Parliamentary Summit, 15th April 2013 - Video and Transcript
On 15th April a panel of academic, political, pharmaceutical and patient experts gathered in Parliament to to start the consultation process for the Halpin Protocol, a legislative blueprint designed to enable patients with life threatening conditions to have more influence on drug development and trials.
Chair: Geoffrey Clifton-Brown MP
- George Freeman MP
- Jamie Reed MP
- Stephen Whitehead –Chief Executive –ABPI
- Sharmila Nebhrajani-Chief Executive-Association of Medical Research Charities (AMRC)
- Steve Bates-Chief Executive-Bio Industry Association
- Lesley Greene-Patient Representative-Committee for Orphan Medicinal Products (COMP) at the European Medicine Agency (EMA)
- Professor Charles Craddock-Co-Founder-Cure Leukaemia
- Graham Hampson Silk- Co-Founder-Cure Leukaemia
- Geoff Thomas-Geoff Thomas Foundation
- Professor Sir Peter Lachmann - Emeritus Professor of Immunology - University of Cambridge
- Simon Lem-Country Manager UK, Ireland and Scandinavia-Vertex Pharmaceuticals
Geoffrey Clifton-Brown MP introduced the proceedings:
He stated that the reason why everyone is gathered here is because of Les; Geoffrey Clifton-Brown MP explained that he met Les nearly a year ago and agreed that they should start this campaign and this was all culminating in this afternoon’s event.
He then asked the panellists to introduce themselves.
Professor Sir Peter Lachmann gave his academic credentials. He said he was involved in the translation of medicine and had been President of the Academy of Medical Sciences and so has some background in medical problems. He said he was also a member of the AMRC from 1997 to 2002.
George Freeman MP said he was the newly elected MP for Mid-Norfolk. He described his previous career, the first part of which was in venture capital for start-up companies in the bio-medical sector and then he started a translation medical company. He said he used this experience when he helped the Government with the launch of their life science strategy eighteen months ago.
Sharmila Nebhrajani gave her credentials. She said the AMRC members fund a billion pounds worth of medical research every year in the UK. She said the majority of that money came from patients, donors and their families because medical research is the UK’s single most popular charitable cause. She then commented on the campaign stating that it was very important to AMRC members and also to the medical charities because people donate to charity in the hope of cures for serious and debilitating diseases.
Professor Charles Craddock explained that he was a director of a bone-marrow transplant programme at Queen Elizabeth Hospital in Birmingham that served about six million (one of the largest catchment areas in Europe). He said he was mindful in his clinical practice of the urgent need to connect patients with new therapies and mentioned he had led two initiatives in the last decade. The first was the development by leukaemia and lymphoma research of a trial’s acceleration programme which served a population of 20m across thirteen leukaemia centres and accelerated trial delivery and development through funding.
Recently with help from George Freeman MP and a number of other people, he secured£24m of funding of for the extension of the Institute of Translational Medicine in Birmingham which, again, has the same mission of accelerating access to new therapies in rare diseases and other treatments for leukaemia and lymphoma diseases.
Stephen Whitehead gave his background. He said the ABPI represents small, medium and large research based bio-pharmaceutical companies and that 30% of RND spent in the UK as a whole was spent by the pharmaceutical industry which represented about £4-5 billion spent by his members. He also stated that the ABPI was looking with urgency at access to new medicines and how they could change the regulatory framework.
Geoffrey Clifton-Brown MP interjected to say that Jamie Reed MP would be joining them a little later on.
Lesley Greene said she had several roles; first, as a mother and a care-giver. Her daughter, Jennifer, was diagnosed with the rare disease cystinosis. As a result of the diagnosis - that it was rare, genetically inherited and untreatable - she and her husband set up an organisation, the Research Trust for Metabolic Diseases in Children, later changed to CLIMB, Children Living with Inherited Metabolic Diseases. She said they were active in helping to get the orphan drug regulation adopted and that they joined forces with other organisations like EURORDIS, the European Organisation for Rare Diseases.
She said that since she retired from CLIMB, she has been working on the Committee for Orphan Medicinal Products at the EMA as a patient representative putting forward the views of the real-term effects on patients of the drugs that they are evaluating. From September of this year she will be the Vice-Chairman of COMP.
Graham Hampson Silk said he was a patient diagnosed twelve years ago with leukaemia. He said that the only reason he was sitting here today was because he was very lucky to have been given access to an untried drug, “to be a guinea pig”. This drug had enormous potential and an early phase trial.
Subsequently, together with Professor Charles Craddock, they started and funded a charitable organisation called Cure Leukaemia which has created an infrastructure and network of drugs trials that allows greater accelerated accessibility to new drugs and treatment regimes for patients. He said the organisation works closely with scientists in the pharma industry and has so far leveraged over £21m of drugs free for patients.
Simon Lem gave his credentials. He explained that Vertex was a pharmaceutical company that focuses on research and development and brings to market medicines in the rare diseases environment. He said he was interested to be invited to the debate because, whilst they don’t have a position on the use of medicines ahead of license, they have brought a medicine to market last year.
Geoff Thomas said he was a former international footballer and patient who was diagnosed with a blood cancer in 2003 and given thirteen months to live. He said that after he had a stem cell transplant and started to feel better he raised money for his campaign. He said it became clear to him that there was both a lack of infrastructure and a lack of early trials to benefit patients. He explained that what he was trying to do with this programme was bring about new innovations to benefit patients.
Steve Bates gave his credentials as Chief Executive of a trade association for the UK bio-tech companies. He said they have been involved down the years in discussions around policy and the need to bring innovative drugs to market.
Geoffrey Clifton-Brown MP then played a short video from Les Halpin.
Afterwards he read out a personal message from Les:
“Thank you for joining us here today. I am sorry I cannot be with you but let me assure you I am there with you in spirit. As you know, despite the limitations of this illness, I commissioned pushing forward this debate. However, I’m not the one who can change things, it is the people in this room who can make a difference. I am realistic enough to know that any change in the current regulatory environment in this area will likely be too late to have any significant impact on my condition.
But this isn’t about me. This is about the next generation of patients who deserve the chance to choose how they fight their battles and the risks they are prepared to take as long as those risks are taken in a controlled environment under strict guidance of their physician. It is the patients who know most about the conditions they are living with, day in and day out. All clinical trials and data should be shared publicly as this is the only way we’re going to speed up evidence based research and innovation.
I am overwhelmed by the support this campaign has already received and I cannot thank you enough for agreeing to be part of this discussion. Today is the day we take the campaign onto a new level and hopefully into the statute books. Please consider the protocol we’re going to launch later and give us your views, contribute to making the proposed change a reality.”
Following this Geoffrey Clifton-Brown MP launched the Halpin Protocol which seeks a relaxation of regulations governing the way drugs for people with serious and life threatening conditions are developed and prescribed to speed up innovation and access in this field.
Geoffrey Clifton-Brown MP then introduced Professor Sir Peter Lachmann and asked him to take everyone through the obstacles to the campaign and any possible solutions.
Professor Sir Peter Lachmann said he had been campaigning in this field for about ten years and that he was grateful to Les Halpin for actually making something happen. He outlined the background to drug development and went through studies on the regulation of drugs. He said that drugs were expensive to develop and that there was a long process of drug development. Only a handful of very large companies with very deep pockets could afford to do this.
Professor Sir Peter Lachmann pointed out that there has been a spectacular advancement in the treatment of rare diseases over the last few years.
He said there were lots of drugs developed by bio-tech companies which are simply lying on shelves; they may be very useful but their development is not taken any further because of the cost. This problem has been exacerbated by the issue of people litigating against companies, not because the companies have been fraudulent or negligent but because people have taken drugs produced by these companies which have been found to increase the chance of their dying earlier. He said this was unreasonable.
He then talked through the graphs from his handout. He explained graphs on post-marketing trials, problems with phase 3 trials and the problem of strict liability for drug companies. He explained how the concept of strict liability developed after the thalidomide scandal and was enshrined in European law so that drugs were subject to the same strictures as other consumer goods.
Professor Sir Peter Lachmann concluded with the admission that it would be time-consuming to bring about change in drug regulation laws, requiring a change in European Law and a change in the Consumer Protection Act 1987.
Geoffrey Clifton-Brown MP then asked George Freeman MP to give his thoughts on the issues surrounding the campaign and the Halpin Protocol.
George Freeman MP said that he completely endorsed what Professor Sir Peter Lachmann had said. He said if one thing was to be taken away from this summit today, it should be two slides on the handout which tell the story of the model of developing, discovering and bringing new drugs to market which is currently not working. He declared they were on the cusp of a profoundly new world of biological medical discovery which they needed to accept and understand. He likened it to an “Arab Spring” in medicine and said he thought it was going to change the assumptions on which policy makers create policy and how patients receive treatment. He talked about the Life Sciences strategy that the Government had implemented and how those in Parliament could make a difference.
He praised Les Halpin for his courage and determination in carrying through the campaign.
He criticised the old model of drug development for taking “between ten and twenty years to bring a drug to market” with a corresponding cost of £500m-£1bn and said the challenge was for those in Government to build public support and momentum.
George Freeman MP then explored the “new model of bio-medicine” that was being created which is more to do with the benefit to patients then profits and which would end the “one size fits all” approach to medicine.
He said this new model was genetically informed and was about a “new healthcare eco-system” where every patient is a research patient and the NHS is a research organisation, putting the UK’s history of patient data to use in pursuit of a much more targeted treatment. A new eco-system where everyone, academics, clinicians and patients are all working together in a much less hierarchical way and in a much more collaborative way centred around patients.
He explained that there would be a more philanthropic element, demonstrated by the activities of people like Bill Gates with his Gates Foundation, and also it would be about preventative therapies and much better diagnostics.
He said that he called it an Arab Spring because the public would not tolerate their relations dying because the Government was slow to regulate a drug which could help them.
George Freeman MP went on to set out in detail the ideas behind the Government’s Life Sciences Strategy. He explained that the Prime Minister had said that “every willing patient was also a research patient” and that this is what patients expect. Patients expect that their data is being used anonymously, they expect great clinician scientists to be using the history of clinical treatment to prevent others suffering.
He said one of the aims of the strategy was to ensure that the NHS integrated with the incredible work of UK academics to help bring about a new generation of treatment for patients. The UK must embrace a fundamentally translational, experimental model of medicine which allows patients to help themselves and unlocks an exciting opportunity to properly use the UK’s drug development budget to transform healthcare in the UK.
Geoffrey Clifton-Brown MP asked George Freeman MP about publishing clinical trial results from an industry perspective. George Freeman MP said that there must be a way of balancing the respectable qualm that these drugs companies invest vast amounts of money in something for their own benefit with the public good requirement.
Geoffrey Clifton-Brown MP then introduced Jamie Reed MP, a Shadow Health Minister since 2011. Jamie Reed MP said that George Freeman MP had made a series of compelling points about the need to champion a new model of medicine.
Jamie Reed MP pointed out that this area did not befit a narrow political interest because it was firmly in the national interest. He said that the underlying principle was that one must continue to encourage and facilitate access to new medicines as soon as possible while also maintaining a critical and fundamental focus on patient safety. Patient wellbeing must be the primary objective.
He said that the path to developing new drugs is littered with unpublished trials and that it was campaigns like this which would increase the power and the pressure and the mobilisation of people from all sides of the House.
Jamie Reed MP concluded by stating that he believed from a national political view that this campaign was the beginning of an effective dialogue, that he was “up for this challenge” and that his “door was always open to address these issues”.
Geoffrey Clifton-Brown MP then introduced Sharmila Nebhrajani.
Sharmila Nebhrajani expressed her appreciation that the entire panel agreed with each other. She said that she wanted to make a few points from a charity funding perspective and a patient perspective.
From a charitable perspective, she said that patients tell her that they want to be involved in research even when they are healthy themselves. She said there was a “groundswell of opinion” in favour of doing this even though the public were aware of being involved in such research. She said there was a “spectrum” of risk and benefit and that patients should be given the choice to decide where on the spectrum they would want to be.
She urged the panel to “encourage a different calculus for the whole regulatory environment” where potential drug cures for the most serious diseases were approved as soon as possible. There must be an “agile and proportionate” response for all drugs discovery.
She then pointed to Professor Sir Peter Lachmann’s charts which illustrate the different phases of the trials and declared that they needed to have a conversation about what the risks are and what the benefits are from pre through to post-marketing. There also needs to be systems in place that capture the data that tells whether drugs are working or not. Critically, they need patients and clinicians to be in this debate, for clinicians to see “where is the benefit for my patients and where is the risk”.
Geoffrey Clifton-Brown MP then introduced Professor Charles Craddock.
Professor Charles Craddock said that he wanted to set out briefly the remarkable progress that had already been made and the challenges ahead. He said George Freeman MP could not have illustrated the opportunities better in his life sciences document. There are a raft of new drugs, devices and transplant therapies that are emerging for introduction to patients. Using the “DNA revolution” there is now a real opportunity to identify which patients will respond and also a big opportunity for the UK as probably the only place internationally that can deliver a large coordinated network of high-quality clinicians.
He then set out the challenges and explained the tragedy inherent in having to tell a patient that there was nothing he could do for them. He pointed out that that tragedy was even more poignant when they both knew that in the past few months a whole of range of potentially active drugs have been developed that could make a difference in saving that patient’s life but there simply wasn’t the capacity in the system to run these trials for the drugs. “The drugs are there and the patients are dying”. He said this needs to change.
He said that this situation can be fixed but he is currently frustrated because policy is being made by people who had never sat in a clinic and who don’t understand the needs of patients.
Large networks of collaborative hospitals are needed so that new therapies can be trialed as rapidly as possible to create a collegiate system between major teaching hospitals. He said that people who looked after patients on the clinical studies needed to be funded properly, the clinical research nurses because it was incredibly difficult to get funding.
Professor Charles Craddock urged the panel to address the regulatory framework, to think about those who delivered the trials and incentivise the partners so that there are rewards for developing a clinical trial. The chief executives and the vice chancellors at universities must also be incentivised to make them drive clinical research as an absolute priority.
He also said that an infrastructure needs to be created that will enable researchers, patients and clinicians to all work together and that a clinical trial programme would have to be integrated properly with the market.
Geoffrey Clifton-Brown MP then introduced Stephen Whitehead.
Stephen Whitehead said that he wanted to challenge the orthodoxy about ‘big pharma’. He pointed out that there is no such thing as big pharma. There is a complex eco-system but big pharma has long since gone and the one model fits all is not relevant anymore.
He said the future for this industry has been represented well by what George Freeman MP has set out in his Life Sciences Review. It will chiefly be achieved through patient partnerships and close partnerships between charities and the NHS.
Stephen Whitehead said that there are many successful orphan medicines on the market and that there is a willingness on the part of the NHS to buy these. He gave the example of Vertex’s funding from the NHS.
He criticised the current regulatory model for not evolving quickly enough and said that there needs to be a better settlement of risk and benefits. This includes looking at the current litigation issues involved in the process.
He also highlighted the cost of producing drugs and said that they needed to bring down both the cost and the length of time taken to develop and approve drugs. They also need to “reduce the cost of failure” by greater exposure in Phase 2 and earlier sentencing of medicines.
He said it was a concern of his industry to find out how to make a commercial return on drugs for shareholders. Nearly all pharmaceutical discoveries have been driven by pharmaceutical companies and so they must ensure that that risk gets reward.
The view of the ABPI is that the way forward is to do a lot more partnering with universities, charities, the Government and the NHS. This would include sharing intellectual property and data but there must also be a much bigger and more active patient voice.
In terms of data transparency Stephen Whitehead said he did not think there was a lack of it. He stated that under the Code of Practice of the ABPI, all data must be published and that the data was there for people to look at.
Geoffrey Clifton-Brown MP then introduced Lesley Greene.
Lesley Greene said that in many ways, the rare disease field had been an “innovator and a pioneer”. She said that there were two stages with the European Commission and drug regulation. In order to be incentivised, a potential orphan drug needs to receive an orphan drug designation and to do this it needs to fulfill specific criteria. She stated that to date there have been 1,083 designations but of those, only 78 have got to market.
She also stated that there was a lot going on in looking at ways to speed up the process, one of which is looking at the problems relating to Phase 3. Another problem is the small population who suffer from the disease.
Geoffrey Clifton-Brown MP then introduced Graham Hampson Silk.
Graham Hampson Silk said that he was a leukaemia patient who suffered from a disease which caused many people to die unnecessarily. He said the big problem was that the necessary infrastructure was not in place to enable the translation process to occur, and this could be created through establishing networks. He stated that there is a need to get more patients on these drug trials and that the trials need to include a large catchment area of the population with a good ethnic mix. .
He said that his organisation had created over 120 jobs and has become a platform for SMEs. However, he admitted that he was still concerned with getting benefit for patients more quickly. “If luck was taken out of the equation, you create a structure where every patient has the same rights, the same access to new medicine.”
Graham Hampson Silk declared that the Halpin Protocol is vital in ensuring a greater patient benefit for thousands and also in causing the acceleration of the drug development process.
Geoffrey Clifton-Brown MP then introduced Simon Lem.
Simon Lem said that the fact that he was able to have his drug developed was a success story but that access to medicine was a complex regulatory issue. He stated that he was participating in the summit to comment on how a small pharma company can bring a medicine to market. Simon Lem stated that he only has one product in the marketplace in Europe and this was a target medicine to treat the underlying symptoms for cystic fibrosis. He said it had taken 14 years to develop the drug and bring it to market and that getting access once you have the licence is another issue (getting the NHS to buy your drug).He questioned what steps the Government was taking for patients to get access to new products once they have been approved.
Geoffrey Clifton-Brown MP then introduced Geoff Thomas.
Geoff Thomas said he was going to speak from a patient’s point of view. He gave the example in Birmingham of uniting sick patients and hospitals and scientists. He also pinned the blame for the slow progress of drug development on the lack of infrastructure.
He said that he had set up his foundation because he wanted to know more about patients and how they could benefit further. He said that, with others, he had met various representatives from the political world who had told him that what he was campaigning for was right and should be happening. It was about using the benefits of the system that were already there and getting a template in place that would make the process quicker.
Geoffrey Clifton-Brown MP said that the UK had some of the best science in the world, including the NHS, and the best clinicians. He said they now needed the right framework to build on.
Geoff Thomas said that there was a lack of clinical research and that they needed to break down all the barriers preventing the rapid development of drugs.
Geoffrey Clifton-Brown MP said that Jeremy Hunt was very patient focused and was sure that Mr Hunt would want to do this.
Geoffrey Clifton-Brown MP then introduced Steve Bates.
Steve Bates started by saying that he fully supported the campaign. He said that he looked at Les as not just a patient but also as a man but also as a businessman. He said that two rules needed to be changed: firstly, the regulatory rules (the risk appetite of patients with terminal diseases is different to that of regulators who have a legal responsibility to regulate medicines) that govern drug development to reduce the time and cost of bringing drugs to market, and secondly, the rules governing investment management so that smaller companies could also be given the opportunities to take risks.
Steve Bates also said that charities should get involved more. He praised the Government’s Life Sciences Strategy as being “fantastic” and said an early access scheme must be implemented to get over the Phase 3 issue.
He said we all need to ask if there are “things on the shelf that are not being used” and also if there are difficulties in the actual “science” of the system.
Geoffrey Clifton-Brown MP then opened the debate to the floor.
Brian Quick, Chairman of Daval International Limited, said he represented a small pharma which has a product which has completed Phase 2 of the trials but that cannot complete Phase 3 as it is too expensive. He stated his options as being either to sell it to another company or get a conditional approval from the Medicines and Healthcare Products Regulatory Agency (MHRA)
He then went on to state that we need hospitals to get onto clinical trials and asked how the Government could help his company and how they can attract more investment.
Another observer, Leslie Galloway, Chairman of the Ethical Medical Industries Group, said there was a company in the UK on Phase 2 study that was given a licence so there is precedence.
George Freeman MP commented that this challenge, regarding how to get investment in the development of drugs, demands some fresh thinking. It is all about value, the tangible value of preventing disease for someone who needs it and the long term investment in the UK, preventing future cost.
Steve Bates said that Brian Quick should go to the MHRA and make the case to them. This has worked in some cases.
Sharmila Nebhrajani said that it was right to talk about value but that we need to think again about where we want to be on risk spectrum. There needs to be a different conversation about risk that is informed by patients.
Stephen Whitehead said that if there was a pool of money available at Phase 2, it would make it much easier for companies. He said they had been here before with HIV and that early access schemes worked.
Professor Sir Peter Lachmann said that the whole of medicine was to do with the consideration of risk benefit. The Halpin Protocol is quite specific; one should go for one thing at a time.
Professor Charles Craddock said there is an urgency to this. One can’t just fix one bit and hope it will affect the rest. For instance, we need to share intellectual property. Unless the problem of culture of middle managers in hospitals is fixed, the patients won’t get the benefits.
Geoffrey Clifton-Brown MP then closed the summit. He said that Les Halpin’s campaign is all about patients and that in this debate the focus should always be on patients. He also stated that in this there is a huge opportunity for the NHS and the country as a whole.
Geoffrey Clifton-Brown MP concluded by saying that we should all embrace the campaign “with energy and enthusiasm” and that he will not give up until we have achieved what Les started.