Martin Andrews

Rare diseases: Q&A with Martin Andrews

Martin Andrews leads our Rare Diseases Unit to help advance the treatment of patients affected by rare diseases. He was appointed Senior Vice President, GSK Rare Diseases in November 2013.

Prior to his appointment Martin was Senior Vice President, Global Vaccines Commercial. He 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 business and therapy areas, in general management, regional and global strategy roles.

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 licensed 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 growing our rare disease business by drawing upon the best of our capabilities across the GSK organisation, which I know well from many years of working in different parts of GSK.

What are your current areas of research?

As one of our six key business areas focussed on delivering an innovative and sustainable presence, our Rare Diseases business currently has active research programmes across a range of rare diseases including amyloidosis, lysosomal storage disorders, primary immune deficiencies and pulmonary arterial hypertension. These frequently involve leading edge technology platforms such as gene silencing or gene therapy.

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 makes GSK Rare Diseases unique in its ability to deliver solutions 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, compared with other therapy areas, 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.  Our scientists have been working hard to further our 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.

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 6,000 – 8,000 rare diseases, we also need to carefully select the diseases where we believe we can make the most difference.

6,000 - 8,000

With 6,000 - 8,000 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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