Blue Matter Blog

Introducing the Blue Matter Breakfast Club Series
March 27, 2019

Recently, our Rare Diseases Team launched the Blue Matter Breakfast Club.  You might ask, “What is the Blue Matter Breakfast Club?”  Well, it’s an ongoing series of quarterly two-hour breakfast forums, hosted by our Rare Diseases Team.  These forums bring together leaders from companies that develop and commercialize rare disease therapies to discuss critical business …

A Framework for Successfully Launching & Commercializing Rare Disease Products
January 28, 2019

Regardless of the product, launching a new biopharmaceutical drug is a time-consuming, expensive, and complex undertaking.  It requires sound strategy, expert planning, and effective cross-functional coordination to get everything done right.  The analogy is certainly not new but launch really is akin to conducting a symphony orchestra, ensuring that all members play their parts correctly, …

The Disruptive Potential of Gene Therapy: When will it fully realize its commercial potential?
December 7, 2018

Gene therapy has made significant advances over the past few years.  However, those therapeutic advances have not been accompanied by spectacular commercial successes, at least not yet.  This raises a simple question: When will gene therapy fully realize its commercial potential? We explored that question with a distinguished panel on November 7, 2018 at the …

Demonstrating the Value of Rare Disease Treatments
July 25, 2018

Part V in a Series on Rare Diseases At various points in this series, we’ve mentioned that rare disease treatments are usually quite expensive.  In fact, the typical RD treatment costs between $300,000 and $750,000 (US) per year.1 Those high costs reflect two key realities:  1.) Developing new therapies is expensive and risky, and 2.) …

Organizing to Enable the “High-Touch” Rare Disease Business Model
June 27, 2018

Part IV in a Series on Rare Diseases In Part I of this series, we introduced the fact that rare disease (RD) patients are quite special.  The same is true for the specialists who treat them.  In RD markets, these dynamics create the need for patient-centric treatment approaches and a “high-touch” business model with key …

Rare Disease Centers of Excellence
June 13, 2018

Part III in a Series on Rare Diseases In parts I and II of this series, we provided an overview of the special commercial challenges associated with rare disease (RD) therapies, and we took a deeper dive into one of the most obvious:  Finding patients.   In this installment, we explore rare disease Centers of Excellence …

Finding Patients: A Key Challenge in Rare Diseases
May 30, 2018

Part 2 in a Series on Rare Diseases Part 1 of this series provided an overview of rare diseases and the special challenges they present to biopharmaceutical companies.  In this installment, we explore one of the most basic—and most confounding—of those challenges:  Finding patients. In the European Union, a disease is considered rare when it …

9th European Conference on Rare Diseases and Orphan Products
ECRD Vienna 2018 Recap
May 15, 2018

Recently (10-12 May), we traveled to Vienna to attend the 9th European Conference on Rare Diseases and Orphan Products (ECRD).  In this article, we describe some of the key takeaways from the conference, and the major implications for biopharmaceutical companies operating in this space. Our Focus on Rare Diseases More than 7,000 diseases are classified …

Rare diseases
Addressing the Special Commercial Challenges in Rare Diseases
March 29, 2018

Part 1 in a Series on Rare Diseases In the European Union, a disease is defined as rare when it affects fewer than five in 10,000 people.  In the United States, the definition is similar:  fewer than seven in 10,000 people.1 For the people affected by rare diseases—and the physicians who treat them—the official definition …

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