Real-world data, between ambition, reality and a path to reach its potential

Gracy Crane
Regulatory Chapter Lead & Global Regulatory Policy Lead (RWD), International Regulatory Policy Team, F. Hoffmann – La-Roche

The importance of real-world data and its relevance for the healthcare system has received increasing attention from stakeholders across the healthcare continuum. Its potential use to accelerate research and development, advance access to medicines, potential to making care more personalized for patients and clinical decision making more informed for HCPs has fueled innovation. New data platforms, methodologies as well as RWD guidelines and recommendations from regulators, think thanks, HTAs and companies are aimed at meeting the demand and provide guidance on best practice at the same time. During this session we will take a look at how the pharmaceutical industry is using RWD already to date and where we anticipate it being used moving forward. We will be putting these into the context of fitness for purpose of the various sources of RWD as well as other consideration around geographic and disease relevant factors. While RWD holds a lot of promise, we will also touch upon external stakeholder expectations and this way close the loop to review particular challenges in regards to quality, representativeness of data and methodologies in the area of precision oncology and rare diseases. We will take a look at some of the large existing databases in the area of oncology and will be closing the session with a look into the future of the European health data space.

Dr. Gracy Crane is a cancer biologist by training with a Master in Biomedical Research and a Ph.D. in Cancer Biology. She has also completed post-doctoral training fellowships at Oxford University (UK) and Massachusetts Institute of Technology (USA).

She has over 12 years of years of experience in the pharmaceutical industry, in varying roles including medical and scientific affairs, clinical development, health outcomes research and real world data. For the past 5 years, she has worked on leading the real world evidence strategy for molecules within the rare cancer space within the global Data Science team of the personalized health care department at Roche. Currently, she is the RWD policy lead within the Regulatory Policy team at Roche, and also works on RWD strategies across the product portfolio.

Gracy is part of a number of external steering/working groups including the ISPOR RWD transparency Initiative (https://www.ispor.org/strategic-initiatives/real-world-evidence/real-world-evidence-transparency-initiative); ISPOR-ISPE Special Joint Taskforce on harmonization of RWD protocols, and CIOMs working group on RWD/E guidelines.

The use of real-world data in clinical studies to support regulatory decisions.

David Brown
Acting Manager, Statistics & Pharmacokinetics Unit, Licensing Division, MHRA

There are vast amounts of data being collected on patients, for example, in electronic health records, and disease and patient registries. Such data is commonly called real-world data, reflecting that it is collected while patients go about their regular lives, as opposed to being specifically collected in a clinical study. While extensively used for monitoring the performance of medicines and devices after regulatory approval, RWD has traditionally been utilised much less frequently when it comes to demonstrating the safety and efficacy of an intervention. Use of such data sources for this purpose in clinical trials has the potential for increasing the speed and reducing the cost of development programmes, which would see effective medicines being approved more quickly, or even programmes which were previously thought to be unfeasible becoming feasible, with the consequent benefit to public health. The MHRA is producing a series of guidelines to provide general points to consider for sponsors planning to conduct clinical research using RWD to support regulatory decision making. This talk will introduce the recently published guidelines, discussing key points from the documents and issues raised in the consultation.

David Brown is the head of the Statistics team at the MHRA. He has over 20 years’ experience as a statistical assessor at the MHRA, and prior to Brexit was a member of both the biostatistics working party and scientific advice working party at the EMA. He was part of the group that formulated the recently published MHRA guidance on real-world data.

Choose between 1 of 3 breakout sessions.

Breakout 1) Turning RWD into RWE – from data acquisition to meeting stakeholder expectations. Gracy Crane

Breakout 2) Learning from the first horseman. COVID19 & RWE. Penny Ward

Breakout 3) PMST workshop. Surgery on expectations for competence- help & tips on how to navigate and display competencies

More information on Breakout 2) Learning from the first horseman. COVID19 & RWE.

This workshop will review the principles of observational research which enabled an understanding of the clinical impact and pathogenesis of COVID-19 and how these lead to the identification of potential therapeutics for hospitalised patients and the later development of vaccines and effective antiviral therapies to control community outbreaks. The lessons learned from this experience relevant to the potential prevention of NHS ‘winter pressures’ will be discussed.

Prof Penny Ward

Professor Penelope (Penny) Ward joined the industry in 1991 after working in the NHS for several years in general practice and hospital based obstetrics and gynaecology. Her career has spanned every aspect of pharmaceutical medicine, from early phase translational research to over-the-counter medicines, diagnostics and devices resulting in the approval and launch of more than 20 novel products supporting the diagnosis and treatment of infectious disease, cancer and inflammatory and autoimmune disorders in the EU, US and Japan. She is founder and Director of a private consulting firm, PWG Consulting (Biopharma) Ltd which supports emerging biotechnology companies to move their novel inventions from the bench into the clinic/into the market as well as supporting investors to take investment decisions. She chairs the Education and Standards Committee of the FPM and is a visiting Professor in Pharmaceutical Medicine at Kings College, London.

Application of RWE in Drug Safety and beyond

Prof Saad Shakir, Dr Debabrata Roy & Miss Samantha Lane
The Drug Safety Research Unit (DSRU)

Observational research is used to produce valuable and increasingly used evidence on medicines and vaccines under their real-world use. While initially observational studies were mostly designed to study the safety of these products, the use of observational research in pharmacoepidemiology has expanded progressively to also include effectiveness, outcomes research, and pharmacoeconomic studies. During the plenary session and the subsequent breakout session, we will provide an overview of observational pharmacoepidemiological research and its use in pharmacovigilance and drug safety, with examples. We will look at the study methods used in observational research, giving practical applications for each study design, and will compare results of observational research with the evidence generated from clinical trials. We will examine the data sources utilised in pharmacoepidemiology, comparing formal epidemiological study designs with the use of real-world data generated from drug and disease registries and electronic health records. We will consider the interpretation of observational data, including the roles of chance, bias, confounding, missing data, and misclassification. We will then consider the use of observational research for decision-making. We will review the evolution of observational research in pharmacoepidemiology, including the scope of the research, new methodologies, and integration with clinical trials.

Prof Saad Shakir

Director, Drug Safety Research Unit

Saad Shakir has been working in pharmacovigilance & pharmacoepidemiology for more than a decade.

He is Director of the Drug Safety Research Unit (DSRU) in Southampton.  The DSRU is an academic Unit associated with the University of Portsmouth.  Here he leads a research team with an active programme for monitoring and studying the safety of medicines.

He has worked and advised on many drug safety issues including product withdrawals and major safety hazards.  He is an author of many publications on pharmacovigilance and pharmacoepidemiology and is a member of the editorial board of the journals of Pharmacoepidemiology and Drug Safety.

Dr Debabrata Roy

Head of Epidemiology and Research, Drug Safety Research Unit

Debabrata is a Pharmacoepidemiologist and the Head of Epidemiology and Research at the DSRU. He has a biomedical science academic research background holding a BSc in Pharmacology (University College London), an MSc in Drug Discovery Skills (King’s College London) and completed his PhD in Neuroendocrinology (Imperial College London). He has trained in Epidemiology, gaining an MSc and in Pharmacovigilance gaining a PgD.

Debabrata conducts research on study design, methodology and the statistical analysis of Post-Authorisation Safety Studies (PASS) involving Modified-Prescription Event Monitoring (M-PEM), Specialist Cohort Event Monitoring (SCEM), Active Surveillance, Registries and Clinical Practice Research Datalink (CPRD) database studies.

Samantha Lane

Senior Research Fellow (Research and Innovation), Drug Safety Research Unit

Samantha is a Senior Research Fellow at the DSRU. She has a background in biomedical science and holds an MSc in Medical Microbiology. Samantha has also recently completed an MSc in Epidemiology through the London School of Hygiene and Tropical Medicine.

Samantha joined the DSRU in 2017, and is mainly involved in methodological research. She often assists with study design and data analysis, and has experience with the CPRD electronic health records database.

Choose between 1 of 3 breakout sessions.

Breakout 1) RWE and drug safety: methods, strengths and limitations. DSRU

Breakout 2) 7+ Habits of Highly Effective Pharmaceutical Physicians. Viraj Rajadhyaksha

Breakout 3) PMST workshop. Surgery on expectations for competence- help & tips on how to navigate and display competencies

More info on Breakout 2) 7+ Habits of Highly Effective Pharmaceutical Physicians. Viraj Rajadhyaksha

Medical Directors in the pharmaceutical industry are responsible for fulfilling multiple roles, including scientific expert, conscience keeper and people leader, as well as being the external face of the organisation.

The capabilities described in the book ‘Seven Habits of Highly Effective People’ are not just the integral qualities of a successful leader, they are important capabilities that are critical to becoming an impactful medical leader too.

As scientific, functional and enterprise leaders, country and cluster Medical Directors must possess these seven qualities. Each of the seven habits can be adapted and utilised to shape the specific areas that Medical Directors are responsible for: intuitive creativity, patient centricity, prioritisation, enterprise leadership, communication and behavioural change, building a team, partnering, and scientific and technological leadership.

To excel in their function, current and future Medical Directors need to sharpen their existing skills and build specific capabilities. Energy management in physical, mental, emotional and spiritual domains is the way forward.

This talk will be based on Viraj’s co-authored article: https://link.springer.com/article/10.1007/s40290-021-00403-x 

Dr Viraj Rajadhyaksha, MD, MS, DBM

Dr Viraj Rajadhyaksha has an MS in Pharmaceutical Medicine from University of Surrey, Guildford, UK. MD from Seth GS Medical College and King Edward VII Memorial Hospital, University of Mumbai (2002). Viraj is currently working as Area Medical Director Middle East and Africa AstraZeneca driving medical strategy across all therapy areas in the Area as well as develop medical talents. Previously he was the International (Emerging markets) Oncology Medical Director preceded by Medical Director- Malaysia, Singapore, Brunei across all therapy areas. Before that, Viraj led the Diabetes portfolio in AstraZeneca for Asia. Viraj has 17 years of experience as a pharmaceutical physician across Emerging countries in primary and leadership positions.