MarketVue® Report
Prion Disease (U.S.), 2024

Syndicated market assessment report driven by primary market research

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Prion Disease (U.S.), August 2024

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The MarketVue®: Prion Disease 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®: Prion Disease report is supported by 8 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
  • Ionis Pharmaceuticals
  • Sangamo Therapeutics
  • Gate Bioscience
 
Key drugs mentioned
  • Antiepileptics (levetiracetam, sodium valproate)
  • Long-acting benzodiazepines (clonazepam, diazepam)
  • Antipsychotics (quetiapine, risperidone, olanzapine)
  • Benzodiazepines (oxazepam, temazepam, alprazolam)
  • Sedatives (trazadone, mirtazapine)
  • ION717
  • hSYN1-ZF-R
1. DISEASE OVERVIEW
Figure 1.1. Prion disease pathophysiology
Table 1.1. Human prion diseases
2. EPIDEMIOLOGY
Figure 2.1. Incident cases of prion disease by region (2023)
Table 2.1. Diagnosed symptomatic incident cases of prion diseases in the U.S. and EU5 (2023)
Figure 2.2. Distribution of types of prion disease
Figure 2.3. Average months from prion disease onset of symptoms to death
Figure 2.4. Trends in prion disease incidence
3. DIAGNOSIS & CURRENT TREATMENT
Figure 3.1. Diagnostic pathway for prion disease
Figure 3.2. Neurologist estimates of diagnosis in prion diseases (n=4)
Figure 3.3. Key recent advancements in prion disease diagnosis
Figure 3.4. Neurologist rates of genetic testing in prion disease patients (n=8)
Figure 3.5. Drivers and barriers of genetic testing in prion disease patients
Table 3.1. Prion disease symptoms
Figure 3.6. Defining disease severity in prion disease
Table 3.2. Treatment goals for prion disease
Table 3.3. Pharmacological and non-pharmacological symptom control approaches for prion disease
4. UNMET NEED
Figure 4.1. Top unmet needs in prion disease
Figure 4.2. Neurologist-reported level of unmet need for novel prion disease treatments (n=8)
5. PIPELINE ANALYSIS
Table 5.1. Comparison of ongoing trials of therapies for prion disease
Figure 5.1. Ionis ASO pre-clinical data
Figure 5.2. Comparison of target prion disease patient populations
6. VALUE & ACCESS
Table 6.1. Pricing analogues for prion disease
7. METHODOLOGY
Primary Market Research Approach
Epidemiology
Value and Access 
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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.

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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.

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