IMI 2 strategic research agenda: Aiming to make a real impact on everyday healthcare and patients
10.09.13
IMI2’s SRA outlines the major challenges currently facing the European healthcare system, the pharmaceutical industry and the regulatory framework and provides an outline of the research required to address each of these in turn. To successfully realise the ambitious goals of IMI2, all of these areas need to be addressed – but they can’t be successfully addressed by any one stakeholder or even by the public-private partnership of IMI alone. To address major bottlenecks in drug discovery and delivery, the SRA has identified four major areas where an integrated approach will have significant impact. By pursuing these four areas, we can increase the probability of success and reduce the cost of new medicines overall – allowing IMI2 to reach its goal of delivering the right prevention and treatment to the right patient at the right time.
(1.) Target Identification and Biomarker Research
The attrition rates of clinical trials today are, unfortunately, extremely high. This results in setbacks for many stakeholders – industry, researchers, patients in need of new medicines, and healthcare systems supporting patients. High attrition rates are largely due to a lack of efficacy in translating results from pre-clinical models to the clinical setting (testing on humans), coupled with the emergence of adverse events not predicted from the pre-clinical models. IMI2 will focus on gaining a better understanding of disease mechanisms to facilitate target identification, and also use this information for the identification of biomarker that can help predict efficacy, as well as biomarkers that can serve as surrogate markers in clinical trials. Efforts will also be made to better understand the molecular mechanisms underlying adverse events, and to identify new methodologies of assessing the response to the medicines at an organ level.
(2.) Driving the adoption of innovative clinical trial design
Clinical trials account for a large proportion of the overall development costs of any new medicine. Increasingly, trial models are moving away from traditional randomised clinical trial (RCT). The drive towards precision medicines is further driving home the need for new clinical trial paradigms, which support the evaluation of benefit/risk in small numbers of stratified patient populations and the development of infrastructures for collection and sharing of trial data. The promise of precision medicines is great but the challenges are daunting – especially because getting patient access to such innovative medicines also requires consideration of health technology assessment (HTA) agencies and other decision makers, who now assess the expected future value of a medicine when used in “real world” clinical practice. “Real world” data is thus becoming ever more relevant to clinical research. Again, there is big promise but also challenges in managing the collection, validation and analysis of data, cost-effectiveness of data collection, and as presented by privacy laws and data security. Addressing these challenges requires an integrated approach exploring the development of novel biomarkers and patient focused clinical endpoints, innovative trial designs, patient centred benefit/risk and effectiveness/risk assessment and alternative regulatory pathways to ensure that future development strategies address the needs of all decision makers from the outset. The level of integration required to achieve this ambitious goal would not be possible without the PPP framework being proposed.
(3.) Innovative Medicines for High Impact Disease Areas
An ageing population and increased incidence of chronic disease is driving the need for investment in preventative medicines. European healthcare systems are already under strain, and it is thus essential to incentivise research in disease areas with a high impact on society – such as Alzheimer’s and osteoarthritis. There is also a need to stimulate research and investment in areas where there is high public health concern, but where companies have largely withdrawn from drug discovery, such as antibiotic resistance and stroke. Taking this into account, the SRA focusses on 11 priority disease areas: Antimicrobial resistance, Osteoarthritis, Cardiovascular diseases, Diabetes, Neurodegenerative diseases, Psychiatric diseases, Respiratory diseases, Autoimmune diseases, Ageing-associated diseases/conditions, Cancer, Orphan Disease. The development of innovative medicines for these high-impact disease areas is essential to advancing public health interests.
(4.) Patient-Tailored Adherence Programmes
One of the biggest challenges facing the pharmaceutical industry and healthcare providers alike is the issue of patients failing to adhere to prescribe dosing regimens which compromises the effectiveness of prescribed medications. This is a problem which is only set to become more prevalent as the trend for healthcare to be pushed outside the hospital setting increases and the age of the population increases. Effective adherence programmes will require an integrated approach which provides a range of services from clinical support and education to patients, the development of new drug delivery systems to the development of robust, portable monitoring devices. Including patients, healthcare providers, the regulatory authorities and HTA agencies in the design of such programmes will be key to their success to ensure they are optimally tailored for the end user while fulfilling regulatory requirements.
These four points pursued by the IMI2 Strategy Research Agenda are based on the Innovative Medicines Initiatives commitment to addressing the changing face of European healthcare and public health challenges. By advancing these areas, we will be moving closer to our goal of the right treatment for the right patient at the right time: This means not only discovering new medicinal molecules but translating those discoveries into tangible treatments – and ensuring that patients have access to them. The end goal is implementation in daily medical practice. This is what the Innovative Medicines Initiative’s research agenda ultimately seeks to impact, ensuring that new medicines will make a real difference in the lives of patients.