pricing and reimbursement efforts.

Methods: A Markov model evaluated the cost-effectiveness of remdesivir in patients hospitalized with COVID-19 from a US healthcare sector perspective. A lifetime time horizon captured potential long-term costs and outcomes.

Performing Cost-Effectiveness Analyses to Support Policy Making: Key Lessons From the Assessment of Aducanumab.

The purpose of this article is to describe the process and the methods of cost-effectiveness analysis for clinicians interested in joining or leading aspects of this branch of evidence-based research. Cost-effectiveness is a useful tool for policymakers and is considered a starting point for discussions of fair pricing. Clinicians are important members of teams conducting cost-effectiveness analyses, particularly as it relates to integrating their clinical expertise into the decisions around the design and conduct of the analysis.

Cost-Effectiveness and Value-Based Pricing of Aducanumab for Patients With Early Alzheimer Disease.

Background And Objectives: Aducanumab was granted accelerated approval with a conflicting evidence base, near-unanimous Food and Drug Administration Advisory Committee vote to reject approval, and a widely criticized launch price of $56,000 per year. The objective of this analysis was to estimate its cost-effectiveness.

Methods: We developed a Markov model to compare aducanumab in addition to supportive care to supportive care alone over a lifetime horizon.

Comparison of HIV Screening Strategies in the Emergency Department: A Randomized Clinical Trial.

Importance: The National HIV Strategic Plan for the US recommends HIV screening in emergency departments (EDs). The most effective approach to ED-based HIV screening remains unknown.

Objective: To compare strategies for HIV screening when integrated into usual ED practice.

Patient and Payer Preferences for Additional Value Criteria.

Defining the value of healthcare is an elusive target, and depends heavily on the decision context and stakeholders involved. Cost-utility analysis and the quality-adjusted life year (QALY) have become the method and value definition of choice for traditional value judgements in coverage and pricing decisions. Other criteria that may influence value are often not measured and therefore omitted from value assessments, or are only used to qualitatively contextualize assessments.

Proposal for capturing patient experience through extended value frameworks of health technologies.

Inclusion of patient experience (PEx) in health technology assessment (HTA) has become increasingly important; however, no harmonized approach exists to help manufacturers or decision makers ensure PEx considerations are fair, consistent, and thorough within global HTA frameworks. To develop a proposal for including PEx in the HTA frameworks of health technologies. A systematic literature review (SLR) on existing value frameworks (VFs) was conducted to capture how PEx-related value judgment is currently considered.

Economic and clinical burden of comorbidities among patients with acromegaly.

Objective: Acromegaly is a rare, pituitary hormonal disorder that requires improved awareness worldwide. The objective of this analysis was to quantify the clinical and economic burden of comorbidities for patients with acromegaly and examine the influence of biochemical control on these outcomes.

Study Design: Markov cohort decision analytic model consisting of two states, including alive (with and without comorbidity) and dead.

Criteria and Scoring Functions Used in Multi-criteria Decision Analysis and Value Frameworks for the Assessment of Rare Disease Therapies: A Systematic Literature Review.

Background: Traditionally, the economic value of health technologies is assessed with cost-effectiveness (CE) and budget impact (BI) analyses. However, the evaluation of rare disease therapies often considers novel value criteria. Multi-criteria decision analysis (MCDA) is a promising tool in the assessment of value criteria that typically cannot be captured with traditional approaches.