What emerging opportunities are fueling growth within the global orphan drug industry?
Approximately 5% of rare diseases (RDs) have obtained approval from the US Food and Drug Administration (FDA) for a drug, while up to 15% of RDs have at least one drug demonstrating potential in disease treatment or prevention.
The escalating number of unmet RD needs serves as a pivotal driver for research and development (R&D). The demand for innovative treatments for RDs with limited therapeutic options is pressing. Recent strides in precision medicine and informatics, including big data analytics, multi-omics, nanomedicine, gene-editing techniques, and next-generation diagnostics, have opened avenues for crafting specific and personalized therapies for RDs. The intersection of cancer and RDs is becoming increasingly apparent. Precision oncology and tailored medicine for rare tumors are emerging as key focal points within the field, fueling the expansion of the orphan drug (OD) industry.
Download a sample of this Growth Opportunity by filling out the form on the right.
Download Sample
Transformative Mega Trends
Sustained financial support to drive the development of rare disease (RD) therapies, as pharmaceutical firms, payers, and patients confront the challenge of high orphan drug (OD) prices.
Competitive Intensity
Leveraging rapid technological advancement, particularly in AI and data analytics, to optimize R&D processes and gain a competitive edge in discovering prospective treatment leads.
Disruptive Technologies
Integrating omics data and advanced technologies into drug discovery and development to enhance disease understanding and identify new therapy targets.
Sign up for a complimentary Growth Pipeline Dialog™
A Growth Pipeline Dialog is a structured discussion with our growth experts providing unparalleled technology intelligence and proven implementation best practices. This discussion will spark innovative thinking and help generate a pipeline of growth opportunities you can leverage to maximize your company’s future growth potential.
