Ryan Haumschild, PharmD, MS, MBA, CPEL

Panelists discuss how the evaluation process must evolve to accommodate therapies that show benefits in slowing atrophy and motor decline over 1 to 2 years, emphasizing the need for early coverage rather than waiting for extensive long-term data.

Panelists discuss how disability progression can be measured through practical clinical tools like patient-reported outcomes, timed walking tests, and dexterity assessments rather than relying solely on relapse rates.

Panelists expressed cautious optimism about high-risk acute myeloid leukemia (AML) treatment, emphasizing the need for ongoing research, personalized therapy based on molecular profiling, and strengthened collaboration between community and academic centers to improve patient outcomes, while recognizing that education and sharing best practices are key to advancing targeted therapies and achieving long-term cures.

Panelists highlight that patient advocacy is crucial in improving high-risk acute myeloid leukemia (AML) outcomes by providing emotional support, funding research, influencing clinical guidelines, promoting patient engagement, facilitating collaboration between community and academic care, addressing systemic access barriers, and implementing innovative programs like medication recycling to enhance treatment availability.

Panelists discuss how payers can evaluate BTK inhibitors by considering specific MS phenotypes and FDA indications rather than applying traditional step therapy protocols that may delay optimal treatment.

Panelists discuss how BTK inhibitors represent a promising new oral therapy class that could address both inflammatory and neurodegenerative aspects of MS, particularly for progressive forms where treatment options are limited.

Panelists advocate for a streamlined approach to diagnosing KMT2A-rearranged acute myeloid leukemia (AML) that includes standardized testing protocols (cytogenetics, fluorescence in situ hybridization [FISH], and next-generation sequencing) at diagnosis, close collaboration with laboratories to clarify complex reports, adoption of rapid testing methods, and flexible sample collection strategies to ensure timely, accurate identification and optimized patient care.

Panelists discuss how payers seek good return on investment when evaluating expensive gene therapies, creating potential friction when innovative treatments come at significant costs, requiring ongoing dialogue between manufacturers, patients, payers, and physicians to determine appropriate value and access.

Panelists highlight that despite established guidelines, delays and gaps in molecular profiling—especially for KMT2A rearrangements—persist due to report complexity, misinterpretation, and misconceptions about patient eligibility, underscoring the urgent need for improved provider education, expert collaboration, and comprehensive testing to ensure accurate diagnosis, optimal treatment selection, and better patient outcomes in acute myeloid leukemia (AML).

Panelists discuss how emerging biomarkers like neurofilament light protein, glial fibrillary acidic protein (GFAP), cervical cord atrophy, and phase rim lesions could enable earlier identification of patients at risk for progression, supporting more effective treatment decisions and payer coverage determinations based on longitudinal disability data rather than just relapse activity.

Panelists stress that timely and accurate diagnosis of KMT2A-rearranged acute myeloid leukemia (AML) is essential for personalized treatment planning, highlighting the need to overcome systemic delays in molecular testing, foster academic-community collaboration, and educate both clinicians and patients on the safety and importance of waiting for complete genetic data before initiating therapy.

Panelists discuss how patient perspectives on new therapies center around clinically meaningful outcomes and survival while balancing individual risk tolerance, with some patients willing to accept higher risks for potentially transformative treatments in devastating diseases.

Panelists discuss how exciting ongoing research efforts are expanding similar gene therapy technologies to other muscular dystrophies like FSHD and myotonic dystrophy, using strategies to knock down rather than restore gene expression for these autosomal dominant conditions.

Panelists emphasize that effective use of menin inhibitors for KMT2A-rearranged acute myeloid leukemia (AML) requires dispelling common misconceptions about oral targeted therapy, ensuring close monitoring for adverse effects, clarifying treatment goals—especially the role of transplant—and supporting patients through comprehensive education and multidisciplinary care.

Panelists discuss how current multiple sclerosis (MS) therapies show limited effectiveness against progression, but emerging Bruton tyrosine kinase (BTK) inhibitors offer promise by targeting both B cells and central nervous system (CNS)–penetrating microglia, with one showing a 31% reduction in confirmed disability progression in clinical trials.

Panelists highlight that the emergence of menin inhibitors is reshaping the treatment paradigm for KMT2A-rearranged acute myeloid leukemia (AML), with ongoing research focused on optimizing their use, overcoming resistance, expanding access, and integrating these targeted therapies into personalized and potentially curative treatment strategies across diverse patient populations.

Panelists discuss how chronic neuroinflammation involves distinct mechanisms from acute relapses—including microglial activation, mitochondrial dysfunction, and iron deposition—necessitating dual therapeutic approaches that address relapsing and progressive disease components.

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.