Bardet-Biedl Syndrome (BBS) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2021 To 2032

Bardet-Biedl Syndrome (BBS) Market Outlook

Thelansis’s “Bardet-Biedl Syndrome (BBS) Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2021 To 2032" 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 Bardet-Biedl Syndrome (BBS) treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).

Bardet-Biedl Syndrome (BBS) Overview

Bardet-Biedl Syndrome (BBS) is a rare autosomal recessive defect belonging to the family of ciliopathies. Children born from consanguineous marriages are mostly affected. The primary manifestations of BBS are rod-cone dystrophy (90 to 93%), postaxial polydactyly (62-82%), obesity (73-91%), learning disabilities (62%), genital abnormalities (59-98%), and renal abnormalities both morphological and functional (53%). The visual prognosis for children with BBS is poor, with a mean age of legal blindness of 15 years. Significant weight gain begins within the first year and becomes a lifelong issue for most individuals. Most individuals have significant learning difficulties, but only a minority have severe impairment on IQ testing. Renal disease is a major cause of morbidity and mortality. Clinical findings establish the diagnosis of BBS. Multiple genes are known to be associated with BBS: BBS1, BBS2, ARL6 (BBS3), BBS4, BBS5, MKKS (BBS6), BBS7, TTC8 (BBS8), BBS9, BBS10, TRIM32 (BBS11), BBS12, MKS1 (BBS13), CEP290 (BBS14), WDPCP (BBS15) SDCCAG8 (BBS16) LZTFL1 (BBS17), and BBTP1 (BBS18). BBS is typically inherited autosomal recessive, but up to 15% of patients do not have identifiable mutations in known genes. Both interfamilial and intrafamilial phenotypic variability exists. Carrier testing and prenatal testing are possible if the disease-causing mutations in a family are known.

• Bardet-Biedl Syndrome (BBS) incidence rates vary from country to country; in North America is found to be 1 to 2 cases per 150,000, and in Europe, it is 1 to 1.5 cases per 150,000 live births.

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: 2021-2032

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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