Ryan Haumschild, PharmD, MS, MBA, CPEL

Panelists discuss how intrathecal delivery of onasemnogene abeparvovec in the STEER study demonstrates statistically significant motor function improvements in older patients with SMA (ages 2-18 years) with favorable safety profiles, potentially expanding gene therapy access beyond the current age restriction of under 2 years.

Panelists discuss how gaps in SMA care persist despite highly effective treatments, particularly regarding racial and ethnic disparities in research participation and global access challenges due to high costs and infrastructure limitations in developing countries.

Panelists discuss how robust clinical evidence from major studies like the EPIC and OPERA trials demonstrates that progression independent of relapse activity (PIRA) is the primary driver of confirmed disability progression in patients with multiple sclerosis (MS), with clinical parameters over 3 to 6 months being the most meaningful measures of treatment impact.

Panelists emphasize that the approval of menin inhibitors has transformed the treatment landscape for KMT2A-rearranged acute myeloid leukemia (AML) by replacing historically limited and toxic chemotherapy-based strategies with a targeted, guideline-endorsed option that aligns with disease biology—offering renewed hope for both adult and pediatric patients, especially in the relapsed/refractory setting.

Panelists report that the AUGMENT-101 trial demonstrated meaningful clinical benefits of menin inhibitors in relapsed/refractory KMT2A-rearranged acute myeloid leukemia (AML)—with a 23% complete remission rate and manageable safety profile including differentiation syndrome—highlighting the importance of early recognition, patient education, and supportive care to optimize outcomes as these agents move toward becoming a new standard of care.

Panelists discuss how progression independent of relapse activity (PIRA) represents a distinct pathological process involving smoldering inflammation and neurodegeneration that drives disability in patients with multiple sclerosis (MS), particularly manifesting around midlife despite being present from disease onset.

Panelists discuss the future of Bruton tyrosine kinase (BTK) inhibitor therapies, considering emerging treatments, regulatory changes, evolving cost dynamics, and the current unmet needs in chronic lymphocytic leukemia (CLL) and mantle cell lymphoma (MCL) care, and how these factors may shape the landscape of treatment options.

Panelists discuss strategies and resources health care providers can offer to help alleviate the financial burden of long-term Bruton tyrosine kinase (BTK) inhibitor therapy for patients with chronic lymphocytic leukemia (CLL) and mantle cell lymphoma (MCL), focusing on support programs, cost-sharing solutions, and patient assistance initiatives.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

Panelists discuss how promising ongoing and upcoming clinical trials may reshape sequencing strategies in ALK+ non–small cell lung cancer (NSCLC).

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.

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.

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.

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.

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.

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.