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

Rare Disease Day 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.


Rare Disease: Where Precision Medicine Was Born

As the mother of a child with a rare disorder, I’ve watched with interest and admiration as this community has…

Tags: , ,


5 Ways to Speed Diagnosis of Our Children

You have probably heard the statistics before. There are 7,000 rare diseases. More than 80% are genetic. Half of those…

Tags: , , , , ,

Guidelines for Movement Marketing

Movement marketing is a great way to empower patients by rallying them around a cause and motivating them to take…

Tags: , , ,

Rare Disease Infographic

Rare Disease Day 2013: Help Spread Awareness

Each year Rare Disease Day is celebrated worldwide on the last day of February. This year is even more special…


The Power of Rare Disease Patients

Thanks to the Pew Internet Project, we have a lot of data about ePatients. These empowered, engaged and educated patients…


Innovation Happens at the Margins

PharmaPhorum asked Siren Interactive to write a series of blog posts about the world of rare diseases. In this six-part…


Unique Celebration of Rare Disease Day

The video below is a great example of how biotech and pharmaceutical companies are supporting Rare Disease Day. The Dutch…


Beth Burnside, Nina Prybula and Tanika Craig raise their hands to support rare diseases

All Hands On Deck for Rare Disease Day

Waking up at 4:00 AM to go stand outside in the cold isn’t usually my idea of fun, but for…


Study from Pew Internet Project Supports Rare Disease Insights

The Pew Internet Project and the California HealthCare Foundation today release a new report, “Peer-to-peer Healthcare.” It includes insights from…


bumpy road

The Journey to Diagnosis Is Often Bumpy

This post is written by a colleague who also works in the rare disease space. She wishes to remain anonymous…


Show Your Strength. Share Your Story.
Create a Rare Disease Video

As part of Rare Disease Day 2011, the National Organization for Rare Disorders (NORD) is inviting patients and family members…


Raise Your Hand to Help Millions of Americans Affected by Rare Diseases

For the first two years of her life my daughter, Casey, had medical issues but no diagnosis of what was…


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: ,

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