A summary of our first policy report - A Year On
Les Halpin was diagnosed with Motor Neurone Disease in May 2011; he was shocked to discover that no new drug had been developed to treat MND since Riluzole was brought to market over 20 years ago. He therefore launched the campaign, Empower: Access to Medicine, dedicated to finding ways of accelerating approval and licensing of drugs for people who suffer from life-threatening conditions.
Following consultation with a number of key stakeholders, and with the backing of his local MP Geoffrey Clifton-Brown, Les created the Halpin Protocol, a proposed blueprint framework for a system of legal indemnities for patients with uncommon, life-threatening diseases.
The Halpin Protocol
1. Does the Patient suffer from a life-threatening illness to which there is currently no sufficient medical treatment?
2. Does the Patient give informed consent to the modification of his/her legal remedies against the Developer of the Drug and against those conducting the Clinical Trials?
3. Has the Developer undertaken that;
a. the Clinical Trials will be conducted in a properly controlled environment and under medical supervision; and
b. the results of the Clinical Trials will be published whatever the results.
The Protocol is now subject to consultation, led by Geoffrey Clifton Brown and Professor Sir Peter Lachmann, to examine obstacles that the Protocol may need to overcome.
As part of that consultation, key figures (listed below) from the pharmaceutical, medical, ethical, regulatory, legal, academic and insurance fields came together on 28th June 2013 to discuss the current barriers that hold back the development and prescription of medical drugs and agree the next steps of the consultation.
In particular they looked at the current legislative and regulatory barriers that impede pharmaceutical companies, especially smaller biotechs, from bringing their products to market.
As well as changes in the law, the attendees looked at possible alternative arrangements that could be brought forward using current law or other protections.
Pathways to Progress
The panel identified a variety of paths that can be explored that would benefit the medical profession, pharmaceuticals and patients more widely.
To a large extent, the solutions lie within existing capacity in current system, to exercise existing powers and raise awareness of what can already be achieved.
Clarifying the requirement of phase III trials for drug approval is a necessary step for medicines that affect less common conditions where there is an existing need. Phase III trials should be “a tool in the armoury” of licensors - randomised control trials are the ‘gold standard’ in research but are not a ‘one size fits all’ solution.
Clarifying the rights and privileges of doctors in prescribing drugs that have not been licenced would help overcome the cultural inertia holding back a practice once common in the profession.
Other answers may lie in companies sharing liability risk more broadly in the form of insurance mutuals. Currently pharmaceuticals and biotechs bear the liability risk of their products individually. This in practice means that only the large pharmaceutical companies can meet the cost of the insurance associated with their products.
Were the industry to pool its risk more broadly, it would drive down the cost of insurance and therefore be able to reinvest the savings into more research.
The Halpin Protocol presents an opportunity for patients with unmet clinical need to take control of their treatment in collaboration with their doctor.
However, there are a great many things that can be done now to drive this agenda forward for patients (adaptive licencing, flexibility with phase III trials, etc.). Implemented effectively and collectively, they would negate the requirement for the Halpin Protocol. Patients would have more control, pharmaceuticals would have more flexibility to get their drugs on the market (and therefore strengthen the product’s evidence base) and doctors would have more confidence in their prescribing decisions.
Professor Sir Peter Lachmann FRS FMedSci, Cambridge Immunology
Professor John Spencer QC, Cambridge University, Faculty of Law
Sir David Cooksey GBE Hon FMedSc, Chairman of the Francis Crick Institute
Dr Richard Barker OBE, Director, Centre for the Advancement of Sustainable Medical Innovation
Professor Sir Gordon Duff FRCP, FRCPE, FMedSci, Chairman, Medicines and Healthcare Products Regulatory Agency
Dr Dougal Goodman FREng, Chief Executive, The Foundation for Science and Technology
Baroness O’Neill of Bengarve
Becky Purvis, Head of Policy, Association of Medical Research Charities
Dr Jack Scannell, Head of Discovery Research, e-Therapeutics PLC
Professor John Harris FMedSci., FRSA., Professor of Bioethics, Manchester University
Dr Richard Smith, Director, Protein Therapeutics Laboratory, Kings College London
Lois Rogers, Freelance Journalist and campaigner
Ryan Hollingsworth, Legal Adviser, Association of the British Pharmaceutical Industry
Mark Bishop, Solicitor, C J Jones Solicitors LLP
James Hargrave, Empower, Access to Medicine