Rare diseases have been an increasing area of focus as three waves have converged in recent years: genomic research and innovations becoming mainstream; the forthcoming regulatory policies and financial incentives put in place by the federal agencies; and the increasingly patient-centric and coordinated efforts of patient advocacy groups, caregivers, and centers of excellence (COEs).

This research service provides an overview of the orphan drugs market dynamics, estimating the market size broken down by different segments. It also dives deeper into the different technological, geographical, and therapeutic opportunities with an objective assessment of the disclosed pipelines. Notably, the study has an expanded coverage of not only rare diseases, but also hard-to-cure and “commercially non-viable” diseases.
The orphan drugs market has been characterized by severe unmet needs with therapies available for less than 8% of the identified diseases, creating an abundant opportunity for established and new companies alike to not only enter the market but also establish absolute dominance. However, this will require companies to navigate through R&D, clinical trial, small-volume manufacturing, and reimbursement/pricing challenges as there is a measurable departure from traditional pharma rules.

This study captures the competitive landscape and the different strategies employed by companies to stay ahead of the curve. It also identifies game-changing companies leading innovation from the front. Acknowledging that the market continues to be dominated by larger pharma companies, the study identifies opportunities for mid and small segment companies to tap this market. The study thus elaborates on opportunities in drug repurposing, personalized medicine, hybrid clinical trials, and untapped therapeutic areas.

Furthermore, this research service lays down strategic imperatives for prioritizing markets for new product launch, working with the right communities which act as conduits to the right patient populations, and the right set of technologies to be leveraged to accelerate innovation. In conjunction with the core manufacturers, the research service also provides in-depth analysis of other ecosystem stakeholders such as logistic service providers, contract service providers, diagnostic companies, investors, and advocacy groups.

Information is also given on some of the leading M&A activities impacting the market as well as unconventional collaboration agreements laying the foundation for propelling innovations toward licensure and delivery. Furthermore, present and future market trends like increasing price transparency and drug affordability, which would shape the market, are discussed.

Key Issues Addressed
  • What is the total market size and what is the projected growth for the global orphan drugs market? Which are the largest growth segments to be pursued?
  • How are the various regions around the globe positioned to monetize the market opportunities? What are the key regional barriers to entry?
  • What are the new risk mitigation strategies and alternative big bets to circumvent R&D challenges? Which are the lucrative partnership models for the long term?
  • What are the market-defining macro trends mandating the need to deploy new digital solutions suite and which cross sections of the value chain will benefit the most?
  • How are product offerings in this market likely to emerge? What is the rate of innovation adoption and how is it solving the unmet needs of customers?
  • Which are the key game-changing companies in the market and how are they riding the growth curve?