Science and Technology CommitteeWritten evidence submitted by Empower

1. Introduction

1.1 Empower is a new platform to open the debate around the lack of drug development for patients with rare or life-threatening conditions.

1.2 Online social networks are helping to revolutionise the way people across the world can share information about their condition with similarly affected individuals, often without the involvement of medical professionals.

1.3 Given how long it can take to find the right combination of drugs to treat life-threatening conditions, Empower is keen to harness this collaboration to ensure that patients and doctors across the world share their experiences in an effort to improve knowledge and accelerate the timescales within which new drugs are developed and approved.

1.4 With this approach in mind Empower has reached out to patients with serious and life threatening conditions and other stakeholders for input and views on a submission to the inquiry. The campaign was lucky enough to have been a central feature of a Westminster Hall debate in the House of Commons (23/01/13) on access to medicine for people with terminal illnesses.

1.5 Empower welcomes the opportunity to make a submission to the Committee’s Inquiry and is particularly encouraged by the Committee’s recognition of the urgency of the issue.

1.6 Below is the Empower response to the Inquiry; instead of addressing the terms of reference point by point we have outlined our views on the broad issues and have included a copy of the recently launched (15/04/13) Halpin Protocol (Appendix 1).

1.7 On 15 April Empower started a formal consultation on “The Halpin Protocol”, a blueprint framework for patients suffering from serious and life-threatening diseases.

1.8 The protocol sets out principles that would allow patients to make their own informed choices about the risks of participation in Clinical Trials or taking drugs and therapies originally intended for other conditions.

1.9 It is the latest stage of the campaign Empower: Access to Medicine which was founded to put the patient voice at the centre of discussions about the law and ethics around drug development for life threatening conditions where there is insufficient treatment.

2. Further Comments

2.1 Empower welcomes the NHS’s aspiration to put patients at the heart of everything it does.

2.2 Recent proposals to amend several rights and pledges in the NHS Constitution, to reflect more clearly that the NHS supports individuals to manage their own health and involve them, is hugely important.

2.3 At the core of the Empower campaign is the drive to give patients more say in their treatment, the use of their data and the medicines they are prescribed. Therefore a proposed new pledge to involve patients in care planning discussions and to offer a written record of their care plan is a step that Empower wholeheartedly supports.

2.4 Particularly relevant in this area is the Constitution right “to be given information about your proposed treatment in advance, including any significant risks and any alternative treatments which may be available, and the risks involved in doing nothing.”

2.5 Empower believes drug regulations should be adapted to allow people with serious and life-threatening diseases to try out new combinations of drugs and to have access to drugs and treatments that are still in the test stage of development—this would include an enhanced ability to participate in clinical trials.

2.6 Further to this are the following three rights that are integral to making Empower’s mission a reality:

2.7 “You have the right to drugs and treatments that have been recommended by NICE for use in the NHS, if your doctor says they are clinically appropriate for you.”

2.8 “You have the right to expect local decisions on funding of other drugs and treatments to be made rationally following a proper consideration of the evidence. If the local NHS decides not to fund a drug or treatment you and your doctor feel would be right for you, they will explain that decision to you.”

2.9 “You have the right to be given information about your proposed treatment in advance, including any significant risks and any alternative treatments which may be available, and the risks involved in doing nothing.”

2.10 Empower believes that the above are fundamentally important rights for a patient.

2.11 However, in practice they are not always applied to their full extent—particularly in relation to patients with serious and life threatening diseases.

2.12 These patients are sometimes denied drugs/treatments that may help them manage their condition. The “risk ratio” (risk of harm vs. potential benefits) is different for these patients due to the severity of their condition.

2.13 With this in mind it is extremely important that they are fully involved in the decision making process of which treatment course to take “following a proper consideration of the evidence.”

2.14 In the spirit of pursuing treatments “rationally”, where a doctor believes a treatment is “clinically appropriate” and it is “recommended by NICE” it can only be right that a patient can make the final call on whether the risk of taking an untested combination of drugs outweighs the risk of “doing nothing.”

2.15 As the Committee has already heard from Dr Fiona Godlee, many patients want to enter clinical trials. Dr Godlee cited a hypothetical patient which we fully recognise “I am entering this trial because I want my results to be used for the widespread advancement of science. I am not in this trial to help industry. I am in it to help science.”

2.16 If patients were freer to decide on their course of treatment, and participation in clinical trials, it would have a number of consequences.

2.17 It would mean more drugs were used to treat a variety of diseases and there would therefore be an improved base of evidence to help fight disease.

2.18 If pharmaceutical companies believed that the drugs they develop could be applied to a wider number of conditions this would encourage significantly more investment in new drugs and benefit society as a whole.

May 2013

APPENDIX 1

THE HALPIN PROTOCOL

Background

Many people wish to assist in creating the conditions for the speed of drug and therapy innovation in rare diseases to accelerate. There is a sense that the joint effect of the regulation of Clinical Trials and the principles of legal liability that currently govern such Trials works against those interests and that 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.

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.

Prepared 16th September 2013