The MarketVue®: Long QT Syndrome (LQTS) market landscape report combines primary (KOL interviews) and secondary market research to empower strategic decision-making and provide a complete view of the market.
Every MarketVue® includes a disease overview, epidemiology (US and EU5), current treatment, unmet needs, pipeline and access and reimbursement chapter.
According to LQTS experts interviewed, most cases of LQTS, especially LQTS type 1, are well-controlled by the current standard of care - beta blockers - despite less the less-than-ideal side effects they cause (e.g., fatigue, depression, decreased libido). However, for high-risk patients that experience breakthrough events despite maximal tolerated beta blocker treatment, physicians turn to surgical options that include implantable cardioverter-defibrillators (ICDs) or left cardiac sympathetic denervation (LCSD). While surgical options are largely effective at controlling symptoms (LCSD) or reliably correcting potentially fatal arrhythmias (ICD), these approaches are used as a last resort and come with notable limitations such as:
Cardiac Electrophysiologist, U.S.: 'I have a bunch of ICD patients who have abandoned leads or who have had multiple lead extraction procedures that are high risk, there's been a perforation, and they had to have a sternotomy and have gone really, really bad. A lot of those are my patients with long QT syndrome who have had an ICD since childhood.
Every MarketVue® includes a disease overview, epidemiology (US and EU5), current treatment, unmet needs, pipeline and access and reimbursement chapter.
Topics covered in this report:
- Disease overview: Review the disease pathophysiology and potential druggable targets
- Epidemiology: Understand prevalence, diagnosed and drug-treated prevalence of the population and key market segments
- Current treatment: Understand the treatment decision tree and strengths and weaknesses of current on-label and off-label treatment
- Unmet needs: Identify opportunities to address treatment or disease management gaps
- Pipeline analysis: Compare current and emerging therapy clinical development strategy; their performance on efficacy, safety, and delivery metrics; and their potential to address unmet needs
- Value and access: Review the evidence needed to assess and communicate value to key stakeholders (e.g., providers, payers, regulators) and learn what competitors have done or are doing
Methodology:
Research for the MarketVue®: Long QT Syndrome report is supported by 10 qualitative interviews with key opinion leaders and secondary research.Geographies covered:
United States plus epidemiology for EU5 (France, Germany, Italy, Spain, United Kingdom).Key companies mentioned:
- Thryv Therapeutics
- Vertex
Key drugs mentioned:
- LQT-1213
- Lumacaftor / Ivacaftor (Orkambi)
Key takeaways from the report:
Long QT syndrome (LQTS) is a rare congenital cardiac arrhythmia characterized by a prolonged recovery in cardiac action potential that can cause deadly arrhythmias.According to LQTS experts interviewed, most cases of LQTS, especially LQTS type 1, are well-controlled by the current standard of care - beta blockers - despite less the less-than-ideal side effects they cause (e.g., fatigue, depression, decreased libido). However, for high-risk patients that experience breakthrough events despite maximal tolerated beta blocker treatment, physicians turn to surgical options that include implantable cardioverter-defibrillators (ICDs) or left cardiac sympathetic denervation (LCSD). While surgical options are largely effective at controlling symptoms (LCSD) or reliably correcting potentially fatal arrhythmias (ICD), these approaches are used as a last resort and come with notable limitations such as:
- ICDs, especially when implanted in children, have a high risk of malfunction and require continued maintenance surgeries like battery replacement
- LCSD recovery can be very painful and there is a shortage of surgeons trained to perform LCSD
Cardiac Electrophysiologist, U.S.: 'I have a bunch of ICD patients who have abandoned leads or who have had multiple lead extraction procedures that are high risk, there's been a perforation, and they had to have a sternotomy and have gone really, really bad. A lot of those are my patients with long QT syndrome who have had an ICD since childhood.
Table of Contents
1. DISEASE OVERVIEW- LQTS subtypes
- Table 1.1. Comparison of the three most common LQTS subtypes
- Disease definition
- Figure 2.1. Diagnosed prevalent cases of LQTS by region
- Incidence of LQTS
- Prevalence of LQTS
- Diagnosis and drug treatment rates
- Table 2.1 Diagnosed incident and prevalent LQTS cases in the US and EU5
- Most common subtypes of LQTS
- Figure 2.2. Proportion of LQTS patients with the three most common subtypes
- Table 2.2. Description of LQTS subtypes
- Diagnosis overview
- Figure 3.1. Diagnosis flow of LQTS patients
- Risk stratification is an important prognostic tool for LQTS
- Figure 3.2. Factors considered in patient risk status
- LQTS has evolved from an often-deadly diagnosis to a chronically managed condition
- Treatment overview
- Figure 3.3. Treatment goals for LQTS - physician versus patient priorities
- Treatment flow for LQTS
- Figure 3.4. Treatment algorithm for LQTS
- Comparison of approved treatments for LQTS
- Figure 3.5. Comparison of approved treatments for LQTS
- Opportunity for better treatments exist for certain groups of high-risk or intolerant patients
- Figure 3.6. KOL estimates of the proportion LQTS patients that are poorly managed
- Table 3.1. Standard of care - upside and downside
- Disease burden and key treatment dynamics that shape disease management in LQTS
- Table 3.2. Must-know LQTS treatment dynamics for now and the future
- No significant changes are anticipated to disrupt the LQTS market in the foreseeable future
- Figure 3.7. Important dynamics of LQTS market evolution
- Overview
- Figure 4.1. Top unmet needs in LQTS
- High unmet need patient types
- Looking backward
- Looking forward
- Figure 5.1. Evolution of pharmaceutical targets for LQTS
- There is only one industry sponsored clinical trial in LQTS
- Table 5.1.Comparison of active trials for LQTS
- Physician insights on various potential LQTS therapies
- Opportunity for novel therapy in LQTS
- Overview
- Table 6.1. Comparison of treatment pricing, U.S.
- Pricing analogue; camzyos (mavacamten) for hypertrophic cardiomyopathy
- Table 6.2. Typical U.S. commercial payer coverage
- Primary market research approach
- Epidemiology methodology
- Long QT syndrome disease definition
- Long QT syndrome incidence estimates
- Long QT syndrome prevalence estimates
- References
Samples
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Companies Mentioned
- Thryv Therapeutics
- Vertex
