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Patients with rare diseases call for radical change in drug development

Patients with rare diseases call for radical change in drug development

Patients with rare, life threatening genetic conditions should be given greater powers to take part in clinical trials and have earlier access to medicines, according to a new campaign launched in the UK.

Empower: Access to Medicine is calling for a review of the law and ethics around drug development for these “orphan” conditions as it takes many years, and billions of pounds, to take a new drug to market.

The campaign has been founded by statistician and serial entrepreneur Les Halpin, who was diagnosed in 2011, and its aim is to put the patient voice front and centre in the wide-ranging discussions around the issue.

Empower: Access to Medicine has been formed to bring together like minded initiatives and campaign for:

  • A relaxation of the regulations to allow patients with life threatening illnesses easier access to drugs, which may have already been approved for other conditions
  • Changes to the testing regime to enable drugs to be to be developed more quickly for terminal illnesses
  • A comprehensive review of treatments for all terminal, life-threatening and seriously life-encroaching conditions
  • The  UK Government to take an international lead in calling for medical research & development (R&D) to extend into minority conditions that are terminal, life-threatening and seriously life-encroaching, by innovating in the use of the latest technology and cost-effective software approaches

Already some of the most senior experts in the field and patient groups have announced their support, including Professor Sir Peter Lachmann FRS FMedSci, President of the UK Academy of Medical Sciences (1998-2002), Richard Barker, director of the Oxford Centre for Accelerating Medical Innovation and Alastair Kent, Director General of Genetic Alliance UK.

Following his diagnosis Les discovered that the only drug available for his illness was the same one that a friend who had died of MND 20 years ago was prescribed.

He said; “No new drug has been approved for Motor Neurone Disease since Riluzole was approved 20 years ago.

“For those of us with life threatening or rare illnesses, the ‘risk-return’ ratio is different and drug regulations should be adapted to allow such people to try out new combinations of drugs.”

He believes drug companies do not have enough financial incentive to invest in developing new drugs for rare or ‘orphan’ diseases due to the small number of the population who are affected and the extremely long timescales as well as the high and uncertain costs of the drug development process.

He added: “Technological change – and the social networks it supports – is leading a revolution in how patients can approach their healthcare. Patients have opportunities to educate themselves and share their knowledge with similarly affected individuals, without the need for professional medical approval.”

Said Barker: “What is different about this campaign is that it is by patients, for patients. This is a voice that has not been sufficiently heard before now and I believe it can make significant impact on how Government, regulators and the pharmaceutical companies view drug development in this area.

“If pharmaceutical companies – especially SMEs – believed that the drugs they develop could reach patients earlier and be applied to a wider number of conditions, and that the testing regime would be simpler, this would help spur significantly more investment in new drugs. The initial focus of Access to Medicine should perhaps be on drugs that have already been approved for other conditions to be made more widely available for patients with life threatening illnesses.”   

Added Kent: “The ability of patients to be able to share information about their condition is key. Given how long it might take to find the right combination of drugs, patients and their doctors across the world should be sharing their experiences in an effort to improve knowledge and accelerate the timescales within which new drugs are developed and approved.”

Please visit the website for more information and join the conversation on Twitter @empoweratm.

For more press information, contact Sarah Rice or Karen White on 0117 9073400,

Notes to Editors

Les Halpin has been an active investor in a number of start-up companies in the financial and high-tech areas, and holds an honorary doctorate from the University of Exeter.

He is also Chairman of Halpin Neurosciences, a privately owned biotech which, since his diagnosis with Motor Neurone Disease last year, now focuses on accelerating the development of Motor Neurone therapeutics. Les was born in London and brought up in Essex.


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