Healthcare policy is a complex field – and sometimes it can be a scary one as well. Much more than in other policy areas policy makers as well as the industry are confronted with decisions that directly affect peoples’ lives and wellbeing.
The issue that currently keeps the entire world on its toes is of course the Ebola crisis. A fierce, life-threatening disease that spreads in West Africa – causing thousands of deaths and devastating fragile economies and health systems.
Despite the massive medical and political challenges of this crisis EU policy-makers and experts are currently making time to work on an other pressing – yet less well-known – health issues: the problem of rare and ultra-rare diseases.
From November 12th - 14th, experts gathered in Brussels for the “World Orphan Drug Congress Europe”, while the European Commission’s Expert Group on Rare Diseases met simultaneously to discuss political answers to this challenging topic. But what exactly are rare and ultra-rare diseases and why is this an issue that policy-makers are concerned with?
Those diseases affect a very small numbers of patients. A rare disease is one that affects fewer than 500 patients per million of population, a disease is generally considered to be ultra-rare if it affects fewer than 20 patients per million of population, but sometimes it affects even fewer patients. Even though the cases are rare, the impact of those often chronic and live-threatening illnesses on patients and their families is profound.
The problem with those diseases is that typically only a limited number of scientists or companies explore them and only few physicians are able to diagnose and treat them, for which reason it is very difficult for patients to get an effective therapy.
When developing so called “orphan drugs” to tackle these diseases, one of the biggest challenges are clinical trials – given the very small number of patients affected it is difficult to identify a sufficient number of participants. Less clinical trials result in less registered medicinal products. Moreover the return on investment for these products is low, and thus there is no real incentive for the industry to engage in this field. To date the European Commission has authorised 106 orphan medicines for the benefit of patients suffering from rare diseases. However, there are between 6,000-8,000 rare and ultra-rare diseases that have been identified so far.
The EU Regulation on orphan medicinal products states that patients suffering from rare conditions deserve the same quality, safety and efficacy in medicinal products as other patients and that they have not benefited from medical innovation to the same extent as patients with common diseases. Thus regulatory and economic incentives are required to ensure that patients suffering from rare conditions can benefit from advances in medical innovation. Furthermore, cooperation between the Member States, the pooling of information and expertise as well as funding for research - e.g. through programmes such as Horizon 2020 - are ways to tackle the barriers that these products face.
Not all of these challenges will be solved in this week’s high-level expert meetings, but all of these activities constitute important steps towards more effective treatment options and healthier, better and longer lives for people who suffer from those afflictions.