Video Series

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 data systems and registries should focus on improving care quality rather than just reimbursement, with patient empowerment strategies that encourage individuals to advocate for better treatments like de-prescribing harmful medications.

Panelists discuss how biomarkers, including genetic testing and tumor profiling, can help identify patients with RAS/MEK/ERK pathway activation or specific NF1 mutations, enabling more personalized and effective treatment with therapies like mirdametinib or gene therapies for progressive, symptomatic NF1-associated plexiform neurofibromas.

A panelist discusses how recent insights into dry eye disease highlight the importance of targeting both inflammation and tear production—showcasing treatments like cyclosporine drops and varenicline nasal spray—while emphasizing ongoing research aimed at addressing meibomian gland dysfunction and improving tear film stability for more effective, comprehensive care.

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 discuss how organizations must create system-wide cultural changes through staff training, appropriate clinical environments, evidence-based treatment access, and incentive structures that support rather than punish providers for addressing obesity as a chronic disease.

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 the future of PAH treatment looks promising with potential for disease remission through reverse remodeling agents, emphasizing the need for continued research focus on patients with other forms of pulmonary hypertension and those with significant comorbidities.

Panelists discuss how transitioning patients from intravenous (IV) to subcutaneous (SubQ) therapies demands a coordinated, multidisciplinary approach—integrating clinical judgment, electronic medical record (EMR) readiness, workflow adaptation, and patient-centered communication to ensure safe, efficient, and individualized care.

Panelists discuss that improving outcomes for patients with idiopathic pulmonary fibrosis (IPF) and progressive pulmonary fibrosis (PPF) requires multifaceted efforts including developing patient-centered clinical measures, managing comorbidities, leveraging digital health tools for real-time monitoring, and addressing health equity to ensure timely, personalized care that truly reflects patients’ lived experiences.

Panelists discuss the shift toward patient-centered outcomes in idiopathic pulmonary fibrosis and progressive pulmonary fibrosis treatment, emphasizing quality of life and symptom relief alongside lung function, while expressing optimism that emerging therapies may eventually reverse or prevent fibrosis, fundamentally transforming disease management.

Panelists discuss how the rise of patient awareness around subcutaneous (SubQ) therapies calls for empathetic, evidence-based education, shared decision-making, and seamless system integration to ensure that treatment choices align with clinical appropriateness, patient preferences, and operational readiness.

An expert discusses how multidisciplinary collaboration can be enhanced through shared knowledge of available drugs and biomarkers among oncologists and pathologists, standardized interpretation methods across different cancer types, and clear communication about trial-specific algorithms to optimize treatment plans and ensure proper biomarker assessment.

Panelists discuss how emerging therapies like CAR T cells and bispecific antibodies may transform frontline treatment by potentially replacing transplant or changing induction regimens, while considering the cost implications and need for sustainable care models.

An expert discusses how the biggest barriers to incorporating antibody-drug conjugates (ADCs) include lacking biomarker knowledge and how payer restrictions can be overcome through early biomarker testing, proper documentation of target expression levels, and following FDA or National Comprehensive Cancer Network (NCCN) guidelines to ensure patient access to appropriate therapies.

Panelists discuss how early relapse in standard-risk patients represents a failure of current risk assessment methods and may require advanced sequencing technologies to identify hidden high-risk features that traditional fluorescence in situ hybridization testing misses.

A panelist discusses early clinical trial data on combining the B-cell maturation antigen–targeting bispecific antibody linvoseltamab with proteasome inhibitors carfilzomib and bortezomib in relapsed/refractory multiple myeloma, highlighting promising response rates, manageable toxicity profiles, and the potential for these combinations to enhance treatment efficacy pending longer-term follow-up.

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.

A panelist discusses how the ASCERTAIN-V study demonstrated that an all-oral combination of decitabine-cedazuridine plus venetoclax achieved a 47% complete response rate and 15.5-month median overall survival in older, unfit AML patients, representing a potential new standard of care that eliminates the need for intravenous infusions and significantly reduces clinic time burden while serving as a backbone for future oral combination therapies.

Panelists discuss how unconscious bias and weight stigma create barriers to care, requiring providers to acknowledge their own biases, create welcoming clinical environments with appropriate accommodations, and approach patients with compassion rather than judgment about willpower.

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 effective provider-patient communication about weight requires opening conversations with empathy and collaboration, using phrases like "I'm concerned about your weight" and avoiding stigmatizing language while understanding patient motivations and readiness for change.

Panelists discuss how mirdametinib's oral administration offers convenience and improves compliance, especially in pediatric patients, and how its ability to stabilize progressive, symptomatic NF1-associated plexiform neurofibromas without the need for invasive procedures fills a critical gap in treatment options while also addressing potential access barriers related to insurance coverage and cost.