Blue Matter Blog

Success in Rare Diseases, Part II: Optimizing Clinical Trial Design for the Rare Disease Ecosystem
September 24, 2020

In Part 1 of our series we introduced the rare disease (RD) ecosystem, which is defined by a small patient population with often heartbreakingly high unmet need. Here, in Part 2 of our series, we will discuss in more detail the overarching design requirements and key considerations for RD clinical trials. The entrance of a …

Success in Rare Diseases, Part I: Understanding the Rare Disease Ecosystem
August 31, 2020

It’s a safe bet that anyone reading this article is familiar with the concept of an ecosystem. When we think of an ecosystem, we most often imagine how one exists in nature: a complex web of living organisms and non-living components (such as water, air, soil, rocks, and so on), all interacting with one another …

Blue Matter “Breakfast Club” Meeting #5: Practical Implications of the COVID-19 Crisis for Biopharma
July 28, 2020

The Blue Matter Breakfast Club is an ongoing series of discussion forums (now virtual due to COVID-19), hosted by our team in Europe.  These forums are by invitation, bringing together leading executives from biopharma companies and other organizations to discuss critical business issues. The fifth Breakfast Club meeting took place on 8th July 2020.  The …

The Rarest of Them All: Why Neglected Rare Diseases Require a Very Different Mindset
April 1, 2020

Introduction In previous articles, we explored several important aspects of rare diseases (RDs).  First, we showed how different authorities define the term “rare disease” (or “orphan disease”).  We also established a spectrum of rarity, ranging from those diseases that are considered common—with well-established treatment paradigms—to those that are “hyper-orphan,” affecting perhaps a couple of people …

How Rare Is Rare? Making Sense of Rare Disease Definitions, Nomenclature, and Patient Numbers
March 20, 2020

Many people working in and around the health care system and the biopharmaceutical industry use the terms “rare disease” and “orphan disease” on a fairly regular basis.  Obviously, they refer to any disease that’s uncommon, often with few treatment options (or no treatment options).  But what do these terms really mean? What’s the actual definition …

How are Rare Diseases Different from Rare Oncology and Specialty Diseases and What are the Implications for Companies?
February 26, 2020

Rare vs. Specialty Diseases: Are They the Same? We’ve often been asked what the differences are between rare diseases and “specialty diseases” and/or rare cancers.  After all, these three types of disease share some similar characteristics.  For example, they’re often complex, require higher-cost and “higher touch” treatments, and a high degree of knowledge and specialization.  …

Blue Matter Breakfast Club Meeting #3 – A Discussion with Theresa Heggie of Alnylam Pharmaceuticals
November 8, 2019

The Blue Matter Breakfast Club is 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 issues. The third Breakfast Club meeting was on 5th November 2019 at Skylounge restaurant in Zug, Switzerland.  …

Blue Matter Breakfast Club 2, Nick Leschly, CEO of bluebird bio
Blue Matter Breakfast Club Meeting #2 – A Discussion with Nick Leschly, CEO of bluebird bio
July 1, 2019

The Blue Matter Breakfast Club is 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 issues. The second Breakfast Club meeting was on 17th June 2019 at Skylounge restaurant in Zug, Switzerland.  The …

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 …