MarketVue® Report
Long QT Syndrome (LQTS) (U.S.), 2023

Syndicated market assessment report driven by primary market research

REPORT STORE

Long QT Syndrome (LQTS) (U.S.), October 2023

DOWNLOAD SAMPLE REPORT
DOWNLOAD REPORT BROCHURE
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.
 
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: Gain insights into the drug pricing landscape and payer controls within the disease market or analogous markets
 
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 by REACH, 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

Consequently, there is a need for additional pharmaceutical treatments to reduce the reliance upon surgical options for refractory patients. Additionally, while a large majority of patients are well-managed by beta blockers, up to half of these patients struggle with the side effects and could benefit from a better-tolerated treatment option. Unfortunately for LQTS patients, the clinical pipeline is sparse, with only one industry-sponsored drug in development therefore it will likely be 5 or more years before any new treatments become available.

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.”

1. DISEASE OVERVIEW
LQTS subtypes
Table 1.1. Comparison of the three most common LQTS subtypes
2. EPIDEMIOLOGY & PATIENT POPULATIONS
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
3. DIAGNOSIS & CURRENT TREATMENT
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
4. UNMET NEED
Overview
Figure 4.1. Top unmet needs in LQTS
High unmet need patient types
5. PIPELINE ANALYSIS
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
6. VALUE & ACCESS
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
7. METHODOLOGY
Primary market research approach
Epidemiology methodology
Long QT syndrome disease definition
Long QT syndrome incidence estimates
Long QT syndrome prevalence estimates
References
What is the difference between purchasing a published report and requesting a new report?

Published reports have been pre-produced on the specified date and can be delivered immediately. If you prefer to refresh an existing report or request an entirely new disease, REACH can create and deliver any new report within 15 business days.

How does REACH produce new primary market research reports in 15 business days?

By deploying our senior team to each of our reports, we can begin insight generation on day 1. The REACH team also recruits Key Opinion Leaders from our internal panel rather than outsourcing recruitment which enables us to quickly begin primary market research interviews.

Who writes REACH reports?

Each REACH report is produced by one of our senior US-based team members with 10+ years of experience in life sciences market research. We are deeply committed to ensuring REACH reports are high quality and strategic to enable informed decision-making for our clients.

Does REACH cover both rare and non-rare diseases?

REACH specializes in covering diseases and subpopulations that are not addressed by traditional market research reports. As such, we focus on rare diseases and niche subpopulations of more common conditions (e.g., Dupixent-refractory atopic dermatitis). Our flexible model enables in-depth assessments across all disease types, allowing us to support research for non-rare conditions as well.

Can I meet with the team to discuss the report?

Contact us to set up a call directly with the analyst who has written the report to answer any questions and discuss the key takeaways.

Published Report

Download immediately.


New Report

Updated upon purchase. Delivered in 15 days.


Undecided about purchasing this report?

» Inquire before buying

Reach out to learn more about report bundle discounts

Reach out to our team to learn more about our capabilities and view sample reports.

Contact us