Drug development: the researchers driving an open-science approach

As the era of personalised medicine and medical genetics continues to ramp up, it brings with it ambitions for more precisely-targeted therapies in a host of indications, and new hope for the treatment of rare diseases and genetic disorders.

But, opening the floodgates of therapeutic possibility comes at a cost. With clinical research and development (R&D) expenses estimated in the billions of dollars for an average drug that makes it to market, targeting treatments at increasingly small patient groups – whether they suffer from rare diseases or versions of more common conditions with distinctive genetic characteristics – raises hard questions for public health systems and private payers with strained budgets.

Drug prices are at the top of the pharma agenda, whether in US Congress or in the board rooms of the world’s largest drugmakers. For rare conditions, the outlook is even more tangled – if Big Pharma is spending billions on developing drugs for a relatively small patient population, how else to recoup investment other than through sky-high pricing? Even for a relatively familiar orphan condition like cystic fibrosis, there is a seemingly unsolvable tension around the pricing of innovative treatments, as a recent UK parliamentary hearing on the price of Vertex’s Orkambi demonstrates.