Trials and tribulations
pharmafile | October 14, 2013 | Feature | Manufacturing and Production, Medical Communications, Research and Development, Sales and Marketing | ABPI, NHS, eric low, myeloma, transparency
I have been chief executive of Myeloma UK since its inception 17 years ago, and its unique bedside to bench and back again organisational model has given me excellent insight into the barriers, vested interests and opportunities of stakeholders across the whole drug discovery, development and approval pathway.
Myeloma UK has an international vision and outlook which has afforded me the opportunity to distil the many ‘global’ factors that impact on the UK.
Above all else, I am a passionate patient advocate but one who understands that our desire to always do the best by patients means that all stakeholders doing their best at all times. It is only by understanding all perspectives and working and thinking in collaboration that we can deliver solutions and benefits that are fair to all.
Ensuring the benefits of data transparency
Few could argue with the recent calls for the proactive publication of clinical trial data to enable access to full data sets by interested parties. Although there are some potential risks in doing so, mostly to the pharmaceutical industry, these are overwhelmingly outweighed by the benefits – especially the benefits to patients.
For forever and a day, patients have been under-valued and have been given the minimum amount of information possible relating to the research they take part in.
Patients take part in research knowing that they may not directly benefit from doing so and, in the process, endure a whole host of often invasive tests and procedures alongside a range of unwanted side-effects. One can only conclude therefore that they do it out of altruism and in the hope and belief that future patients will benefit.
Despite this, patients are often not given the credit and thanks they deserve for their courage and endeavours – they are not even formally given the results of the research that they have taken part in.
If this isn’t bad enough, patients still get recruited to studies that are not addressing critical research questions, are poorly designed, have end-points and comparators that will not withstand the rigour of health technology appraisals, and which rarely impact on clinical practice.
Understanding what conclusions were made from the clinical research they participated in, and to know that new research is able to progress as a consequence of their participation, are both important issues and benefits to patient volunteers taking part in research.
So although the starting point for the proactive publication of clinical trial data may have been Brussels’ keenness for transparency, if done correctly, the proposed measures to increase transparency in clinical research could prove to be valuable to patients as well as to industry, researchers and ultimately payers in the following ways:
• Driving better designed and more cost effective clinical development programmes. For example, it could enable clinical research to be designed more quickly and targeted more effectively to specific patient populations, thereby supporting the increasing focus on stratified medicine
• Reducing the number of unnecessary clinical development programmes with resulting benefits to R&D portfolio management
• Vastly improving clinical trial planning, as well as patient safety issues
• Potentially enhancing the cost effectiveness of new drugs by providing health technology appraisal bodies with more complete data, and thus the ability to carry out a higher quality of evidence synthesis.
However – as is always the case but seldom carried out – very careful thought must be given to the practical on-the-ground implementation of ‘politicised’ policy to ensure that the perspectives of those who stand to benefit or lose most, are taken fully into account.
There has been wide frustration about the lack of clarity from EMA about the rationale for its intent to release clinical trial data. What is perfectly clear is that their need to do so has been predicated fundamentally on the growing expectation within the EU Commission over the last few years that all EU public organisations will move from a default position of privacy to one of openness.
In achieving this, however, it is important that we resist the temptation or the need to throw the baby out with the bath water. Transparency for its own sake is not good for anybody, so the trick is to link the political desire for it with the potential to benefit patients and other stakeholders at the frontline.
I would best describe myself as a ‘critical friend’ of industry and while much of what it does and how it does it leaves me scratching my head (and at times speechless), I am quite prepared to stand shoulder to shoulder with industry on issues that are important. This is one such issue.
I am pleased that, on the whole, industry has positively and enthusiastically embraced the need for better transparency in clinical research. Industry is right to be concerned about the vagueness of the proposals and, although it has a lot to gain from better transparency and access to data, the ‘why’, ‘who’, ‘how’ and ‘when’ questions must be carefully addressed.
The ‘method’ of access or release of data needs be clearly defined. If it is the intention to provide full access to all data, I see no issue or need for stringent management of data in which key parameters have been analysed – and of course there should be no barriers to accessing safety data.
That said, if raw data is to be released then I would argue that this needs very careful management. One way to deal with this might be to put in place a straightforward but stringent ‘application’ process underpinned by a clear rationale from the applicant as to why they are seeking access to the data – this could prevent unhelpful ‘fishing’ exercises and ‘cherry-picking’ of specific parts of the data that may not be representative or in context.
In order for this to happen, EMA would need to put in place an appropriate peer review or gatekeeping entity. A second concern for industry may surround the potential ‘commercial sensitivity’ of clinical research data.
If the intent is to only publish the part of the regulatory submission that relates to the clinical research (i.e., design methodologies, raw data and results) and not the other pre-clinical structural and formulation data which could be highly commercially sensitive, it is difficult to see what is truly commercially sensitive, but if all data were to be published, then this may be cause for concerns for industry.
Perhaps therefore the issue that needs to be addressed is not so much about the release of clinical data per se, but how it could be used by competitors in certain circumstances.
For instance, not all products are covered by patents and instead rely on data protection legislation. The EMA has said that it will not allow submissions within the EU from anyone other than the Marketing Authorisation Holder, using data released after an initial application, but there is nothing to stop a generics company or anyone else from compiling an application package and submitting it in other parts of the world.
Without mitigation, this could have significant downstream unintended consequences for patients in the EU. A scenario can be envisaged where EU submissions occur only after applications have been made in other parts of the world. This may achieve the unwanted outcome of putting patients in the EU at the back of the queue for some new medicines.
In summary, the move towards better transparency of clinical research data should be cautiously welcomed by all. If done properly, there could be huge benefits for both patients and industry. As is with all matters, the devil will be in the detail and much rests upon the ability of us all to translate the will of politicians and their broad policies into meaningful, practical and implementable policy that has significantly more upsides than downsides for key stakeholders at the front line.
Enough with the vagueness too. We now need the detail and in the in-depth discussion to turn the huge potential of better data transparency into reality. More importantly, we need to use the current spotlight on data transparency in clinical research to emphasise and recognise the incredible contribution that patient volunteers make.
We need to always put their needs and perspectives at the centre of decision-making making and planning across the whole clinical development programme. We must leave no stone unturned or any energy unused in pursuit of translating the results of clinical research into clinical practice where patients benefit.
Related Content
Digital mental health technologies – a valuable tool in supporting people with depression and anxiety
The potential benefits of digital mental health technology for managing depression, anxiety and stress, together …
A community-first future: which pathways will get us there?
In the final Gateway to Local Adoption article of 2025, Visions4Health caught up with Julian …
The Pharma Files: with Dr Ewen Cameron, Chief Executive of West Suffolk NHS Foundation Trust
Pharmafile chats with Dr Ewen Cameron, Chief Executive of West Suffolk NHS Foundation Trust, about …
