Introduzione

Regulators, health technology assessment (HTA) bodies, academics, bio-pharmaceuticals companies, payers, and patients are presently rethinking the healthcare ecosystem and facing diverse and increasing challenges at different levels. In this conceptual modelling of the healthcare ecosystem, the potential value of Real-World Data (RWD) for decision making has never been more relevant.
Real-World studies seek to provide complementary evidence to that provided by randomized controlled trials. While the latter provide evidence of efficacy, Real-World Data provide information on therapeutic effectiveness in a real-world practice setting.
Pharmaceutical companies need to focus on innovative and targeted therapies both due to the increased competition in the sector, and of the number of better structured and demanding patients’ groups or associations. Pharmaceutical companies are presently discussing on:

  • How does RWD contribute to translational research?
  • Whether there are any companies optimally leveraging RWD and how its application to biobanks can better advance innovation and discovery of targeted therapies?
  • What are the structural and logistical needs for this to be effective?
  • What have we already learned about the process?

Regulatory bodies face the challenge of ensuring that only safe and effective medicines reach patients; a decision which so far has only been substantiated upon data from RCTs. They actually need to know:

  • Whether other stakeholders can help them in the process and how?
  • Where and how do we using RWD in the drug development and approval process?
  • What has already been achieved in the field and what the success factors are?

Payers, and HTA bodies want to make sure that the most innovative and novel medicines reach patients who need them most as quickly as possible; and that these medicines provide value for money to all parties: patients and health care systems. They may ask themselves:

  • How can sponsors better demonstrate the value of their innovative medicines using RWD?
  • How can they work with decision-makers to establish agreed upon standards for RWE research?
  • How do recent frameworks (FDA, NICE, etc) help advance RWE research?

What brings all stakeholders at the same level is the need for new paradigms for a more performing healthcare ecosystem that reduce the gap between its representation and its implementation. Emerging e-health models and applications can greatly contribute to it, particularly, if hand-in-hand with Real-World Data Studies. The European Epidemiological Forum will review the progress and perspectives on the value of RWD in the drug life cycle, will discuss what RWD promises, what the barriers may be and what a successful story may look like.

Scientific Board:
Christian Agboton - Sr Global Brand Medical Director at Takeda
Maurille Feudjo Tepie - Observational Research Director at Amgen
Michele Intorcia - HEOR Senior Director at Apellis Pharmaceuticals

Who should attend?
The conference is addressed to professionals with a deep interest in drug development, healthcare decision making, academia and healthcare research, belonging to department such as: Medical Affairs, R&D, HTA/Market access, Medical Informatics, Clinical Operations, Regulatory Affairs, Pharmacovigilance, Biostatistics and Data Management from Pharmaceutical, Biotechnology and Medical Device companies, CROs, Universities/Hospitals, Academic Research, Patient Associations and Healthcare Organizations.

Programma

All times indicated are Central Europe Summer Time

10:00
10:20
Welcome
10:20
10:45
Fostering trust among RWE stakeholders: Would medium to long-term partnerships help?
Maurille Feudjo Tepie - Observational Research Director at Amgen

This session will present an overview on the value of Real-World Data (RWE) and Real-World Evidence (RWE) going beyond data and evidence themselves!
Bearing in mind the recent COVID-19 crisis, we will address the importance of open long-term collaborations between private and public stakeholders to collect and use RWD and RWE. These may benefit from the healthcare ecosystem and help build trust among the public and end-users of health advancements. We need a change of mentality that brings together innovative approaches and intensive analytics!

10:45
11:10
Critical thinking and Real world Evidence
Elena Peruzzi - Evidence Generation & Data Analytics Head at Novartis

Evidence needs are increasing and evolving. Ideally, one may start local strategic Real-World Evidence (RWE) planning at the Drug Development Program (DDP)/end of phase 1 (launch –5 years) and should continue during all drug development phases and after launch. This enables a successful collaboration and discussion with National Health Services (NHS), which leads to a better impact on public health. RWE is crucial to better understand how drugs perform in broader patients groups.

We are nowadays asked to produce all of the clinical, epidemiological, organizational evidence that may support adopting a health value-based approach. Partnerships between Pharma companies and NHS are the only solution to face the complex present challenges in health care, which are increasing more and more.

The pandemic of Sars-CoV-2 has accelerated this process, highlighting the need for timely, valuable evidence, better data, and registries, open collaboration between all the stakeholders, flexibility, and the ability to make decisions based on RWE.

Are we ready? Do we have the right mindset? Do we have the needed know-how? Are we fast and smart enough in conducting studies? Are we focusing on real gaps? Are we able to reach patients, pharmacies, GPs, and caregivers also to understand how a new therapeutic approach can properly work? Are we capitalizing on the experiences of other countries? Are we able to partner with the Health System early on and in a collaborative way?

I don’t have all of the answers, however, I would be happy to share some experiences about:

– Partnering with NHS: the implementation research
– Innovation and operational aspects in RWE generation
– How to better listen to patients’ voice

11:10
11:20
Break
11:20
11:45
NICE Guidance - Widening the evidence base: use of broader data and applied analytics in NICE’s work
Adrian Jonas - Associate Director for Data and Analytics at National Institute for Health and Care Excellence (NICE)

The National Institute for Health and Care Excellence (NICE) provides national guidance and advice to improve health and social care. In January 2020 we published a Statement of Intent to increase and extend the use of data in the development and evaluation of our guidance. This explores:

  • What kind of evidence does NICE currently use to develop guidance
  • What broader types of data are available
  • When and why should broader types of data be considered
  • Practical considerations associated with data analytics

We have since defined the programme to develop a methods and standards framework for activities involving broader sources of data and applied analytics. We accelerated some aspects of this to ensure fit-for-purpose analysis during the COVID-19 pandemic. It can be summarised into five key topics, which are crucial to address to ensure best practice in conducting high quality analyses of data. These are Research Governance; Data; Analysis; Results and Dissemination. In addition to these topics, the programme will consider some cross cutting issues ranging from transparency and public trust, to the validation and evaluation of artificial intelligence [AI] in digital technology.

The presentation will give an overview of the transformational journey to increase the use of broader data and applied analytics in NICE Guidance. It will also touch on the some of the issues we are considering with Standards; which will be crucial for any organisation seeking to ensure their approach is suitable for regulatory assessment/evaluation.

11:45
12:10
HTA, RWD and RWE: Validating and leveraging the different sources of evidence
Oriol Solà-Morales - CEO at HITT - Health Innovation Technology Transfer

In the past couple of years, development of IT systems and IT capacity have brought a huge amount of data that could be potentially used to support clinical, managerial and policy decision-making. However, so far the utilisation of RWD or RWE in the approval or reimbursement of drugs is very limited. There may be some reasons behind it notwithstanding the methodological concerns. HTA is a formal process to review structured evidence, but HTA has never been designed to evaluate neither systematic RWD nor RWE, in spite of major transformations in healthcare stem at RWE.

The objective of this presentation will be to review both the methodological and non-methodological concerns from payers and decision makers regarding the use RWE, and how can we try to overcome those limitations, with particular focus on non-methodological concerns. Equally we will try to build on some examples of submissions that have been used in the past in country-specific jurisdictions to approve/reject the usage of a new drug.

Main takeaways for the audience should be understanding how to better use the data available to support their submissions.

12:10
12:30
What did we learn this morning?
12:30
13:50
Lunch break
13:50
14:15
Value Based agreements & Innovative Contracting with Medicines: The value of Real-World Evidence for improving access & reimbursement
Omar Ali - Visiting Lecturer Value Based Pricing at University of Portsmouth & Former Adviser to NICE

Regulators, health technology assessment (HTA) bodies, academics, bio-pharmaceuticals companies, payers, and patients are presently rethinking the healthcare ecosystem and facing diverse and increasing challenges at different levels. With ever increasing demands on healthcare costs and considerable focus on drug pricing across global markets, conceptual modelling within value-based agreements between drug manufacturers and payers has never been more important.

The potential for Real-World studies to provide complementary evidence to that provided by randomized controlled trials are becoming more established within formal assessment and appraisal processes across the globe. Whilst RCTs provide evidence of efficacy, Real-World Data provide information on therapeutic effectiveness in a real-world practice setting. Pharmaceutical companies need to focus on innovative and targeted therapies both due to the increased competition in the sector, and of the number of better structured and demanding patients’ groups or associations.

Payers, and HTA bodies want to make sure that the most innovative and novel medicines reach patients who need them most as quickly as possible; and that these medicines provide value for money to all parties: patients and health care systems. They will need to address the following:

  • How can sponsors better demonstrate the value of their innovative medicines using RWD?
  • How can they work with decision-makers to establish agreed upon standards for RWE research?
  • How do recent frameworks (FDA, NICE, etc) help advance RWE research?
  • How does RWE address critical issues such as cost effectiveness, budget impact and uncertainty?

What brings all stakeholders at the same level is the need for new paradigms for a more performing healthcare ecosystem that reduce the gap between its representation and its implementation. Emerging e-health models and applications can greatly contribute to it, particularly, if hand-in-hand with Real-World Data Studies. The European Epidemiological Forum will review the progress and perspectives on the value of RWD in ensuring access and reimbursement of new innovative medicines, will discuss what RWD promises, what the barriers may be and what a successful story may look like.

14:15
14:40
Data Transparency and Privacy in Real-World Research
Raquel Billiones - Subject Matter Expert on Medical/Regulatory/Scientific Writing, Data Disclosure and Protection, Pharma/Medical Devices

We live in a data-driven environment. As clinical researchers, epidemiologists and scientists, we generate, use, and share data on a daily basis.

The current drug regulatory landscape supports the use of non-conventional clinical data sources to compensate for the lack of generalisability of data from clinical trials. Recent guidelines are encouraging the use real-world data (RWD) collected outside the controlled environment of clinical trials as clinical evidence to support efficacy and safety of health products. Real world evidence (RWE) is collected in many different ways, including patient registries, mobile devices, geolocation information and wearables, and is largely technology driven.

Any data we collect needs to be used appropriately and responsibly. Two key aspects in data use that we need to consider are data transparency and data privacy. Though these may be contradictory first glance, these two principles are actually complementary.

The new EU regulations and updated ICH guidelines are pushing for public disclosure of data through many different platforms, such as publicly accessible clinical trial databases and open access biomedical publications. The aim of transparency is to promote innovation and trust in the healthcare industry.

Coupled to data sharing is maintaining data privacy of the individual. The General Data Protection Regulation was adopted to harmonise data protection across the EU, provide better protection to data subjects, and clarify the responsibilities of data controllers and processors.

Taken together, these regulations and guidelines provide researchers and scientists a solid framework to perform research that is scientifically sound, fair, and patient-centric.

This presentation will discuss the interplay between data collection, data transparency and data protection and the regulations governing them in the context of RWE generation, use, and dissemination. The relevant guidelines and regulations will be discussed and case studies will be presented.

14:40
15:05
Digitalization in Data Capturing Impacts Scalability of Patient Recruitment - Using the 2D Matrix Code of Outer Drug Packages as Patient Identifier for ePRO studies – The DePRO study
Christian Müller - Teamleader Data Generation at Bayer

A fully digital data collection system, including patient identification, would represent a substantial advance in how patient-reported outcomes (PRO) data are measured. Within the European Union, one way to identify patients without the involvement of healthcare professionals is to use the unique 2D matrix codes on the packaging of prescription medication.

DePRO is an observational, multi-center, cross-sectional, digital, patient-driven study conducted in Germany. Patients with a prescription for a metformin-containing medication are given a postcard by their pharmacist, which includes a download link for the my ePRO app. Participants use their own mobile device to download the my ePRO app and access the DePRO study, for which they can register using the 2D matrix code on their medication. An electronic informed consent is collected.

The PRO instruments used in the study are the summary of diabetes self-care activities scale, the diabetes treatment satisfaction questionnaire and the EQ-5D questionnaire.

Patients are also asked to complete a questionnaire with items addressing demographics, patient characteristics, disease history, complications and concomitant medications. Enrollment of the first participants started in June 2020 (recruitment period, 3 months; planned sample size, 300 patients). The design of the DePRO study represents a substantial advance in scalability using digital capturing of PRO data.

15:05
15:15
Break
15:15
16:15
WORKSHOP | Covid-19 Pandemic: RWE, Opportunities and Challenges

The COVID-19 pandemic provided a dramatic example of how big data can provide quick access to invaluable information and a unique opportunity for decision-makers to respond rapidly to a public health emergency while posing new challenges for the scientific community on data integrity and reliability.

Some analyses and interpretations were controversial – leading to 3 retracted papers in 3 leading medical journals; public health authorities did contribute to misinformation (e.g. use of ibuprofen) and decision-makers were not prepared.

During the workshop we will study the anatomy of a crisis that illustrates the need for specific approaches to big data and collaboration at different levels of the decision-making process.

Managed by

Christian Agboton – Sr Global Brand Medical Director at Takeda
Maurille Feudjo Tepie – Observational Research Director at Amgen
Michele Intorcia – HEOR Senior Director at Apellis Pharmaceuticals

16:15
16:30
Conclusion
Relatori
Christian Agboton
Info Scientific Board

Christian Agboton

Sr Global Brand Medical Director at Takeda
Christian J. Agboton currently serves as Senior Global Brand Medical Director at Takeda based in Zurich, Switzerland. He previously served as Medical Director of Biosimilars Immunology / inflammation at Sandoz and Hospira/Pfizer. Christian also worked at GSK vaccines, Merck Serono and UCB Pharma in Clinical and Medical Affairs roles. Channeling innovation to deliver evidence while keeping quality and timelines has always been at the heart of his approach to data generation. Christian is Fellow of the Royal Faculty of Pharmaceutical Medicine (FFPM).
Maurille Feudjo Tepie
Info Scientific Board

Maurille Feudjo Tepie

Observational Research Director at Amgen
Dr. Maurille Feudjo Tepie is an Observational Research Director at Amgen and leads the Amgen European Team of the Centre for Observational Research. Prior to joining Amgen in 2009, he spent six years in the Worldwide Epidemiology Department at GSK. Maurille obtained his PhD in Medical Statistics from the London School of Hygiene and Tropical Medicine and then completed a one year post-doctoral fellowship working on a collaborative methodological project between the Worldwide Epidemiology Department of GSK and the London School of Hygiene and Tropical Medicine, under the supervision of Profs Stuart Pocock and Steven Evans. He has considerable experience designing and executing appropriate observational research strategies to inform drug development and drug commercialization and; is a keen contributor to scientific discussions pertaining to the challenges and/or opportunities offered to regulators, payers, academics, prescribers and to the bio-pharmaceutical industry by advances in computing science and the increased availability of real world data. He has been active in European-wide initiatives aimed at improving biomedical research (IMI PROTECT and EMIF) and is also the chair of the PSI Epidemiology Special Interest Group.
Michele Intorcia
Info Scientific Board

Michele Intorcia

HEOR Senior Director at Apellis Pharmaceuticals
Michele Intorcia is currently HEOR Senior Director at Apellis Pharmaceuticals. He holds a Master of Science in International Health Policy from London School of Economics and worked in Public Health and Academia. With 16 years experience in the Pharmaceutical Industry he formerly worked for Bristol Myers Squibb, Amgen and established the HEOR Team at Vifor Pharma. He focuses on the evidence generation and pharmacoeconomics strategies to secure access and reimbursement for innovative drugs.
Omar Ali
Info Speaker

Omar Ali

Visiting Lecturer Value Based Pricing at University of Portsmouth & Former Adviser to NICE
Qualified with a hospital pharmacy background, Omar has been working as the Formulary Advisor for Surrey & Sussex Healthcare NHS Trust for over 15 years, sitting on the regional Joint Drugs & Therapeutics Committee as well as the CCG/Commissioning Prescribing Clinical Network. Omar has been a visiting Lecturer at UCLH Pharmacy Programme and was both Lecturer & Examiner on the Independent Prescribing V300 Course at the University of Surrey and has over 30 publications to date. He has been appointed as Visiting Lecturer on Value Based Pricing & Innovative Contracting at the University of Portsmouth, is an Editorial Content Adviser to Guidelines, and has recently been invited to the position of Associate Editor to the Canadian Journal of Population Therapeutics & Clinical Pharmacology. Omar has served a position on the External Reference Group on Cost Impact Modelling for NICE for 6 years and was subsequently appointed Panel Member for the newly formed Adoption & Impact Program Reference for NICE. He advises foreign investors (US Embassy) on ‘Value Based Assessments (hosted by the UK Department of Trade & Industry) and recently delivered a Healthcare NHS Reform program to over 40 Healthcare Insurance Provider delegates visiting from the US. Last year he was invited to address the Italian Healthcare Senate [Senato della Repubblica] on Pharmaco-economic Evaluation & Sustainability Models of Healthcare and featured at the Westminster Health Forum Keynote Seminars [chaired by Lord Howe, All Party Parliamentary Group on Pharmacy] on the Future for Pharmacy Commissioning, Organisation & Delivery. At this current time he has embarked on a PhD with his doctorate thesis entitled “Value Based Pricing & Outcomes Based, Innovative Contracting of New Medicines”.
Raquel Billiones
Info Speaker

Raquel Billiones

Subject Matter Expert on Medical/Regulatory/Scientific Writing, Data Disclosure and Protection, Pharma/Medical Devices
Raquel Billiones has a PhD in Biology and more than 25 years combined experience in scientific and clinical research. She has been a medical writer for >14 years, with core competencies in writing clinical trial and regulatory documents for pharmaceuticals and medical devices. Her experience also includes transparency, disclosure and patient data protection in clinical trial data reporting. Previously, she served as Head of Medical Writing at Takeda Vaccines. She also worked for a mid-sized global CRO for many years where she served as Country Director of the CRO affiliate in Switzerland. In addition, she established and led a small team of CRO medical writers and support staff located in the US, Europe, and Asia. Raquel is an active member of EMWA, serving in various roles, including as Executive Committee member (2015-2017), associate editor of MEW (2010-present), internship advocate, workshop leader, and member of the Medical Device Special Interest Group (MD SIG).
Adrian Jonas
Info Speaker

Adrian Jonas

Associate Director for Data and Analytics at National Institute for Health and Care Excellence (NICE)
Adrian leads the central data and analytics team in the UK National Institute for Health and Care Excellence (NICE). This provides central oversight, advice and support on all aspects of data and analytics; spearheading the transformation programme to enable more sophisticated collection, management, storage and exploitation of data across NICE to enable better evidence based decisions across all its business. Adrian joined NICE in 2018 following ten+ years working in a variety of roles in central government including:
  • Assistant Director Strategic and Cross-Cutting Policy within HMRC
  • Principal Analyst for Home and Foreign Affairs (Prime Minister’s Implementation Unit) & Head of Profession for Operational Research within the Cabinet Office;
  • Head of Analysis and Management Information within the Foreign and Commonwealth Office.
Christian Müller
Info Speaker

Christian Müller

Teamleader Data Generation at Bayer
Christian is a consultant pharmacist for medical information. He holds a Diploma in pharmaceutical technology from University of Leipzig and PhD in Medical Sciences of Charité University Medicine of Berlin. After several years working in public pharmacies he joined Riemser Arzneimittel AG as Medical Advisor. He gained broad experiences in Medical Information, Medical Marketing, Clinical Operations and as Information officer. From 2012 to 2014 he has set up the German Operational Business Unit of LA-SER Analytica, a French CRO specialized in the management of pharmacoepidemiological studies. In 2014 he took over the responsibility of Non-Interventional Studies Team and in 2018 the responsibility of Data Generation Team of BAYER Vital GmbH in Leverkusen, Germany. In this position he drives digitalization and innovation in observational study conduct at BAYER.
Elena Peruzzi
Info Speaker

Elena Peruzzi

Evidence Generation & Data Analytics Head at Novartis
Biography available soon
Oriol Solà-Morales
Info Speaker

Oriol Solà-Morales

CEO at HITT - Health Innovation Technology Transfer
Biography available soon
Quote di iscrizione

€ 570,00* Early Bird fee until September 17th, 2020
€ 680,00* Ordinary fee
€ 365,00* Freelance, Academy, Public Administration

* for Italian companies: +22% VAT

Fee includes: access to the virtual conference, organizational support, certificate of attendance, slide presentations in pdf format provided post-event.

Edizioni Passate
2019
New Approaches to Drugs Value Demonstration in the age of Big Data: Anatomy of a Paradigm Shift
2018
From Volume to Value - The use of Real Word Evidence to improve Clinical Programs and Patients’ lives
2017
Real World Data and Pharmacoepidemiology in Europe: understanding the importance of RWD in today’s setting
2016
Observational Research and Pharmacoepidemiology in Europe: deeper insights to drive health research and discovery
2015
European Epidemiological Forum 2015
2014
European Epidemiological Forum 2014
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<p style="text-align: center;">Virtual conference with presentations, slots for Q&amp;A and discussion among delegates.<br />
<em>LS Academy will provide the link to join the conference some days before.</em></p>

Virtual conference with presentations, slots for Q&A and discussion among delegates.
LS Academy will provide the link to join the conference some days before.

<p style="text-align: center;">Virtual conference with presentations, slots for Q&amp;A and discussion among delegates.<br />
<em>LS Academy will provide the link to join the conference some days before.</em></p>