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
  • Patient Engagement
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
Advance your patient engagement strategy

Discover new patient engagement possibilities with IQVIA insights, expertise, and technology-driven solutions.

Patient Engagement Overview
Patient Engagement 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 2024

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
  • IQVIA Connected Intelligence™
  • IQVIA Healthcare-grade AI™
  • Human Data Science Cloud
  • IQVIA Innovation Hub
  • Decentralized Trials
  • Patient Experience powered by Apple
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
IQVIA AI is Healthcare-grade AI

Building on a rich history of developing AI for healthcare, IQVIA AI connects the right data, technology, and expertise to address the unique needs of healthcare. It's what we call Healthcare-grade AI.

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
IQVIA Patient Experience Solutions powered by Apple

Empowering patients to personalize their healthcare and connecting them to caregivers has the potential to change the care delivery paradigm. IQVIA and Apple are collaborating to bring this exciting future of personalized care directly to devices patients already have and use.

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/
WHITE PAPER
Orphan Drugs: An Update on Key Selected Pipeline Developments for Rare Diseases
IQVIA Pipeline Link
Jul 17, 2023
To download files, please accept Functional Cookies. These cookies provide enhanced functionality for a better user experience.

A rare disease is defined as a condition that affects less than 200,000 people in the USA and fewer than 1 in 2,000 people in the EU. The development of orphan drugs was previously rare due to the small size of patient populations, which acted as a barrier for commercial development in the research and development of rare diseases. Regulatory bodies in major markets have introduced new legislation with the aim of providing incentives for the development of medicines for rare diseases, including the US FDA's Orphan Drug Act in 1983, followed by Japan in 1993 and the EU in 2000. These incentives, including extended market exclusivity (7 years in USA and 10 years in EU), and the advancement in new technologies have driven the growth in ground-breaking research against rare diseases and the development of orphan drugs.

The Orphan Drug Act has resulted in a greater number of orphan drugs approved in the USA since its enactment, driven by the approval of rare oncology products and secondary indications. As of March 2023, there have been 6,506 orphan drug designations granted by the US FDA of which 1,144 have led to orphan-designated approval from the agency. This review provides an insight into key selected significant therapeutic developments in rare diseases with data sourced from IQVIA Pipeline Link, a proprietary drug pipeline database. 

Key selected orphan drugs in development

Rare disease drug development has recently focused on hematology, neurology, infectious disease, metabolism/endocrinology, rheumatology/immunology, cardiovascular, gastroenterology, pulmonary and ophthalmology therapeutic categories. There are currently several key orphan drugs in development that may emerge as the next rare disease therapies.

To read more, please fill in your details on the left to download the article.

You may also be interested in

Article
Therapeutic Breakthroughs Drive Dealmaking in Endocrine and Metabolic Disorders
Article
An Update on Treatment Options and Pipeline Developments for Type 1 and 2 Diabetes
Fact Sheet
Access granular patient numbers to truly understand market dynamics in your market
Fact Sheet
Access 10-Year Sales Value and Volume Forecasts
Article
How Do Differences in Cancer Types Lead to Differences in Cancer Treatments?
Blog
Orphan Drugs: A Spotlight on Rare Diseases
Prev
Next

Related solutions

Product Pipeline

Access 470,000 products and nearly 40,000 drug profiles to analyze successful launch strategies and be ready for what’s next.

Commercialization

Take advantage of the latest tools, techniques, and deep healthcare expertise to create scalable resources, precision insights, and actionable ideas.

Contact Us