Patient perspectives: Les Halpin

Motor Neurone Disease (MND) patient Les Halpin speaks with pharmaphorum's Rebecca Aris on how we need to speed up the drug approval processes for diseases such as MND.

You may recall that a few months ago Les Halpin, Motor Neurone Diseases (MND) patient, was featured in the UK press calling for drug laws to be reformed to allow terminally ill patients to be able to try unlicensed drugs in return for giving up the right to sue pharmaceutical companies.

Les speaks with pharmaphorum here in our latest Patient Perspective interview to explain how he thinks this could work and offers advice on how to support his campaign. He also highlights why he believes that publicly sharing all trial data is critical.

Interview summary

RA: Les, thank you for agreeing to take part. Could you please start by sharing with us your background prior to your diagnosis?

LH: I qualified as an applied statistician from Exeter University in 1979 and since then have worked in business, including 23 years with Record Treasury Management, offering currency investment and hedging services to pension funds.

I joined Lightfoot Solutions, in 2007 which uses statistical analysis to improve organisational performance, becoming Chairman in January 2009. Today I am an active investor in a number of start-up companies, specialising in transformation performance, and a Fellow of the University of Exeter.

I met my wife Claire at Exeter University and over the years we have enjoyed shared and varied interests, including a lot of foreign travel, walking and skiing. Claire is passionate about conservation so many of our travels have been centred on this passion. We maintain close links with Exeter University where we have supported research into the plant diease rice blast through the Halpin PhD Scholarship Programme.

I was diagnosed with MND in May 2011, aged 54, after suffering some weakness and instability on my legs.

RA: Since your diagnosis with Motor Neurone disease you have committed yourself to accelerating the drug approval and licensing processes for life-threatening illnesses. What inspired you to do this and can you tell us more about your work in this area?

LH: When I was diagnosed I was staggered to discover that no new drug had been approved for Motor Neurone Disease for 20 years.

Currently, drug companies do not have sufficient financial incentive to invest in developing new drugs for rare or 'orphan' diseases due to the small number of the population who are affected as well as the high and uncertain costs of the drug development process.

MND is a complicated disease. It's more than likely that it will require more than one drug to treat. Clinical drug trials normally only test one drug at a time, and therefore it's quite possible that a drug that could be effective in conjunction with another, is rejected during clinical trials as not being of any use in treating MND.

Imagine a world where MND patients worldwide have access to drugs at this stage of testing – they are proven safe for humans, and possibly known to be efficacious in other neurological diseases, just not for MND. Patients are given the freedom to choose which drugs they think might help them; the process is monitored, and patients and doctors alike can report on their effects. Data is stored centrally, and thus can be analysed to determine the effects of individual drugs and of drug combinations. Ideally this requires some way of objectively measuring the progress of the disease – something which has not been possible in the case of MND in the past. However, huge strides have been made recently in determining biomarkers for MND. Biomarkers are also being developed or are available for other rare diseases that would benefit from this approach. Once a volume of data has been collected from thousands of patients worldwide, this can then be analysed and used to inform future research into these diseases, and influence investment from pharmaceutical companies.

This is my vision, and the reason I started the campaign. The first step to achieve this vision is to relax the rules on drug testing, which requires a change to the law in the UK. Therefore I have launched the Halpin Protocol, a blueprint calling for this and the ability for the patient to take responsibility for any liability, rather than the prescriber. I am working closely with politicians, the medical profession, pharmaceutical companies and patient groups and have been overwhelmed by the support I have received at this early stage of consultation.

RA: What do you think needs to change to speed up the drug approval processes for diseases such as MND?

LH: If pharmaceutical companies believe that the drugs they develop could 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.

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 drugs at an earlier stage of development as well as innovative, new combinations of drugs. We also want all clinical trials data to be shared publicly as this is the only way we are going to speed up the knowledge process.

RA: You recently, very publically, suggested that terminally ill patients should be allowed to take experimental drugs, how would you see this working and what needs to change to allow this to happen?

LH: A mechanism should be devised that provides a framework for patients suffering from certain life-threatening diseases to make their own informed choices about the risks of participation in Clinical Trials of unregistered drugs and therapies.

This will require the collaborative participation of patients, regulators, physicians and the developers of drugs and therapies.

Regulators will be required to approve a Schedule of Unmet Clinical Need where acceleration of drug and therapy innovation is particularly important, justifying the controlled relaxation of restrictions on participation in clinical trials of unregistered drugs and therapies for patients suffering from those life-threatening diseases.

Physicians will be required to certify that the Patient has an acute unmet clinical need and to supervise the administration of the drug or therapy during the Clinical Trials.

Patients will be required to give informed consent to their participation in the Clinical Trials and also to give informed consent to the required modification of their right to strict liability protection.

Developers will be required to undertake that the Clinical Trials will be conducted in a properly controlled environment, under medical supervision and that the results of the Clinical Trials will be fully published whatever the results.

This is the basis of the Halpin Protocol.

RA: What advice would you offer to other sufferers of MND?

LH: Work closely with your consultant and GP to explore any possible drugs that may help you under their close supervision. Don't be afraid to discuss this with them. Also, if you and your family. friends and colleagues support what I am trying to do, please contact your local MP and sign the petition. We have moved the debate on significantly in the past six months at a political and industry level but we need as much support from individuals and patient groups as possible so the patient voice is heard.

"Public sharing of all trial data is critical..."

RA: If you could tell the pharma industry one thing, what would it be?

LH: Public sharing of all trial data is critical, a 2010 study by the National Institute of Health Research estimates "half of all clinical trials have never been published and that trials with positive results were twice as likely to be published as those without." That cannot be in the patient's interest. When addressing a problem it is just as important to have a full understanding of what doesn't work as it is to have an understanding of what does.

Legislation already exists in this country compelling drug companies to publish the adverse effects of a drug but not, at this stage, the full results of trials. This needs to change.

It is only through a full understanding of the efficacy of a drug or treatment that a patient can make an informed decision about what they want. For those with life threatening illnesses the risk-ratio of "doing nothing" is significant. Therefore they, above all others, must be able to access all the information on a drug that they need - even if the risk of adverse effects or failure are great.

RA: Thank you for your time.

About the interviewee:

As a statistician, 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.

Les was born in London and brought up in Essex when his father moved to England after escaping from Burma during the Japanese invasion of 1942.

He attended the local grammar school and was the first member of his family to attend university. He graduated from Exeter University in 1976 with a first class degree in Mathematical Statistics and Operational Research, during which time he met his wife Claire who was studying Biological Sciences.

After gaining experience with Barclays Bank, British Gas and Lloyds Bank International, Les went on to spend 23 years with Record Treasury Management, offering currency investment and hedging services to pension funds.

When the company was floated on the stock exchange in 2007 he joined Lightfoot Solutions, which uses statistical analysis to improve organisational performance, becoming Chairman in January 2009.

He and his wife are passionate philanthropists. They have donated over one million pounds to the University of Exeter to create the Halpin PhD Scholarship Programme which provides opportunities to students from rice growing developing countries and funding research into 'rice blast', a plant disease that each year kills enough rice to feed 60 million people. Exeter researchers are now leading the way in understanding the molecular biology of the disease, helping develop effective and durable controls. He is also a significant donor towards Exeter Business School's new café and learning space and sits on the Business School's Advisory Board.

Since his diagnosis, Les has committed to a personal legacy of accelerating drug approval and licensing for people with life-threatening illnesses.

How can we ensure that all clinical trial data is shared to allow patients to make an informed decision?