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
    improving patient experience
    6 Ways to Improve Patient Satisfaction Within Hospitals
    December 1, 2021
    degree for healthcare job
    What Are The Health Benefits Of Having A Degree?
    March 9, 2022
    custom software development is changing healthcare
    Digital Customer Journey Mapping and its Importance for Healthcare
    July 21, 2022
    Latest News
    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
    5 Steps to a Promising Career as a Healthcare Administrator
    August 3, 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
    Options For Seniors Who Outlive Their Life Insurance Policy
    February 16, 2021
    What are the Protections for Mentally Ill People Under the American with Disabilities Act?
    April 2, 2018
    5 Key Healthcare Technology Predictions For 2019
    September 28, 2020
    Latest News
    How Social Security Disability Shapes Access to Care and Everyday Health
    August 20, 2025
    How a DUI Lawyer Can Help When Your Future Health Feels Uncertain
    August 20, 2025
    How One Fall Can Lead to a Long Road of Medical Complications
    August 20, 2025
    How IT and Marketing Teams Can Collaborate to Protect Patient Trust
    July 17, 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 Identify and Treat Psoriasis
How Good is Screening Colonoscopy for Average-risk Adults?
How COVID-19 Impacted The Dental Industry As A Whole
Overlooked Factors That Affect Skin Health
The Triple Aim: Better Health, Better Care, Lower Cost

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

engineer fitting prosthetic arm
How Social Security Disability Shapes Access to Care and Everyday Health
Health care
August 20, 2025
a woman explaining the document
How a DUI Lawyer Can Help When Your Future Health Feels Uncertain
Public Health
August 20, 2025
physiotherapist at work
How One Fall Can Lead to a Long Road of Medical Complications
Health care
August 20, 2025
Common Healthcare Accreditation Programs
7 Most Common Healthcare Accreditation Programs: Which Should You Use?
Health News
August 20, 2025

You Might also Like

skin allergy causes
Allergy

What Are The Common Triggers Of Skin Allergy?

June 8, 2023

Occupy With Grace

November 24, 2011

Why Are There Drug Shortages?

December 3, 2011

America Spends More on Emergency Medicine Than Previously Thought. Good or Bad?

May 19, 2013
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?