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Improving access to medicines: empowering patients in the quest to improve treatment for rare lethal diseases
  1. Les Halpin1,
  2. Julian Savulescu2,
  3. Kevin Talbot3,
  4. Martin Turner3,
  5. Paul Talman4
  1. 1Lightfoot Solutions Group Limited, Bracknell, Berkshire, UK
  2. 2Oxford Centre for Neuroethics, University of Oxford, Oxford, UK
  3. 3Nuffield Department of Clinical Neurosciences, University of Oxford, John Radcliffe Hospital, Oxford, UK
  4. 4Neurosciences Department, Barwon Health, The Geelong Hospital, Geelong, Victoria, Australia
  1. Correspondence to Professor Paul Talman, Neurosciences Department, Barwon Health, The Geelong Hospital, Ryrie Street Geelong, Victoria 3220, Australia; pault{at}

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A patient's perspective

In 2011 Les Halpin was diagnosed with motor neuron disease (MND), also known as amyotrophic lateral sclerosis (ALS). Since his diagnosis, Les has been committed to accelerating the development, approval and licensing of novel therapies for people with life threatening illnesses. Two years ago he also founded Halpin Neurosciences, a privately owned biotechnology company, to focus on accelerating the development of therapies for MND. In 2012 Les formed a campaigning organisation, ‘Access to Medicine’, a platform to open up a wider debate about the lack of drug development for people with life threatening illnesses.

A very good friend of mine died of motor neuron disease (MND) 20 years ago, as did a non-familial uncle 15 years ago. They received no therapies which could slow the disease progress, and from my perspective not a lot has changed since that time. This disease has affected me in a profound way, leaving me unable to walk and to use my limbs for everyday activities, affecting my speech, swallowing and breathing and robbing me of independence. The single licensed drug for MND, riluzole, is having no measurable impact on the progression of my disease nor on my quality of life.

The current drug approval process is cumbersome—it can take years, and requires extremely large and risky investment, to take a drug from the development stage through to Phase III and beyond. As an example, no new drug has been approved for MND since riluzole, almost 20 years ago. The drug regulatory regime, therefore, must change in order to help people with life threatening and rare illnesses. For such individuals, the ‘risk-return ratio’ is different compared with patients with more benign conditions and drug regulations should be adapted to allow such people the opportunity to try out new combinations of drugs. Currently drug companies do not …

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  • Funding The Wellcome Trust.

  • Competing interests None.

  • Provenance and peer review Not commissioned; internally peer reviewed.

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