The European Commission just published a documento de trabajo assessing the EU’s orphan and pediatric drug strategies. Read here why incentives for research are key to extending patients’ lives:
A rare disease is a medical condition that meets the criteria defined in Article 3 of Regulation (EC) No 141/2000; a life-threatening or chronically debilitating condition affecting no more than 5 in 10,000 persons in the EU. Although so-called rare diseases affect a limited number of people per disease, collectively they affect one person in every 17 people within Europe. There are over 7,000 different rare diseases patients suffer from.
Regulators see an ‘imbalance of risk and reward’ for the industry to find cures and treatments for those diseases. Hence US, Japanese and EU regulators increased options for longer market exclusivity for drugs tackling diseases in children and rare diseases. In 2000, Regulation (EC) No 141/2000 and 2006 Regulation (EC) No 1901/2006 were adopted by the European Commission. The ‘standard’ incentives provided by the general legislative framework for pharmaceuticals in the EU are 10 years of market protection and 20 years of patent protection. For pediatric and orphan drugs manufacturers can apply for extended market exclusivity.
The purpose of this strategy is to improve and expedite patients’ access to safe and affordable medicines and to support innovation in the EU pharmaceutical industry. Adding prolonged exclusivity worked: A massive increase in orphan drugs could be seen in the last 20 years! Between 2012 and 2017 over three times as many orphan drugs entered the EU compared to 2000-2005. The EU Commission estimated that between 200,000 and 440,000 additional quality-adjusted life years were gained thanks to more incentives for these drugs.
Voices who call now for less protection of orphan and pediatric drugs want to undo the successes of the last two decades. The 142 orphan medicines authorized between 2000 and 2017 have helped up to 6.3 million patients in the EU to either cure or cope with their health conditions.
But there are still millions of patients waiting for a breakthrough that can help to treat their rare or pediatric disease – For this, we need to have incentives and not populism. Intellectual property is key in allowing the inventor and her investors to reward them for their massive risk they undertook in trying to find a cure or treatment for a rare disease. The EU’s approach to orphan and pediatric drugs by increasing incentives for inventors and manufacturers has worked. The successes of the past 20 years should not be undermined by populist calls to nationalize research and IP. If we care about patients with rare diseases, we should not question the importance of protecting intellectual property but see it as a precondition for future innovations.
To sum it up: Think of the children and allow medical innovation to take place!