In the second of a series of eBooks, we will be examining the rare disease research and orphan products being developed for Multiple Myeloma, a cancer of a type of white blood cell called a plasma cell, normally responsible for producing antibodies. In multiple myeloma, collections of abnormal plasma cells accumulate in the bone marrow, where they interfere with the production of normal blood cells.
Unlike other rare diseases that have received tremendous attention from orphan drug developers, several medicines are already authorised for the treatment of multiple myeloma in Europe.
Even with existing treatments for patients, research continues to push the boundaries when it comes to efficacy. That being said, at the time of market authorisation, sponsors will have to provide evidence that their treatments provides a significantly larger benefit and that it works in a different way to existing treatments. It may end up being used in combination with other medicines to improve the outcome of patients with multiple myeloma.
Some open ended thoughts:
Should the powers that be incentivise researchers to explore treatments for more neglected rare diseases?
Are existing treatments, if approved, deemed good enough?
How can the orphan drug industry maintain this momentum and sustain long term research for all rare diseases?