Orphan drugs are a hot topic, and there is huge potential in the fact that there are 5 – 8,000 rare diseases and only around 350 drugs that have been approved in the US, with less than 100 in Europe. The incentives put in place to stimulate the industry to develop drugs for rare diseases also play a key role.
However, the rare disease field has, for a long time, been characterised by patient focus, partnership and passion, which means that the rare disease community sees the situation from the patient's perspective. There is a huge unmet medical need in orphan diseases – about 95% of rare diseases lack treatment; 80% of the diseases are of genetic origin, but 40% of new drugs are in oncology.
There are several stakeholders within rare diseases – patients, physicians, networks, governments and regulatory agencies – and a commitment to collaborate. Patients are highly engaged and empowered and take an active role in both research activities and HTA (health technology assessment) activities.
There is also a focus from the US Congress and the European Commission on stimulating and funding research for rare diseases. A consortium has recently been established for international collaboration on research for rare diseases – the International Rare Disease Research Consortium (IRDiRC). The objective is not only to facilitate research for patients with rare diseases; they are also considered as models for more common diseases. This means that a breakthrough for a rare disease might have implications in developing drugs for more common diseases.
There are challenges on the journey from proof of concept to patient access to a new drug. There are challenges in diagnosing patients and designing clinical trials (few patients, heterogeneous diseases and lack of optimal end point). And when the drug is approved by regulators, the next hurdle is reimbursement. Small clinical trials are not able to provide convincing data on cost effectiveness and are therefore not always reimbursed, so new models are now being discussed for assuring patient access to new treatments.
Getting new drugs to patients more quickly and overcoming all of the associated hurdles remains the focus of the orphan drugs arena. The 3 Day MBA in Orphan Drugs will equip you with ways in which to do so, as well as getting you up-to-date on the latest developments and teaching you ways in which to collaborate and interact with key industry stakeholders. Don't miss out – register now!