Inside the rare disease centre which aims to accelerate biotech growth

Emmes has launched a rare disease centre, Orphan Reach, to combine its health research across the public sector and biopharmaceutical industry. DDW’s Megan Thomas spoke with Thomas Ogorka, Managing Director of Orphan Reach: Emmes’ Rare Disease Center, about its potential. MT: What will the expanded Orphan Reach centre add to what Emmes already offers? TO: First and foremost, specialised rare disease expertise allowing us to partner and guide even the smallest of biotechs through what are, very often, more complicated trial design protocols and regulatory pathways. It also means that we have a core team globally and in both the United States and Europe, which gives us extra flexibility when working with innovators and greater access to a wider pool of potential sites and patients. MT: Why is specialised clinical trial design and execution a game-changer? TO: If you look at the drugs pipeline now and particular from innovators trying to develop therapies for more than 7000 rare diseases, you see that these are increasingly coming from small innovators. This coupled with the fact that, by definition, we are almost always talking about diseases with no existing treatment options and therefore no regulatory precedence in the trials. When you work with a large CRO, and perhaps more traditional larger patient cohort trial designs, there is a certain amount of learning that can be applied from previous trials, from existing approvals and applied out of the box. The major challenge in other CROs is that there is a temptation to try and work the trial around what they have done before – orphan and rare disease trials rarely work like that. What you need, if you want to the innovator to have the best possible chance of success, is design a trial specifically for this therapy and disease as every rare disease trial is unique. MT: It is expected that the Orphan Reach clinical study portfolio will double over the next 12 months. What is the company’s plan to facilitate this expansion? TO: We are already building the team out quickly in both Europe and the USA and bringing in specialist expertise from across the wider Emmes Group. Over the next year, we will grow the team’s resources alongside its trial roster, and we are already looking to engage specialist scientists and operations professionals from both our US and European offices to introduce complimentary services and expanded capabilities as we move on with our mission. MT: What opportunities does this expansion offer Emmes? TO: We expect rare disease trials will be a very fast-growing area of Emmes, not only because of the number of new therapies in discovery, but also because there is a clear lack of specialised CRO services that can work more closely with innovators. However, there is also the opportunity for the new centre to cover a wider spread of rare disease indications by building on the strength of Emmes’ experience – for example, in ophthalmology and immunology. MT: What obstacles currently stand in the way of accelerated development of new treatment options for rare disease? TO: It’s increasingly common that rare disease-focused biotechs in the United States and Europe find it difficult to access appropriate CRO expertise, especially partners that can work with them to design bespoke protocols, trials strategies and anticipate any regulatory nuances that come with rarer disease trials. What we find is that a lot of the biotechs Orphan Reach works with have small, but experienced in-house teams, and they are therefore looking for a like-minded CRO. A partner that can be nimble and adapt designs and strategies quickly so that trials can advance more quickly through what are, nearly always, accelerated pathways. Rare disease trials, by definition, will require more complexity in regulatory submissions and trial design and, added to this, the CRO obviously has to develop a plan to find, enroll and manage patients in these trials. Therefore, not only is the strength of therapeutic experiences important, but also the relationships they have with trial sites and patient advocacy groups.