Blue Matter Blog
June 7, 2021
We gratefully acknowledge the contributions of Alyssa Manz, Ph.D. and Quinn Civik for their work on the data analysis which supports this publication. Each rare disease (RD) only impacts a relatively small population, and given the clinical breadth of these conditions, new RD product development may feel like forging a completely new path towards a …
May 3, 2021
Some of our Rare Disease team spent a day last week listening to patient experts and industry specialists in a series of virtual panels and fireside chats organized by the World Orphan Drug Congress. Amongst a wealth of interesting topics and a consistent focus on accelerating the delivery of rare disease (RD) therapies, a few …
March 1, 2021
Throughout this series, we’ve covered a wide range of areas that biopharma companies must address if they are to succeed in the rare disease (RD) ecosystem including clinical trial design, clinical trial implementation, and ongoing evidence generation. In this installment, we cover the important topic of market access. To help drive commercial success, a therapy …
December 8, 2020
In the first three parts of our series, we introduced the rare disease (RD) ecosystem and its interconnected stakeholders. We also discussed the challenges and key considerations for RD clinical trial design – both (pre)registrational as well as ongoing evidence generation after the first approval. Here, we examine the practical considerations of successful clinical trial …
October 26, 2020
In Parts 1 and 2 of our series, we introduced the rare disease (RD) ecosystem as a uniquely interrelated set of stakeholders and discussed the key challenges and considerations for successful RD clinical trials. We focused on (pre)registrational clinical trial design, but of course regulatory approval does not mark the end of evidence generation. Here …
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 …
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 …
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 …
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 …
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 …
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. …
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. …
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 …
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 …
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, …
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 …
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.) …
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 …
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 …
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 …
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 …
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 …