Rare Diseases Market

Rare Diseases: The Pharmaceutical Landscape and Opportunity Areas

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Rare Diseases Market Overview

The service oriented and dynamic nature of the healthcare industry has led to the discovery of a plethora of treatment options for a wide range of ailments.However, there are certain diseases, termed as rare, that continue to remain untreated and neglected. Rare / orphan diseases, as the name suggests, are those diseases which are relatively infrequent in occurrence. Many orphan diseases are life-threatening and progressive in nature. Absence of potential treatment regimes catering to these diseases is a big unmet need in the market.

The prevalence and adversity of these ailments has gained significant attention in the global healthcare industry. Stakeholders have realized the necessity and importance of urgently addressing the widening concerns and the need for discovering treatment options for several rare diseases. There is a tremendous scope offered by the innovation driven research being executed by various organizations in an effort to bridge gaps in the current market landscape.

The enactment of the Orphan Drugs Act in the US and EU emerged as ground breaking and provided the necessary support to guide research focused on rare diseases worldwide.The orphan designated drugs are granted important incentives which include market exclusivity and fees reductions. This regulatory assistance has provided a strong impetus to the pharmaceutical companies and led to their active participation in the market. Collaborative agreements and partnerships, amongst the pharmaceutical firms and other active organizations (such as EURORDIS, NORD and Global Genes), are also playing a key role in addressing the needs of this market.

The wide gap between the demand and supply of drugs is likely to provide a major boost to the market in the coming decade. Several drugs for various rare diseases are currently under development and, once approved, are likely to be readily accepted by the untreated patient population.

Recent Developments in Gene Therapy Market:

Several recent developments have taken place in the field of gene therapy. We have outlined some of these recent initiatives below. These developments, even if they took place post the release of our market report, substantiate the overall market trends that have been outlined in our analysis.

  • In August 2023, Rett syndrome received fast track designation from the FDA for TSHA-102 used for the treatment of patients suffering from Rett syndrome.  
  • In August 2023, Xcell Biosciences entered into a collaboration with Labcorp to develop and validate cell and gene therapies.  
  • In May 2023, Ray Therapeutics closes USD 100 million in a Series A funding round with an aim to advance optogenetic gene therapies.

Scope of the Report

The ‘Rare Diseases: The Pharmaceutical Landscape and Opportunity Areas’ report is a comprehensive study of the market of drugs being developed by the top pharmaceutical companies for rare diseases. The focus of this study is primarily to uncover several initiatives currently underway to harness the potential presented in different therapeutic areas. One of the basic building blocks for analysing the market was to prepare an extensive pipeline of drugs for rare diseases. This effort encompassed reviewing several databases, company websites and other information available in the public domain.

We confined the focus of our study to 50 large pharmaceutical companies which invest a proportion of their R&D spend on drugs for rare diseases. These pharmaceutical companies were profiled to get an in-depth view of the work being done by them in this domain. The profiles include a general overview of the firms, financial performance over the last five to six years, and the current marketed and clinical pipeline products specific to rare diseases.

With a robust pipeline in place, we then analysed where the industry’s current vision lies and highlighted a number of efforts which have been taken by pharmaceutical companies, healthcare organizations and other industry stakeholders. Specifically, our market potential analysis outlines key patient demographics and underscores potential areas anticipated to emerge as major revenue generators for the pharmaceutical companies. In addition to the above, we have also provided our own independent perspective on the key drivers behind the growth of this market, and the associated challenges. 


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