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Dr. Wolfram Lux is a Senior Director and Dr. Simone Seiter is a Partner in the global Pharma & Biotech practice within Simon-Kucher & Partners Life Sciences Division (Frankfurt, Germany).
Looking at four building blocks of success, Wolfram Lux and Simone Seiter investigate what it takes to win and be successful when commercializing an orphan drug
Orphan and ultra-orphan diseases are the new Promised Land for the biopharmaceutical industry. Although tiny in patient population compared to well-known diseases, the 7,000 rare diseases known today affect up to 30 million patients in the US, almost 10% of its population. An alarming 95% of these diseases have not yet a medication or therapy, and diagnosis takes five to seven years on average, as they are not top-of-mind for physicians.
However, today they have a voice. Countless patient advocacy groups have driven the attention of the industry, payers, and policy makers toward diseases so rare that sometimes not even 50 patients in a country are affected.
While there were only a few players addressing the medical needs of these diseases ten years ago, the field of small research-driven biotech companies has expanded to an entire new industry that has begun harvesting the fruits of their labor and paving the way for others. On that journey, they have resolved tremendous biologic, scientific, and technical challenges, while also learning that commercializing these drugs does not follow the usual principles of the established pharmaceutical industry.
What makes rare diseases different from specialty diseases?
The fact that there are only few patients in rare diseases means they are distinguished from specialty diseases in several ways:
These characteristics establish an entire new business model, set apart from the ethical pharma, generics, and OTC business. However, the rules of the game have not yet been fully laid out, and many things remain in flux while new ideas and approaches are tested along the product life-cycle. As a consequence, most biotechs in the orphan space have failed to meet their own expectations when looking at orphan launches between 2015 and 2019:
Three in four orphans launches missed launch goals so far, as stated by analysts. Established pharma only missed out on 55% of their launches. In the EU, almost 90% of orphan drugs continue to fail in year two after launch. In the US, still over 60% of orphan drugs continue to fail in year two after launch.
Whilst an entire new industry is still trying to find its ways around, Simon-Kucher has been looking at the findings so far and identified four prerequisites for commercial success.
Based on these four building blocks of success, we investigated what it specifically takes to win and be successful commercializing an orphan drug. We analyzed previous orphan launches and spoke with selected players to understand what made them succeed, identifying four major areas that made the difference in commercialization success:
Launch process planning for an orphan drug is different in four ways:
Many players apply what has worked successfully for the pharma industry over the years. Earlier in this article, we identified answering common question in more granularity as a key success factor. This is especially true when looking at the patient journey in orphan diseases. Here successfully customer engagement truly starts at the very beginning.
After having mastered all of the abovementioned challenges in pre-launch times and during set-up of your organization, now the market holds the fruits for you to harvest. BUT these fruits are not low-hanging. In fact they rather sit toward the top of the tree. Now it is about people … and the following elements have proven to make or break the deal for an orphan once you have made it to market:
PAGs have always been an integral part of customer engagement in the pharmaceutical industry. However, their role was long marginalized to rather being add-on support for general marketing and sales activities. Their role and benefit is a completely different story in orphan diseases, where many research and development initiatives have not only been influenced but also substantially initiated, driven, and funded by them.
PAGs play various roles along the drug development pathway and contribute significantly to value generation. Winning players have done a good job of living up to their expectations, right from the start of drug development:
PAGs in the course of go-to-market preparations are key stakeholders and should be embedded tightly in the commercial planning and clinical development process.
After evaluating the orphan disease and manufacturer scene, and analyzing the findings from the frontrunners in ideal and not so ideal conditions, we came to the conclusion that the orphan drug business constitutes a new business model, set apart from the ethical Rx-business in pharma, OTC, and generics.
Preparing a launch and commercializing an orphan drug has to be done differently, with launch excellence in this space built on a few specific areas. And the timelines for engagement and launch start much earlier than we know from ethical pharma, beginning as early as Phases I and II.
The question is, are you also prepared for it and are you prepared in a timely manner?
Dr. Wolfram Lux is a Senior Director and Dr. Simone Seiter is a Partner, both in the global Pharma & Biotech practice within Simon-Kucher & Partners Life Sciences Division (Frankfurt, Germany).