Payer Policies and Access Strategies
Panelists discuss how payer policies and clinical pathways currently influence clinical decision-making for chronic lymphocytic leukemia (CLL) and mantle cell lymphoma (MCL) treatments and explore strategies that managed care organizations can implement to control costs while ensuring patients have access to the most appropriate BTK inhibitor therapy.
Overview of Spinal Muscular Atrophy
Panelists discuss how spinal muscular atrophy is an autosomal recessive genetic disease affecting motor neurons with 3 currently approved disease-modifying therapies that restore SMN protein expression, including 2 requiring ongoing treatment and 1 gene transfer therapy (onasemnogene abeparvovec) approved only for patients under 2 years of age.
Key Takeaways From DMD Treatment Data
Panelists discuss how the pooled 3-year data for delandistrogene moxeparvovec show sustained motor function stabilization and less deterioration compared with natural history, providing confidence in the therapy’s long-term benefits while acknowledging the need for survival data and addressing concerns about potential transgene dilution over time.
Targeting Menin: A New Frontier in AML
Panelists highlight that menin inhibitors represent a breakthrough in the treatment of KMT2A-rearranged AML by targeting the disease’s core epigenetic drivers, with early clinical trials like AUGMENT-101 showing promising results in heavily pretreated, high-risk patients and supporting the potential for a new therapeutic standard in this aggressive leukemia subtype.
Patient Profiles: Demographics and Disease Biology
Panelists explain that KMT2A-rearranged acute myeloid leukemia (AML) is a biologically aggressive and diagnostically complex leukemia subtype—especially prevalent in pediatric and therapy-related cases—with variable prognostic implications depending on fusion partners, underscoring the need for early molecular identification, personalized treatment strategies, and expanded access to targeted therapies and advanced diagnostics.
Inflation Reduction Act: Clinical Challenges
Panelists discuss the specific challenges and barriers clinicians might encounter when utilizing different BTK inhibitors under the regulations of the Inflation Reduction Act (IRA), including issues related to cost, access, and treatment guidelines.
Inflation Reduction Act: Impact on Clinical Pathways for BTK Inhibitors
Panelists discuss potential changes to clinical pathways for Bruton tyrosine kinase (BTK) inhibitor therapies as a result of the Inflation Reduction Act (IRA), focusing on how shifting costs and access regulations may impact treatment decisions and patient care strategies.
Data on Duchenne Muscular Dystrophy Treatment From MDA 2025
Panelists discuss how data from MDA 2025 demonstrate that delandistrogene moxeparvovec (gene therapy for DMD) shows statistically significant improvements in motor function outcomes including North Star Ambulatory Assessment, time to rise from floor, and 10-m walk/run compared with external control groups over 2 years of treatment.
Advancements & Challenges in Muscular Dystrophy Treatment
Panelists discuss how recent advancements in muscular dystrophy treatment have evolved toward truly disease-modifying therapies using gene replacement, antisense oligonucleotides, and gene transfer technologies, while highlighting the ongoing challenges of identifying at-risk populations and collecting comprehensive safety and efficacy data.
Prognosis and Perception of KMT2A AML
Panelists emphasize that patients with KMT2A-rearranged acute myeloid leukemia (AML) often face steep educational challenges at diagnosis, requiring clear communication about the subtype’s adverse-risk classification, biological complexity, and treatment implications—highlighting the need for accessible education, academic-community collaboration, and early referral to specialized care for optimal outcomes.
KMT2A AML Pathogenesis and Presentation
Panelists explain that KMT2A-rearranged acute myeloid leukemia (AML) is an aggressive, molecularly distinct leukemia subtype marked by menin-dependent transcriptional dysregulation, poor response to standard therapy, diagnostic challenges, and high relapse risk—underscoring the urgency of RNA-based diagnostics, early transplant consideration, and emerging menin-targeted treatments to improve outcomes.
Working With Policy Makers to Effectively Implement Biosimilars
Panelists discuss how policy makers can increase biosimilar adoption through financial incentives, streamlined interchangeability rules, and addressing patent litigation delays, emphasizing that biosimilar savings will ultimately help fund the next wave of health care innovation such as cell and gene therapies.
Key Pearls for Collaborating With Manufacturers for Successful Uptake of Biosimilars
Panelists discuss how biosimilar manufacturers can increase uptake by ensuring positive financial outcomes for all stakeholders, maintaining strong patient assistance programs, and working closely with health care providers who significantly influence biosimilar implementation.
Inflation Reduction Act: Impact on Health Care Systems’ Utilization of BTK Inhibitors
Panelists discuss how the Inflation Reduction Act (IRA) might influence health care systems’ selection and utilization of Bruton tyrosine kinase (BTK) inhibitors, particularly through changes in pricing, reimbursement policies, and treatment access.
Inflation Reduction Act: Impact on Affordability of BTK Inhibitors
Panelists discuss how lower out-of-pocket costs resulting from ibrutinib’s inclusion in the Inflation Reduction Act (IRA) might improve the affordability and utilization of BTK inhibitor treatments in chronic lymphocytic leukemia (CLL) and mantle cell lymphoma (MCL), potentially expanding access for patients.
Looking Ahead: The Next Frontier in ALK+ NSCLC Therapy
Panelists discuss how promising ongoing and upcoming clinical trials may reshape sequencing strategies in ALK+ non–small cell lung cancer (NSCLC).
Education and Support After Progression ALK Inhibitors
Panelists discuss how to approach patient education after progression on an ALK inhibitor, address real-world challenges during therapy transitions, and determine best options for patients progressing on lorlatinib.
Optimizing the Implementation of Biosimilars Into Formularies
Panelists discuss how health systems can learn from published case examples when implementing biosimilars, noting that biosimilars can create a “win-win” situation where patients see lower costs, payers see lower charges, and providers may experience better margins.
Examining the Role of Interchangeability in Clinical Practice
Panelists discuss how implementing interchangeable biosimilars requires effective communication with providers and patients, emphasizing that educating stakeholders before making changes is essential for successful adoption despite the legal permissions afforded by interchangeability designation.
Balancing Clinical Benefits and Costs of BTK Inhibitors
Panelists discuss how to balance the clinical benefits of Bruton tyrosine kinase (BTK) inhibitor therapies with their associated costs when making treatment decisions for chronic lymphocytic leukemia (CLL) and mantle cell lymphoma (MCL), considering both patient outcomes and financial constraints.
Impact of Access Challenges on Patient Outcomes in CLL and MCL Care
Panelists discuss the primary access challenges in chronic lymphocytic leukemia (CLL) and mantle cell lymphoma (MCL) treatment, including financial barriers and health care system limitations, and how these obstacles impact patient outcomes and treatment efficacy.
Next Steps: Post-Frontline Therapies and Resistance in ALK+ NSCLC
Panelists discuss how alectinib use in the adjuvant setting might shift sequencing strategies in metastatic ALK+ non–small cell lung cancer (NSCLC) and how resistance mutations influence post-frontline therapy selection.
Equitable Access to Molecular Testing and Treatment Prioritization
Panelists discuss how to overcome barriers to comprehensive molecular testing and how to prioritize treatment when ALK+ non–small cell lung cancer (NSCLC) coexists with other actionable biomarkers.
Cost Savings Potential Associated With the Use of Biosimilars
Panelists discuss how biosimilars for adalimumab have faced unique challenges in the prescription benefit space compared with earlier oncology biosimilars, highlighting the importance of interchangeability designation, advance planning, and stakeholder education when implementing biosimilar adoption strategies.
How the IRA is Reshaping Biosimilar Adoption
Panelists discuss how the Inflation Reduction Act (IRA) of 2022 has influenced biosimilar utilization in the US health care system through its pharmacy provisions targeting Medicare patients, examining institutional impacts on adoption patterns, exploring payer preferences between high- and low-wholesale acquisition cost (WAC) therapies under the new regulatory framework, forecasting the evolving role of biosimilars at health care institutions, and identifying persistent barriers to uptake alongside potential strategies to overcome these challenges.
Long-Term Success: Educating and Supporting Patients on ALK Inhibitors
Panelists discuss how proactive patient education, lipid-lowering interventions, and monitoring tools can help patients stay on ALK inhibitor therapy to realize long-term benefits.
Lorlatinib’s Unique Challenges: Managing Cognitive and Metabolic AEs
Panelists discuss how lorlatinib’s unique adverse event (AE) profile, including cognitive effects, hypercholesterolemia, and peripheral neuropathy, impacts treatment selection and patient management.
The Road to Biosimilar Adoption: Navigating Interchangeability
Panelists discuss how stakeholders can prepare for the upcoming interchangeability designation of more biosimilars later this year, debating whether interchangeability status or formulary-level changes are more impactful and identifying necessary systemic shifts required to ensure both patients and health systems realize meaningful cost savings when transitioning from reference products to biosimilars.
The Biosimilar Shift: How PBMs Are Reshaping Formularies
Panelists discuss how major PBMs' (pharmacy benefit managers) decisions to prefer biosimilars over reference biologics provide valuable insights into successful adoption strategies, specifically examining CVS Caremark's removal of reference biologic adalimumab from national commercial template formularies, which resulted in 97% of prescriptions being filled with preferred biosimilars, and analyzing the key implementation factors driving this remarkable conversion rate.
Navigating ALK Inhibitor Toxicities
Panelists discuss how ALK inhibitors compare in terms of tolerability and best practices for managing associated toxicities.