Nordic Life Science 1
SUSTAINING LAUNCH SUCCESS AS ORPHAN MEDICINES COM
E OF AGE IN 2018, BOTH THE AMERICAN FDA and the European Medicines Agency approved a greater number of new medicines for rare diseases than for mainstream conditions for the first time. The dream of making more medicines available for patients with very rare diseases, started decades ago with the US Orphan Drugs Act and the EU Orphan Regulation, has succeeded. Although there remains huge untreated and unmet need, 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. However, for the pharmaceutical companies behind them, not just bringing orphan medicines to approval, but launching orphan medicines excellently will become even more challenging and important over the next 5 years. 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. Huge advances in therapies are enormously promising, but even though the total sales of individual Orphan Medicines are mostly modest, payer concern on costs has risen, and with it the number of negative market access outcomes. Orphan medicines companies must not only get an optimal label, they must successfully address market access in a more challenging environment, with the right data, stakeholder relationships, insight and flexibility. Orphan Medicines companies face substantial challenges of patient identification and recruitment for clinical trials, and support on the path to effective treatment, sometimes even when a product is available and budget is agreed. Patients suffering from rare diseases are likely to be geographically spread, and in many cases may have experienced mis-diagnosis and lack of awareness of their symptoms which means that they can spend an average of 4.8 years presenting symptoms before an accurate diagnosis is made. Clinical trials for rare disease patients can be hampered by slow recruitment due to slow diagnosis, or due to the inability of patients to reach scarce treatment centres. More effective use of Human Data Science to understand and identify patients using real world, genomic and other information, will underpin the next step in Orphan Medicine evolution. TO READ THE full whitepaper on this topic and to learn more about IQVIA and our presence in the Nordics, visit www.iqvia.com. NLS FACTS IQVIA IQVIA (NYSE:IQV) is a leading global provider of advanced analytics, technology solutions and contract research services to the life sciences industry dedicated to delivering actionable insights. IQVIA is a leading authority on Launch Excellence, publishing a white paper on global Launch Excellence every two years since 2007, and establishing many key concepts used to manage pharmaceutical launches, including the importance of the six-month window. IQVIA has also published widely on specific launch issues, including launch and multichannel, medical affairs, emerging markets and orphan medicines.