By Sarah Rickwood
The 2020s are the age of orphan medicines.
Orphan medicines are available for a larger number of diseases and patients than ever before, a testament to the success of legislation established decades ago encouraging the development of these medicines in both the U.S. and European Union.
Orphan medicines were the majority of European Medicines Agency (EMA) approvals for the first time ever in 2016 (59%) and the majority of U.S. Food and Drug Administration (FDA) approvals for the first time ever in 2018 (58%).
Although orphans have not always been the majority in subsequent years, the share of approvals with an orphan designation has been generally high, and was once again the majority in 2020. This new cohort of orphan medicines included the leading edge of innovation in personalized medicine — the cell therapies Kymriah and Yescarta, and the gene therapies Luxturna and Zolgensma — treatments that were not only cutting-edge, but also commercially successful.
At the start of the 2020s, as the types of pharmaceutical innovations to launch became much more specialist, the companies behind these launches also evolved. While the top 20 companies remained formidable launch machines, the specialized nature of much innovation coming to market, including orphan medicines, means that smaller companies with the right medicine, can, and do, launch highly successfully. Orphan medicines continue to be a part of the commercial prescription medicine market where emerging biopharmaceutical companies can make their mark.
The pandemic environment of 2020 was, of course, hugely challenging for all aspects of health care delivery. For the introduction of innovative new medicines, this was especially the case. While innovative medicines got approved and entered the markets of major countries in 2020 at levels that were at or better than historic levels, the opportunity for doctors to see patients face-to-face, overwhelmingly the preferred setting for new diagnoses and prescription of new medicines, went down considerably. Where patient journeys to diagnosis and treatment are long and complex, as they are for many rare diseases, this impact was even higher.
However, one clear contrast in 2020 has been between the success of introduction of orphan and non-orphan medicines into major markets. Orphan launches proved much more resilient to 2020’s adverse launch environment and are over-represented in 2020’s commercial successes. According to the IQVIA MIDAS audit data, orphan medicines launched in 2020 outperformed historic norms of the first six months of orphan medicines sales in the U.S., U.K., Germany, France, Spain, and China. They under-performed previous cohorts in Japan and Italy. The orphans driving outperformance varied by country, including Tepezza and Trikafta/Kaftrio in the U.S., Zolgensma in Germany, Yescarta in Spain and Kymriah in France. The strongest launch of all (in the U.S.), Tepezza, is the first pharmacotherapeutic ever for thyroid eye disease, a rare but debilitating condition.
It is reassuring that patients with debilitating rare diseases still managed to get access to innovative therapeutics during the pandemic, and that many health systems were strong enough to be able to continue to prioritize orphan medicines.
However, for many rare diseases, new medicines enter the market with a significant group of patients identified and waiting for the approval of the treatment — and for 2020 orphan medicines — that “pipeline” of patients built in 2019 and 2018, pre-pandemic. And it remains to be seen whether the patient pipeline was built as effectively in 2020 and 2021 for the orphan medicines that are yet to be launched (i.e., in 2021, 2022, and 2023).
Sarah Rickwood is Vice President, EMEA Marketing and Thought Leadership at IQVIA.