A blog exploring pharmaceutical relationship marketing, emarketing and innovation with a focus on rare disorders.
The convergence of rare disease, digital communications, and pharmaceutical marketing communications

NORD Archive

Delivering Value in Rare Diseases

Posted by | 10:56am on Monday, February 29, 2016 | No Comments
Hidden Value of Orphan Drugs

Debates about high drug prices have dominated many of the headlines this year.  Raise the price of a drug 5,000% and you will definitely get everyone’s attention, including the feds.

It’s unfortunate to see the occasional bad actors getting so much press, given the inspiring progress made by all of the companies and communities diligently working to find treatments, especially when you consider those with rare diseases. Thirty million people in America are impacted by rare disease and 95% of those disorders lack treatment. Don’t let the headlines mislead you. The real news is that continued investment in rare disease innovation helps everyone.

We hope you’ll share our 4th annual Rare Disease Infographic that focuses on these innovations:

  • Record levels of investment leading to record levels of FDA approvals
  • NIH and advocacy groups funding early research
  • Research on orphan drugs that opens the door to new treatments for common ailments
  • New distribution models that reduce costs through direct patient engagement and personal support
  • Foundations and compliance experts working to accelerate the approval process by improving research and public policy

As the number of orphan drug designations and approvals rises, there will likely be more questions raised about the cost.  The customary response is that research is more challenging in rare diseases or that knowledge gained from rare informs the common, both of which are true.

But as the emphasis on reducing costs in healthcare grows, perhaps the best response orphan drug makers can make is to demonstrate value. That isn’t just about costs.  It’s about patient experience, quality of care and maximizing outcomes.

Understanding how best to accomplish this requires a paradigm shift. Delivering value in orphan drugs requires a fundamentally different strategy, because rare patients and families face a whole host of financial, medical and lifestyle challenges that require a sophisticated system of support.

In the end, it will be the patients themselves that teach us what good looks like in rare disease. Whether it’s a matter of determining meaningful endpoints for clinical trials or designing a program to increase adherence, the best way to deliver value is to listen to them.

NJ RARE 2013 Jane Castello Julie Raskin Francois Nader Debbie Hart Mary Cobb

Rare Disease and the Power of Partnership

One of the issues raised by the Affordable Care Act is the importance of decisions made at the state level…

Tags: , ,


Get Ready for Healthcare Reform

During a recent phone call arranged by the National Organization for Rare Disorders (NORD) to help members prepare for healthcare…

Tags: , ,

Celebrating 30 Years of NORD & The Orphan Drug Act

I had the privilege of attending the National Organization for Rare Disorders (NORD) Gala on Tuesday evening. I say “privilege”…


Groundbreaking Legislation for Rare Disease Patients: FDA Safety & Innovation Act

The Food and Drug Administration Safety and Innovation Act, signed into law on July 9, 2012, includes important measures for…


A Day of Dialogue with NORD, FDA, NIH

On May 17, I attended the National Organization for Rare Disorders (NORD) Corporate Council meeting in Washington DC. This was…


photo of hand with zebra print

The Power of Patient Stories

The New York Times recently published an interactive feature with photos and audio of rare disease patients telling their stories….


NORD Helps Improve the Lives of Rare Disease Patients

In a win for rare disease patients and families, the U.S. House of Representatives yesterday passed the Improving Access to…



Many Rare Disease Patients Unsure of Treatments

The National Organization for Rare Disorders (NORD) and Inspire recently surveyed members of their online rare disease community. The data…

Tags: ,

Alone we are rare.
Together we are strong.
Rare Disease Day 2/28/10

Imagine that your health is failing or that your child is very ill, but the doctor can’t figure out what’s…

Tags: ,


NORD Urges Action on Healthcare Reform

Jason Ross, Project Manager at Siren Interactive, contributes this post: Many Americans — especially those with rare disorders — are…

Tags: ,

The Need For A Rare Disorder Patient Registry

I went to the first National Organization for Rare Disorders (NORD) Summit a few weeks ago in DC and learned…


Rare Disease Day

Raise Awareness of the Reality of Rare Disorders

This is a cross post from “e-patients.net“. One in ten Americans is living with a rare disorder, but they are…


Siren Interactive
  • Siren Interactive
  • Rare Disease Relationship Marketing Experts
  • 626 West Jackson Blvd, Suite 100
  • Chicago, IL 60661
  • 312.204.6700
  • 866.502.6714 (Toll Free)
  • www.sireninteractive.com