The Role of Public-Private Partnerships & Evidence Generation Christina Donatti LLB, BSc, MSc, PsyD IMI Lead, RWE EMEA Strategic Organisation. Janssen Cilag Janssen is proud to feature artwork created by people affected by the illnesses and diseases we are committed to treating and preventing.
Context The value and the need of health data and the journey so far Health data has the potential to change the way we deliver healthcare, shaping future innovation, improving patient outcomes and putting healthcare systems in Europe on a more sustainable path Payers and HTAs, demand real world data in order to grant access to innovative treatments and to engage in outcomes based financing schemes. However, access to this data is still challenging in Europe Growing trend that performance-based schemes will play a relevant role in the pricing of new medicines Only ~60% of European countries with EHR systems and different levels of maturity and coverage of diseases among European registries Several health data initiatives successfully demonstrated the value for Pharma in all the segments of the value chain. However, there is still a need for a scaled-up solution, since initiatives are fragmented in terms of scope, ownership and modalities for data governance
Key trends influencing health data Healthcare system trends Technology and innovation trends Evidence needs Payers demand more data to achieve/ maintain reimbursement in addition to classic clinical trial results Big data Rising awareness of the relevance of big data analysis across all sectors for real-time data-driven decision making Pay for outcomes Some payers see potential in linking payments to outcomes achieved Shift to the cloud Scalable processing, Storage & Software as a Service allowing huge capacity and ubiquitous access Patient willingness Patient willingness to share data high in fatal/ pediatric conditions and slowly increasing in other areas Smart devices "Smarter" objects with connection to the Internet, apps and cloud services, low power consumption and faster devices Transparency movement Following clinical trial data sharing debate, calls for observational data transparency can be expected next Cognitive computing Intelligent "AI-like" personalities able to perform complex tasks and offer nuanced recommendations
Health data is needed to improve patient health outcomes, HC system sustainability and drive innovation 1 The promise of data for public health is enormous Outcomes data reveals which practices work best and how to improve care quality Outcomes transparency allows informed provider choice Outcomes-driven healthcare could avoid up to 270.000 deaths in Europe p.a. 2 Data is necessary to achieve sustainable healthcare Outcomes data can identify ineffective practices, reducing cost of care and waste, freeing up resources for innovation Better care leads to fewer complications and follow-on cost Economic value of outcomes-driven healthcare is 60-80bn in Europe p.a. 3 Data is a key success factor for innovation New types and sources (i.e. smart devices, wearables) of health data stimulate innovation in healthcare Identification of patient sub-populations and focussing of research efforts on their specific characteristics Many EFPIA flagship initiatives rely heavily on access to health data
Growing trend suggesting performance-based schemes will play an important role in the pricing of new medicines France UK Pricing contracts are reviewed every five years to assess the performance of new medicines in real life settings Through the Early Access Medicines Scheme, the UK is actively exploring how to accelerate pre-approval regulatory pathways and how this can then be integrated into commissioning decisions by the NHS Italy Comprehensive infrastructure of patient registries for cancer care that allows an almost personalized reimbursement of new oncology drugs on the basis of actual patient response This system has resulted in prices that are more affordable, accelerated approval times and improved patient access Source: Paying for outcomes, 2016, EFPIA
However, generation and access to RWD still challenging in Europe... EHR Registries Only ~60% of European countries with a EHR systems Different levels of maturity and coverage of diseases among European registries "59 per cent (27 countries) report having a national EHR system" World Health Organization % of Member States using available national EHR systems in >50% of health care facilities 50 0 62 Primary care Clinics Health Care centers 60 Secondary care Hospitals Emergency Care centers 55 Tertiary care Specialised Care centers DISEASE GROUP Incl. registries identified by ICHOM for collaboration No of registries Coronary artery dis. 10 Colorectal cancer 6 Hip osteoarthritis 5 Breast cancer 4 Cerebrovascular dis. 4 Knee osteoarthritis 4 Chronic kidney dis. 3 Leukemia & lymphoma 3 Lung cancer 3 Cataract 2 Chronic back pain 2 COPD 2 Dementia 2 Depression 2 Diabetes 2 Schizophrenia 1 No of registries 13 7 4 2 1 1 Source: World Health Organisation "From innovation to implementation. ehealth in the European Region", 2016; ICHOM
Need for public-private collaboration To work and connect with real world data is necessarily collaborative and multidimensional in challenges In an increasing value and outcome-focused domain in healthcare, evidence is the critical enabler to an informed process of care delivery and optimal outcomes for patients There are many and varied actors in this Building on IMI1 programmes, e.g. EHR4CR, EMIF, ADVANCE, GetReal and other programmes, EFPIA is attempting to utilise the outputs of those PPPs, the relationships and their contributions There is a complex relationship between data custodians and data users, 80% of which is sociological, and 20% technological In IMI2 a new call, the EHDN will be supporting the PPP BD4BO initiative programmes Not one data source is the whole truth in the real world
Public Private Partnerships Are Critical To Solving The Challenges
The Innovative Medicines Initiative: the largest public-private partnership for health research worldwide 5 billion 2008 to 2024 Part of the EU Horizon 2020 R&D funding
IMI Has Delivered Across A Broad Spectrum Of The Pharma Value Chain DISCOVERY New lines of enquiry in drug development (thanks to large data sets) Identification of new hits and leads (screening center with proprietary compounds) First human Beta cell line for diabetes research Regulatory qualified safety biomarkers Computer predictive models for safety Novel approaches in toxicology de-risked EARLY DEVELOPMENT
IMI Has Delivered Across A Broad Spectrum Of The Pharma Value Chain LATE DEVELOPMENT Definition of regulatory endpoints. e.g. for autism. geriatric research. asthma. pain Development of antibiotics (new compounds, new formulations) Development of Ebola vaccines and diagnostics (clinical trials, manufacturing, diagnostics, deployment) Clinical trial infrastructure and fast-fail cohorts (pediatrics, antimicrobial resistance, autism) PATIENT ACCESS Definition of evidentiary standards for pragmatic trials Defining and measuring outcomes relevant for and aligned between patients, payers, regulators Progress in de-risking adaptive models based on real world evidence Integration of patient voice in benefit risk evaluations Methodological standards in Pharmacovigilance adopted by regulators
IMI Has Supported Key Projects
IMI Benefits for Pharma (in general) Stimulates interaction between pharmaceutical companies and academic institutions and SMEs in order to remove bottlenecks in drug discovery and development Free Access for research use to all project results cost efficient way of having precompetitive research done Provides forum for influencing development of regulations and guidelines by regulatory bodies Knowledge sharing and networking opportunities Enables evidence generation on a much larger scale 13
IMI The non-tangible benefits Proof of concept for new public private collaborations in pharmaceuticals - today the largest PPP in healthcare research IMI has driven the development of innovative collaboration models supporting unprecedented data and knowledge sharing, maximizing investment already made in R&D and delivering Regulators, health technology assessment agencies and patient groups all play pivotal roles in many projects leading to the generation of solutions acceptable to our major stakeholders A neutral platform for dialogue defining common agenda/priorities (participation of regulators, payers, patients, academia, SMEs) IMI works: tangible deliverables after less than 2 years a pace that no other funding scheme allows 14
IMI - Results beyond the state of the art Established robust validated models for Alzheimer, diabetes, schizophrenia, asthma, haematological malignancies, cardiovascular disease. Developed clinically relevant biomarkers for Alzheimer, diabetes, schizophrenia, asthma, RSV, and haematological malignancies Establishment and regulatory submission of key standards and tools for drug development in infectious diseases, COPD, diabetes Improved clinical trial design and process in schizophrenia, pain, autism Projects launched and planned on use of real life data and alignment of regulators and payers data requirement 15
Pharma industry already tackling opportunities along the value chain Research Development Public Health Market Access Medical Identification of novel target pathways by integrating diverse data sets Improvement of trials design and simulation EHR4CR Personalised prevention and targeted interventions Flexible pathways to provide early patient access MAPPs Identification of best practices to improve health outcomes and increase efficiency BD4BO Operations Integrated mechanisms to ensure consistency across the whole value chain Initiatives still fragmented in terms of scope, ownership and modalities for data governance Source: "An industry perspective on Big Data"; B. Vannieuwenhuyse; EFPIA; BCG expertise
Support research using health data: EMIF project overview EMIF-Metabolic EMIF-AD 57 partners from 14 European countries Identify predictors of metabolic complications in obesity Identify predictors of Alzheimer's Disease (AD) in the pre-clinical phase 56 million worth of resources Three projects in one EMIF-Platform Develop a framework for evaluating, enhancing and providing access to human health data across Europe, support EMIF-Metabolic and EMIF-AD (the specific topics above) as well as support research using human health data in general Academic Partners SME Partners Efpia Partners Patient Organisation Source: "An industry perspective on Big Data"; B. Vannieuwenhuyse; BCG expertise
Network of hospitals to overcome challenges in clinical development: EHR4CR Only 18% in Europe, and 7% in the US complete their enrolment on time 50% of today's clinical trials fail to achieve the target recruitment Almost 50% of all trial delays caused by patient recruitment problems 1/3 of protocol amendments are avoidable, at a cost of $0.5m Site dependent process No patient data leaves the hospitals EHR ETL CD W InSite Local Install InSite Central Platform Secure access for researchers Local InSite applications The hospital remains in control of its data EFPIA Partners Source: "An industry perspective on Big Data"; B. Vannieuwenhuyse; BCG expertise
HARMONY is currently the largest program under the IMI "Big Data for Better Outcomes" umbrella
The patient is at the center of HARMONY: Collaboration of all stakeholders needed to meet patients' needs Hematologists Bioinformaticians Data scientists & ICT Pharma Industry Economists Patients Policy makers Regulators HTAs Ethics
HARMONY aims to meet major unmet needs in HMs Need for harmonisation of outcome measures and endpoint definitions for HMs at European level Increasing omics data available, but limited application in clinical practice Healthcare Alliance for Resourceful Medicines Offensive against Neoplasms in HematologY Need for speeding up drug development, access pathways and "bench-to-bedside" process
Aims and Outputs 1 2 3 4 5 6 7 8 Establish a clinical data-sharing platform that empowers clinicians, patients and policy stakeholders to improve decision-making Create a community of European hematological malignancies stakeholders Improve access and management of Big Data series from HM patients Define meaningful and harmonized clinical endpoints and outcomes in HMs Provide tools for analyzing complex data sets Identify biomarkers that will contribute to timely patient access to more effective and better tolerated innovative therapies Share relevant information and knowledge on HMs Provide a framework for legal, ethical and governance issues
Need for public-private collaboration- The EHDN
Objectives of the full project: You cannot change, what you do not measure The first goal of the EHDN is to implement the approaches pioneered in these earlier research projects and develop a standard methodology The second goal of EHDN is to help mature both the supply side and the demand side of this health data eco-system in compliance with robust privacy and ethics governance The third goal of EHDN is to stimulate development of new and augmented health services through available and expanded technologies, in the interest of health outcomes
Objectives of the full project
Pre-competitive nature EHDN will be conducted as per all IMI programmes in a pre-competitive environment: The programme requires diverse collaboration for the development of an underpinning data network supportive of a value-based research ecosytem for all actors in this domain: It is primarily focused on real world data and infrastructure, and not on explicit health research Data and analytical methodologies for transitioning varied real world data to real world evidence EHDN is an enabler for other BD4BO research projects
Expected impact of the EHDN Pivotal: Network of diverse data sources facilitating increasing requirement for real world evidence to support expanding value-based and outcomes-focused healthcare delivery in Europe Critical: Technology deployment based on prior or relevant programmes (e.g. EHR4CR, EMIF, ADVANCE, OHDSI/OMOP) to facilitate the bi-directional relationship between data custodians and data users Enablers: Evolution of research methodologies, inclusive of harmonisation, to meet the evidence challenge of value and outcomes-based care within the context of multicentre, real world, observational research