The global orphan drugs market presents many opportunities for new drug development...
The global orphan drugs market presents many opportunities for new drug development, and advances in drug discovery capabilities coupled with regulatory and financial incentives are helping to generate rich pipelines of breakthrough treatments with true disease modifying properties, according to new analysis from Frost & Sullivan’s Product and Pipeline Assessment of the Global Orphan Drugs Market.
While there are only 172 approved orphan therapies, over 6,800 orphan diseases exist, according to the U.S. National Institute of Health (NIH), the authors say. Pharmaceutical and biotechnology companies are now rolling out therapies for serious, rare diseases, going beyond palliative care and targeting the underlying pathology to slow down or stop disease progression.
The report identifies rare cancers as the orphan therapeutic area with the highest level of drug development activity. Other disease areas witnessing considerable drug development activity include blood/lymphatic system diseases, infectious/parasitic diseases, neurological diseases, metabolic diseases, and immunological/inflammatory diseases.
“In the past, pharmaceutical and biotechnology companies rarely developed new drugs to treat rare diseases due to the low return on investment realized because of the small patient population,” said Debbie Toscano, Senior Industry Analyst at Frost & Sullivan Life Sciences. “Now, drug discovery for orphan diseases is becoming an important element of the business models of numerous small and large pharmaceutical and biotechnology companies looking to strengthen their presence in the global market.”
As a result, companies are introducing orphan drugs that use diverse approaches such as small molecules, antisense, gene therapy, monoclonal antibodies, bi-specific antibodies, peptide therapies, and stem cell therapies. Currently, such therapies command premium prices due to the huge clinical benefits they offer and the lack of alternative treatments for patients. Soon, however, they will have to be sold at competitive prices as the existing level of reimbursement will become untenable due to the anticipated approval and commercialization of several orphan drugs for neglected diseases.
“As drug developers abandon the “blockbuster model” in favor of greater focus on drug development for rare conditions, the global orphan drugs market is becoming increasingly competitive,” noted Toscano. “It is imperative that drug developers continually keep a tab of competitors’ pipelines since approval and reimbursement of new orphan drugs are highly dependent on the availability of alternative therapies.”
Read the full release here.
Unifying Industry to Better Understand GCP Guidance
May 7th 2025In this episode of the Applied Clinical Trials Podcast, David Nickerson, head of clinical quality management at EMD Serono; and Arlene Lee, director of product management, data quality & risk management solutions at Medidata, discuss the newest ICH E6(R3) GCP guidelines as well as how TransCelerate and ACRO have partnered to help stakeholders better acclimate to these guidelines.
Putting Collective Insights Into Action to Advance Cancer Care: Key Examples From ASCO 2025
June 27th 2025At ASCO 2025, clinical operations leaders gained critical insights into how AI tools, bispecific antibodies, and evolving treatment paradigms are reshaping trial design, endpoint selection, and patient stratification.
Funding Cuts Threaten Diversity in Clinical Research
June 27th 2025In this video interview, Kyle McAllister, co-founder, CEO, Trially, discusses how recent federal funding cuts are likely to undermine research focused on underrepresented populations, and why long-term investment in community-based studies is essential to closing persistent health equity gaps.
Pfizer Reports Strong Phase III Results for Hympavzi in Hemophilia Patients with Inhibitors
June 26th 2025The Phase III BASIS trial found that once-weekly subcutaneous Hympavzi reduced treated bleed rates by 93% in patients with hemophilia A or B with inhibitors, offering a promising new prophylactic option for a population with limited therapeutic choices.