Bringing better medicines to patients faster: adaptive pathways and health outcomes in IMI (ADAPT SMART & BD4BO)
Can patients, doctors, healthcare systems, and companies afford waiting 12 to 15 years until a new medicine reaches those in need? What is the precise value of these medicines for patients, healthcare and social security systems? The Innovative Medicines Initiative is addressing these questions in a transparent and evidence-based way, through a series of public-private projects that would bring to the forefront and place under scrutiny questions that today are debated in specialist circles and closed fora.
The Adapt Smart consortium kicks off today and will assess under which conditions it would be possible to bring medicines to patients as early as possible, without compromising on safety, quality or efficacy. The adaptive pathways concept relies on a different – but no less robust – set of data than those produced under more conventional development routes. It also generates information on how a medicine behaves not in artificial controlled trial environment, but in real life and in real patients who often suffer from more than one disease and have a lifestyle that would anyway exclude them from those standard controlled trials. This means we can measure whether the claimed effects can be verified.
Adapt Smart – a multi-stakeholder platform led by EMA and linking in HTA bodies, regulators, payers, patients, academics and industry – is set to address the myriad of questions raised by sceptics. They include: what products qualify; which patients; what data; what is the impact on healthcare and social security systems; how do we re-evaluate medicines based on real world evidence and with what resources; and – most importantly – how can we do it within the current legal framework, which sets formidable standards of safety, quality and efficacy.
Crucially, the work will not be limited to the consortium alone. In fact. the workplan allows for engagement with wider audiences across stakeholders groups and geographies at each milestone. You can already view the website here: http://adaptsmart.eu/
How do we really measure value – in terms of health outcomes – and how can this de defined? Answering these questions supports a more rational development of medicines based on what matters to patients, public health and society. It will also enable adaptive approaches and ultimately help to identify what works most efficiently for them and brings best value to healthcare, social security and labour organisations.
The concept of leveraging big data (and real world data, i.e. from real use of healthcare interventions in real patient populations) for measuring outcomes, materialised in a proposal for an IMI programme on Big Data for Better Outcomes. Now it will turn the spotlight on questions relating to what constitutes meaningful outcomes for different diseases and interventions, how to measure them, how to get information from patients and healthcare professionals in real time, and how to interpret the data. The first topics and call for expressions of interest under IMI will be published in September. The concept though has already been the subject of broad public consultation via the internet (see here) and the preliminary topics are also available on line (see here).
These are two complementary endeavours that will only deliver if based on robust evidence and aligned understanding of what is expected to happen in real medical and healthcare practice. There are no ready-made solutions. If all stakeholders, including large and small companies, put their brains together to rise to a considerable challenge and creative out-of-the-box thinking, it should help translate the promise of science into healthcare solutions. If we fail to find replies to each and every question – science is unpredictable – we nevertheless will have made some meaningful progress.
I am keeping my fingers crossed.
PS: Why IMI? Notwithstanding all critics of this programme: do you know any other platform which can bring together all stakeholders, public and private resources, based on transparent process and work subject to continued scrutiny…? www.imi.europa.eu1