Primary Ciliary Dyskinesia (PCD) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2023 To 2033

Primary Ciliary Dyskinesia (PCD) Market Outlook

Thelansis’s “Primary Ciliary Dyskinesia (PCD) Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2023 To 2033" covers disease overview, epidemiology, drug utilization, prescription share analysis, competitive landscape, clinical practice, regulatory landscape, patient share, market uptake, market forecast, and key market insights under the potential Primary Ciliary Dyskinesia treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).

Primary Ciliary Dyskinesia (PCD) Overview

Primary ciliary dyskinesia (PCD) is predominantly inherited as an autosomal recessive disorder, leading to recurrent and chronic upper and lower respiratory tract infections. In 40–50% of cases, PCD is associated with mirror-image organ arrangement and other forms of heterotaxy. Currently, pathogenic variants in 45 genes have been identified as causes of PCD. While PCD is typically inherited in an autosomal recessive manner, exceptions include FOXJ1-PCD (autosomal dominant), PIH1D3-PCD, and OFD1-PCD (X-linked). Notably, 20%-30% of individuals with well-characterized PCD lack identifiable pathogenic variants in any associated genes. The progression and severity of lung disease in PCD vary among individuals. More than 75% of full-term neonates with PCD experience "neonatal respiratory distress," necessitating supplemental oxygen for days to weeks despite term gestation. However, few are diagnosed with PCD at this age. Chronic airway infection becomes evident in early childhood, with most children presenting chronic year-round wet cough, sputum production, and chronic wheezing. Lung function tests typically indicate obstructive lung disease. Sputum cultures commonly reveal oropharyngeal flora, Haemophilus influenzae, Streptococcus pneumoniae, and Staphylococcus aureus starting in early childhood, followed by an increased prevalence of Pseudomonas aeruginosa (initially smooth, later mucoid). While rare in childhood, non-tuberculous mycobacterial infections occur in more than 10% of adults with PCD. The diagnosis of PCD requires the presence of characteristic clinical phenotypes and either specific ultrastructural ciliary defects identified by transmission electron microscopy or evidence of abnormal ciliary function. Unlike PCD, other disorders associated with chronic sinopulmonary disease and bronchiectasis, such as cystic fibrosis, immunodeficiency (e.g., immunoglobulin G (IgG) subclass deficiency), allergic rhinitis, gastroesophageal reflux disease, and Wegener's granulomatosis (upper- and lower-airway disease), are not associated with situs abnormalities. Currently, there are no specific therapies that can correct ciliary dysfunction in PCD. The treatments described are empiric and aimed at managing the consequences of dysfunctional cilia and sperm flagella. Limited evidence supports the use of specific therapeutic modalities for PCD. Symptomatic treatments include antibiotics to prevent infection and nasal irrigation. Surgical interventions, such as endoscopic sinus surgery, can improve sinus drainage in patients with recurrent infections. Pulmonary therapies, similar to those used in cystic fibrosis, are often employed to reduce pulmonary diseases.

Geography Covered:

North America- the United States and Canada

Europe- EU5 (Germany, France, Italy, Spain, and the United Kingdom)

Other countries- Japan & China

Study Period: 2023-2033

Current Clinical Practice and Treatment Algorithm

This section of the study covers country-specific current clinical practice, the standard of care, and significant limitations around addressing the unmet needs. Retrospective analysis and bench-marking of clinical study outcomes are presented in terms of Pre-treatment & post-treatment clinical and demographic patient characteristics. Essentially, this section will cover the evolution of the current competitive landscape and its impact on the future treatment paradigm.

KOL Insights:

KOLs across 8 MM markets from the center of Excellence/ Public/ Private hospitals participated in the study. Insights around current treatment landscape, epidemiology, clinical characteristics, future treatment paradigm, and Unmet needs

Market Forecast: Patient Based Forecast Model (MS. Excel Based Automated Dashboard)

- Data Inputs with sourcing

- Market Event and Product Event

- Country-specific Forecast Model

- Market uptake and patient share uptake

- Attribute Analysis

- Analog Analysis

- Disease burden and pricing scenario

- Summary and Insights

NPV/ IRR Calculator-

Optimization of cash flow/ revenue flow concerning all fixed and variable investments throughout the product development process. The rate of return on an investment is a critical indicator to ensure the profitability and break-even of the project.

Competitive Landscape:

The competitive landscape includes country-specific approved as well as pipeline therapies. Any asset/product-specific designation or review such as Orphan drug designation, Fast track, Priority Review, Breakthrough Therapy Designation, Rare Pediatric Disease Designation, and Accelerated Approval are tracked and supplemented with analyst commentary.

Clinical Trial Assessment-

Detailed clinical trial data analysis and critical product positioning include trial design, primary outcomes, secondary outcomes, dosing and schedules, inclusion and exclusion criteria, recruitment status and essentially covers the reported adverse events. Majorly the trial analysis helps determine the potential of the critical assets and their probable filing and launch date.

Unmet Medical Needs Overview-

This report presents the most important clinical unmet needs in the treatment, according to Thelansis research and analysis. Other essential unmet needs identified through our study include decreased cost burden on patients, improved administration convenience, and improved patient compliance.

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