Rare diseases: Q&A with Martin Andrews, GSK

In Clinical Development by Freya Smale

Martin Andrews leads GSK’s efforts to advance the treatment of rare diseases. He was appointed Senior Vice President, GSK Rare Diseases in November 2013.

Prior to his appointment he was Senior Vice President, Global Vaccines Commercial. Martin joined the company in 1985 as a medical representative and worked through a succession of roles of increasing responsibility in marketing and sales, across multiple therapy areas, at local operating company, regional and global strategy levels.

What led you to head up GSK Rare Diseases?
I moved from leading a large organisation in vaccines where we deliver products to millions of healthy people, and in doing so have a global public health impact, to this role in rare diseases where we aim to bring medical solutions to very small numbers of patients who suffer from debilitating diseases. Whilst the nature and scale of the businesses are vastly different, what unifies them is the potential to make a visible and significant impact on peoples’ lives. The fact that less than 5% of known rare diseases have approved medicines is my main source of motivation. It is clear that patients suffering from rare diseases have a significant need and I want to do my part in helping to bring treatments to them. I also look forward to the challenge of creating a successful rare disease business by leveraging the best of our capabilities across the GSK organisation, which I know well from many years of working in different parts of GSK.

You’ve had some setbacks with your late-stage pipeline, what is your plan going forward?
Encountering disappointments is part and parcel of the R&D process for any disease area. The important thing is that we learn as much from what doesn’t go well as from what does go well to support future programmes. We currently have active research programmes across a range of rare diseases including amyloidosis, lysosomal storage disorders, primary immune deficiencies and pulmonary arterial hypertension. In addition, we have several rare disease programmes in early stages of development, and are also exploring whether some of our currently available and investigational medicines could have potential benefit for some rare disease indications.

What sets GSK Rare Diseases apart from other companies in the rare disease space?
There are several factors – firstly we have a highly productive base of scientific research across a broad range of disease areas and technical platforms through our research teams; secondly we have a hugely capable new product development and manufacturing capability; thirdly is our global reach; and lastly I believe we are a good partner for others who don’t have access to these resources and vice versa. We recognise that GSK needs to take a different approach to medicine development and access in rare diseases which is why we have created an integrated Rare Diseases Unit – a dedicated team that can access the resources GSK brings while learning how to operate in a more personalised way with the rare disease community through seeking their insights.

How will success be evaluated?
Patients and their families are at the heart of everything we do. Our success will ultimately be determined by whether or not we succeed in bringing valuable medicines to patients, and I believe this will happen. Our scientists have been working hard to further the understanding of the rare diseases that we work on and have been successful in publishing our research efforts in a number of top-tier journals including Nature, Science and Nature Genetics. We hope these findings will help the field to advance. Recently, as a result of our efforts in Duchenne muscular dystrophy, we gathered the largest clinical dataset related to this condition in over 300 boys, which we hope will go a long way to help the scientific community’s understanding of DMD.

What do you see as the biggest challenges for delivering medicines for rare disease?
We are often operating in uncharted territory where little or nothing has been published regarding the natural history of the diseases we are developing medicines for – this makes establishing meaningful endpoints to evaluate the impact of investigational medicines challenging. With over 7000 rare diseases, we also need to carefully select the diseases where we believe we can make the most difference. But the biggest challenge I suspect for anyone working in this area is to be ultra resilient – it is heart breaking when things don’t go as planned and you know what that means for the patient and their family who are anxiously waiting for a solution.

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