Regions
MAIN/REGIONS
regions/
Regions
  1. Americas
  2. Asia & Oceania
  3. Europe
  4. Middle East & Africa
  1. Solutions
  2. Products
  3. Insights
  4. About
  5. Careers
  6. Contact Us
MAIN/search
MAIN/Solutions
Solutions/
SOLUTIONS
  • Research & Development
  • Real World Evidence
  • Commercialization
  • Safety & Regulatory Compliance
  • Technologies
LIFE SCIENCE SEGMENTS
HEALTHCARE SEGMENTS
THERAPEUTIC AREAS

Impacting People's Lives

"We strive to help improve outcomes and create a healthier, more sustainable world for people everywhere.

LEARN MORE
Harness the power to transform clinical development

Reimagine clinical development by intelligently connecting data, technology, and analytics to optimize your trials. The result? Faster decision making and reduced risk so you can deliver life-changing therapies faster.

Research & Development Overview
Research & Development Quick Links
Real World Evidence. Real Confidence. Real Results.

Generate and disseminate evidence that answers crucial clinical, regulatory and commercial questions, enabling you to drive smarter decisions and meet your stakeholder needs with confidence.

REAL WORLD EVIDENCE OVERVIEW
Real World Evidence Quick Links
See markets more clearly. Opportunities more often.

Elevate commercial models with precision and speed using AI-driven analytics and technology that illuminate hidden insights in data.

COMMERCIALIZATION OVERVIEW
Commercialization Quick Links
Service driven. Tech-enabled. Integrated compliance.

Orchestrate your success across the complete compliance lifecycle with best-in-class services and solutions for safety, regulatory, quality and medical information.

COMPLIANCE OVERVIEW
Safety & Regulatory Compliance Quick Links
Intelligence that transforms life sciences end-to-end.

When your destination is a healthier world, making intelligent connections between data, technology, and services is your roadmap.

TECHNOLOGIES OVERVIEW
Technology Quick Links
MAIN/Products
Products/
CLINICAL PRODUCTS
COMMERCIAL PRODUCTS
COMPLIANCE, SAFETY, REG PRODUCTS
REAL WORLD PRODUCTS

CASE STUDY

"Data in a Day

LEARN MORE
MAIN/Insights
Insights/
BLOGS, WHITE PAPERS & CASE STUDIES

Explore our library of insights, thought leadership, and the latest topics & trends in healthcare.

DISCOVER INSIGHTS
THE IQVIA INSTITUTE

An in-depth exploration of the global healthcare ecosystem with timely research, insightful analysis, and scientific expertise.

SEE LATEST REPORTS

INSTITUTE REPORT

"Global Trends in R&D 2023

READ MORE

EMEA Insights

"IQVIA thought leadership with a focus on EMEA.

Learn more

WHITE PAPER

"DCTs Deliver Big ROI

LEARN MORE

WHITE PAPER

"Capturing value at scale: The $4 billion RWE imperative

LEARN MORE
MAIN/About
About/
FEATURED INNOVATIONS
  • Connected Intelligence
  • AI & Machine Learning
  • Human Data Science Cloud
  • IQVIA Innovation Hub
  • Decentralized Trials
  • Genomics
WHO WE ARE
NEWS & RESOURCES

INVESTOR RELATIONS

"Visit our investor relations site for more information.

LEARN MORE
Unlock your potential to drive healthcare forward

By making intelligent connections between your needs, our capabilities, and the healthcare ecosystem, we can help you be more agile, accelerate results, and improve patient outcomes.

LEARN MORE
AI & Machine Learning with a healthcare IQ.

Artificial intelligence is accelerating opportunities. Realizing the benefits for healthcare requires the right expertise. Together, we can solve the problems of today, and drive the breakthroughs of tomorrow.

LEARN MORE
Your healthcare data deserves more than just a cloud.

The IQVIA Human Data Science Cloud is our unique capability designed to enable healthcare-grade analytics, tools, and data management solutions to deliver fit-for-purpose global data at scale.

LEARN MORE
Innovations make an impact when bold ideas meet powerful partnerships

The IQVIA Innovation Hub connects start-ups with the extensive IQVIA network of assets, resources, clients, and partners. Together, we can help lead the future of healthcare with the extensive IQVIA network of assets, resources, clients, and partners.

LEARN MORE
Proven, faster DCT solutions

IQVIA Decentralized Trials deliver purpose-built clinical services and technologies that engage the right patients wherever they are. Our hybrid and fully virtual solutions have been used more than any others.

LEARN MORE
Unlock the power of genomic research.

IQVIA enables genomic research via global access to a network of genomic-clinical data, proprietary technologies that enable federated analytics, and therapeutic area & bioinformatics expertise to help you answer your most pressing research questions.

LEARN MORE
MAIN/Careers
Careers/
WORKING AT IQVIA

Our mission is to accelerate innovation for a healthier world. Together, we can solve customer challenges and improve patient lives.

LEARN MORE
LIFE AT IQVIA

Careers, culture and everything in between. Find out what’s going on right here, right now.

LEARN MORE

WE’RE HIRING

"Improving human health requires brave thinkers who are willing to explore new ideas and build on successes. Unleash your potential with us.

SEARCH JOBS
MAIN/Contact Us
Contact Us/
Blog
Orphan Drugs: A Spotlight on Rare Diseases
Vipresha Jain, Analyst, IQVIA Pipeline Intelligence
Shweta Parihar, Associate Consultant, IQVIA Pipeline Intelligence
Jan 13, 2020
  • Blogs
  • Orphan Drugs: A Spotlight on Rare Diseases

With new legislative initiatives from regulatory bodies in a number of healthcare markets, coupled with a high-unmet medical need, pharmaceutical companies are continuing to invest in the development of orphan drugs. This article provides an analysis of key 2019 orphan drug approvals, using IQVIA Pipeline Intelligence data.

Introduction

Rare diseases, for which orphan drugs are indicated to treat, are characterized by a low frequency of occurrence in the general population of a geographical region. There are over 8000 described rare diseases, of which only 340 conditions currently have treatment options1. Some of these diseases, such as progeria, occur in 1 in every 4 million births, while others may be comparatively more common, such as multiple sclerosis, cystic fibrosis and Duchenne muscular dystrophy. The prevalence of these diseases varies between populations, making an indication rare for one region but common for another. The effects of these rare diseases range from premature mortality to severe disability due to a lack of efficacious treatment options. Growing understanding of numerous aspects of these diseases, along with improving government policies, are expanding the framework for research, development and market penetration through the introduction of novel and renovated therapies.

2019 Orphan Drug Approval Overview

The improvement in government policies over the years has been mirrored by an increase in the development activities for candidates addressing rare diseases; with less than 10 approved rare disease therapies on the market in the 1970s increasing to more than 830 US FDA approved orphan therapies as of 20192. New treatment options have been brought to market through the launch of either new molecular entities or through the renovation of older, established therapies.

As shown in Figure 1, the US FDA granted approval to more than 65 therapies2 in various rare disease therapeutic areas with the most approvals seen in cancer. Among them, hematological cancers have seen the maximum approvals, with 14 therapies for a range of indications including; multiple myeloma, myelofibrosis, acute lymphoid leukemia and various forms of lymphomas approved in 2019. This is followed by 11 registered therapies for solid tumors, including small cell lung cancer, non-small cell lung cancer, ovarian cancer, hepatocellular cancer, esophageal cancer, neuroendocrine tumor, melanoma and NTRK fusion-positive tumors. Furthermore, 9 therapies were approved for infectious diseases, including 2 therapies for tuberculosis, 3 for hepatitis C virus infection, 2 for human immunodeficiency virus infection and a therapy for fascioliasis. With 8 therapy approvals in the USA for disorders of the central nervous system, including Lambert-Eaton myasthenic syndrome, neuromyelitis optica, spinal muscular atrophy, amyotrophic lateral sclerosis and narcolepsy, the needs of another major therapeutic area have to some extent, been catered to. Additionally, 2 biologic therapies have been approved for Churg-Strauss Syndrome, 1 for graft versus host disease and 1 for systemic sclerosis in the field of autoimmune diseases.

Conclusion

With increasing focus from governments and regulatory bodies, coupled with a high-unmet medical need, pharmaceutical companies will continue to invest in the development of orphan drugs. In particular, ‘Big Pharma’ will continue to explore this field, through its own organic development and through the acquisition of small start-ups as well as established players. Additionally, the recent launch of a one-time adeno-associated virus mediated human RPE65 gene therapy for Leber congenital amaurosis, clearly establishes the will of big pharma to explore the orphan drug space, despite the financial and research headwinds.

References

  1. Ekins, S. Industrializing rare disease therapy discovery and development. Nature Biotechnology 2017; 35(2), 117–118.
  2. US FDA Orphan Drug list access portal. Available at: http://bit.ly/34xkdDg
  3. Melnikova, I. Rare diseases and orphan drugs. Nature Reviews Drug Discovery, 2012;11(4), 267–268.

To find out more about IQVIA Pipeline Intelligence is, please visit
https://www.iqvia.com/solutions/commercialization/brand-strategy-and-management/product-pipeline 

Related solutions

Rare Diseases

Build on our experience of more than 245 rare disease studies in 96 countries to fulfill your promise of hope to millions around the world.

Therapeutic & Specialty Expertise

Specialized expertise and customized solutions across 14 therapeutic centers of excellence, including oncology, GI/NASH, pediatrics, neurology and rare diseases.

Contact Us