By using this site, you agree to the Privacy Policy and Terms of Use.
Accept
Health Works CollectiveHealth Works CollectiveHealth Works Collective
  • Health
    • Mental Health
    Health
    Healthcare organizations are operating on slimmer profit margins than ever. One report in August showed that they are even lower than the beginning of the…
    Show More
    Top News
    mosquito misting spray to fight malaria
    Avoid Malaria with Mosquito Misting Systems
    June 12, 2023
    Medical Surveys
    Beyond the Clinic: Medical Surveys Are a Roadmap to Passive Income for Doctors
    September 23, 2023
    Glutathione
    What Are The Benefits of Glutathione?
    January 22, 2024
    Latest News
    6 Easy Healthcare Ways to Sit Less and Move More Every Day
    September 10, 2025
    7 Most Common Healthcare Accreditation Programs: Which Should You Use?
    August 20, 2025
    Hospital Pest Control and the Fight Against Superbugs
    August 20, 2025
    Hygiene Beyond The Clinic: Attention To Overlooked Non-Clinical Spaces
    August 13, 2025
  • Policy and Law
    • Global Healthcare
    • Medical Ethics
    Policy and Law
    Get the latest updates about Insurance policies and Laws in the Healthcare industry for different geographical locations.
    Show More
    Top News
    Tips for Older Travelers
    April 14, 2012
    Anti-RUC Suit Challenges Process for Setting Doc Pay Scales
    October 25, 2011
    Math Matters: Dosing Errors Can Be Deadly
    May 1, 2012
    Latest News
    Healthcare at a Crossroads: Why Leadership Matters More Than Ever
    September 9, 2025
    How Social Security Disability Shapes Access to Care and Everyday Health
    August 22, 2025
    How a DUI Lawyer Can Help When Your Future Health Feels Uncertain
    August 22, 2025
    How One Fall Can Lead to a Long Road of Medical Complications
    August 22, 2025
  • Medical Innovations
  • News
  • Wellness
  • Tech
Search
© 2023 HealthWorks Collective. All Rights Reserved.
Reading: The Orphan Drug Renaissance
Share
Notification Show More
Font ResizerAa
Health Works CollectiveHealth Works Collective
Font ResizerAa
Search
Follow US
  • About
  • Contact
  • Privacy
© 2023 HealthWorks Collective. All Rights Reserved.
Health Works Collective > Policy & Law > Global Healthcare > The Orphan Drug Renaissance
Global HealthcarePublic HealthSpecialties

The Orphan Drug Renaissance

Wendy White
Wendy White
Share
4 Min Read
orphan drugs
SHARE

orphan drugsA period of a renewal of life, vigor, interest is how renaissance is defined by Random House Dictionary. I think we are currently undergoing an orphan drug renaissance.

orphan drugsA period of a renewal of life, vigor, interest is how renaissance is defined by Random House Dictionary. I think we are currently undergoing an orphan drug renaissance. This renewed interest is displayed in the cover story of Chemical & Engineering News featuring my friend Melissa Hogan and her son Case. In addition to lovely photos of rare disease families, there are three well written articles by Lisa M. Jarvis that provide an overview of the orphan drug niche.

In Orphans Find A Home, Jarvis explains the changes that have made the tremendous growth of orphan drugs possible: The Orphan Drug Act of 1983, companies like Genzyme and BioMarin have shown orphan drugs are a sustainable business model and the mapping of the human genome. In the piece, Small Audience, Large Payoff, Jarvis describes the orphan drug pricing model and the interest of larger pharmaceutical companies in the space.

HIV activists with the power of social media
I like to compare the role that rare disease advocates play in awareness, funding, research and governmental policymaking to HIV activists. In the late 1980s and 1990s, HIV activists brought attention and funding to the disease and put pressure on the FDA to change. Their efforts made a tremendous difference. Similarly, today there are rare disease advocates who are working to impact individual rare diseases and the overall space. And they have the power of the internet and social media at their disposal!

More Read

How To Take Care Of Your Feet During Pregnancy
How Technology Shapes Dentistry’s Future
What You Should Know About Drug Interactions
Man Bites Police Dog Then Sues
Despite High EHR Adoption Rates, Physicians Want Stage 2 Delay

Jarvis illustrates the important role that rare disease advocates play in Patients Advocate for Change. She tells the story of Jill Wood whose son Jonah has Sanfilippo syndrome type C (MPS III). Wood recently founded a virtual biotech company to develop any drug candidates that might arise from their research.

This paragraph neatly sums up the reaction so many rare disease advocates get:

Whether it’s to a congressman or a reporter, Wood speaks about Jonah’s disease with urgency, rattling off scientific facts at a speed that can be disorienting for rare disease newbies. After she walks away from a group, there’s often a moment of stunned silence while people digest what they’ve heard, followed by a quiet comment: “Wow. She is amazing.”

The article also mentions the incredible accomplishments of Lori Sames and the Duchenne Muscular Dystrophy families. Sharing these type of inspiring stories was the impetus for Siren’s book, Uncommon Challenges; Shared Journeys. It’s available on Amazon with the profits going to the rare disease organizations mentioned in the book.

Significant growth
A recent report from EvaluatePharma predicts worldwide orphan drug sales to total $127 billion by 2018 with a compound annual growth rate of +7.4% per year between 2012 and 2018. The authors forecast that orphan drugs will be 15.9% of worldwide prescription sales by 2018 (excluding generics). In 1998 orphan drugs represented only 5.1% of worldwide prescription sales.

Do you agree that the rare disease space is experiencing a renaissance?

TAGGED:orphan drugs
Share This Article
Facebook Copy Link Print
Share

Stay Connected

1.5kFollowersLike
4.5kFollowersFollow
2.8kFollowersPin
136kSubscribersSubscribe

Latest News

a woman walking on the hallway
6 Easy Healthcare Ways to Sit Less and Move More Every Day
Health
September 9, 2025
Clinical Expertise
Healthcare at a Crossroads: Why Leadership Matters More Than Ever
Global Healthcare
September 9, 2025
travel nurse in north carolina
Balancing Speed and Scope: Choosing the Nursing Degree That Fits Your Goals
Nursing
September 1, 2025
intimacy
How to Keep Intimacy Comfortable as You Age
Relationship and Lifestyle Senior Care
September 1, 2025

You Might also Like

Ribs on BBQ
NewsPublic HealthWellness

Meat and Mortality: Does Eating Meat Decrease Your Lifespan?

March 10, 2013

XBOX Kinect Helps with Stroke Rehabilitation

November 28, 2012

Massachusetts Health Policy Commission Cost Trends Report

December 31, 2013
dental veneers benefits
Dental healthSpecialties

What Are the Benefits of Dental Veneers and How Do They Work?

December 8, 2022
Subscribe
Subscribe to our newsletter to get our newest articles instantly!
Follow US
© 2008-2025 HealthWorks Collective. All Rights Reserved.
  • About
  • Contact
  • Privacy
Welcome Back!

Sign in to your account

Username or Email Address
Password

Lost your password?