Orphan Medicines Launch Excellence
White Paper
Mar 21, 2019

Sustaining launch success as Orphan Medicines come of age

Launching Orphan Medicines excellently will become even more important over the next 5 years. More patients with rare diseases have pharmacotherapies available, and there are a growing number of disease-focussed registries, increasing public and policy maker awareness, and significant R&D investment in pharmacotherapies and in digital technologies to support trials and treatment. A new frontier of challenge faces Orphan Medicines companies as the gap between Orphan Medicines and mainstream specialty products narrows. To understand how to succeed in launching an Orphan Medicine in the coming years, companies must learn from past launches and apply an Orphan Medicines-focused Launch Excellence framework for success.

Since 2007, IQVIA has identified the world’s most commercially successful launches and researched the success factors behind their Launch Excellence in a series of influential white papers. These papers focused on the key developed markets of the US, top 5 Europe and Japan, but used as their starting point all innovative branded launches. In a twenty-year period (1995–2015) of launches, the nature of the launches coming to market, and the ones which are most successful, has changed dramatically. The first Launch Excellence study, covering launches from 1995 to 2003, saw (by the IQVIA measures of Launch Excellence) 28 excellent launches, of which 82% were launches entering primary care markets. By the fifth Launch Excellence study, covering launches from 2011–2015, 87% of the 31 launches fulfilling our Excellence criteria were in specialty care.

When Orphan Medicine legislation was first introduced in the US in 1983, the mainstream pharmaceutical industry was ruled by primary care blockbusters – the world’s top selling launch was the H2 receptor antagonist Tagamet (cimetidine). Even when European Orphan Medicine legislation was enacted in 2000, the innovative pharmaceutical market was still about primary care blockbusters for conditions with billions to hundreds of millions of patients – the top selling product in the world that year was the proton pump inhibitor Losec (omeprazole). Since then, mainstream pharmaceutical innovation has changed, dramatically fast – the top two products by global sales (2018) are now Humira (adalimumab), a monoclonal antibody for autoimmune conditions, such as rheumatoid arthritis with a global patient population estimated at 80m, and Revlimid (lenalidomide), for multiple myeloma, myelodysplastic syndrome and mantle cell lymphoma (with global incidences each below 1m patients annually).

This IQVIA Orphan Medicine Launch Excellence whitepaper covers the changing launch excellence paradigm, which will evolve as innovations for rare diseases develop, drawing on case studies from across the lifecycle of Orphan Medicines. The environment for Orphan Medicines is moving into a new, more competitive stage, and this will make it more challenging for companies seeking to optimise their Orphan Medicine launch. We outline the key areas of focus for Orphan Medicines companies to address this environment and prepare for an excellent launch.


Orphan Medicine legislation has expanded in the 36 years since pioneer legislation was introduced in the US. In addition to the major step of EU legislation in 2000, there are now initiatives, ranging from patientand clinician-lead to formal legislation, for designating medicines as Orphans and defining rare diseases in Canada, Argentina, Peru, Colombia, Brazil, Japan, Singapore, Taiwan, South Korea, China, Australia, New Zealand, Russia, Ukraine, and Kazakhstan.

It remains the case that only an estimated 5% of rare diseases have an approved drug treatment. There’s still unmet medical need and a major opportunity to commercialise innovative medicines, although not all the remaining 95% of untreated diseases may be druggable.

Pharmaceutical manufacturers are faced with rising drug development costs. Relative to the cost of drug development for traditional medicines, Orphan Medicine development costs ~30% of a non-Orphan Medicine3, and historically Orphan Medicine development have been more certain in terms of getting an Orphan designated product to approval, although getting Orphan designation is tough. Falling returns on investment for innovative medicines globally makes Orphan Medicines an attractive prospect for companies looking to launch an innovative molecule in areas of high unmet need, one which is increasingly aligned to the overall specialty direction of the majority of the world’s largest pharmaceutical companies’ portfolio.

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