Workshop title – “R WE Ready?” – “Real World Evidence” Methods!

Organisers – Co-organised by Organisation of Pharmaceutical Producers of India (OPPI) and ISPOR India-Mumbai Chapter
Objectives – To enhance skill set of the professionals of the medical affairs community in area of real world evidence generation to create patient value
Date – June 23, 2017 with ISPOR India-Mumbai Chapter
Venue – Imperial Hall, Hotel St. Regis, Lower Parel, Mumbai

Workshop Agenda

  • Real world evidence: what it is, how it is important for medical department, developing a standard framework to help generate RWE
  • Health economics and outcomes research studies and its methodology; how HEOR studies can help in clinical decision making and shaping policy
  • Regulatory aspects: Current regulations governing RWE generation in India and regulated countries.

Mr. Sanjeev Navangul
Keynote speaker, Vice President, OPPI; Chair, Medical & Regulatory Work Group; Managing Director, Janseen India, J&J

“Every drug that is discovered and launched in the market is a promise based on the evidence obtained from a structured clinical trial program”


  • Effort to deliver the real world evidence (RWE) is far more crucial than clinical trials
  • More the real world evidence, more closer we go to the promise we make for better healthcare delivery
  • Health Economics is a much more important topic for countries like India that are self-pay (out of pocket) markets than countries where reimbursed markets exist

Mr. Navangul welcomed the audience while expressing his views on the importance of generation of real world data and the healthcare professionals (HCPs) must constantly strive to deliver the real world data, dealing with the ‘real’ patients, since the clinical trial data may not suffice in the real world clinical practice. Also, in India, where the healthcare services are paid for out-of-pocket, incorporating health economics is the need of the hour, which will justify the ‘value’ of service and not just the price.

Dr. Suresh Menon
Keynote speaker, Co-Chair, Medical & Regulatory Work Group, OPPI; Chief Scientific Officer, Novartis

“Huge variability in the label approvals and the clinician’s use (prescriptions) in real world practice”


  • His own experience with the real-world evidence data from a global study by Novartis
  • Difference between RCTs and real world evidence

Dr. Menon set the context for the session by giving an example of a global pivotal study and how they experienced the variability in the approved label and the real world data. He also opined on the differences between RCTs and RWE and importance of meaningful RWE.

Mr. Desmond Lim (Special Invitee)
Business Lead, Connected Health & Innovation, Asia-Pacific, Quintiles IMS, Singapore

“Digitization of patient data for easy healthcare delivery”


  • Some insights on problems faced by patients in clinical trials
  • Ways to regulate clinical processes
  • An app for sites and patients in order for them to enter their own data and accelerate easy access and data collection- This is not quite prevalent in Asia

Mr. Lim stressed on the importance of digitization of patient data by means of patient/user-friendly apps. Such apps are the future of healthcare and they’re expected to encourage electronic data collection, thereby facilitating easy healthcare delivery by deploying simple technologies. This use of technology can assure data reliability and quality, save time and also be cost-effective in the long run.

Dr. Viraj Suvarna [Chairperson: Real world evidence methods]
Medical Director, Boehringer Ingelheim

“Need of professionals (with both personal and professional traits) and ‘heart’ workers who’ll put their heart in their work”


  • Competitive collaboration to facilitate RWE studies
  • Hybrid studies

Dr. Suvarna headed the first session and while he started the session, he spoke about the collaborative studies where the doctors will perform RWE studies along with RCTs to know the patient responses/ profiles. He used the term ‘hybrid studies’ or ‘large simple trials’ which follow after efficacy, effectiveness, phase IIB stages of a clinical trial and incorporate real world data.

Ms. Richa Goyal
Senior Manager, Real World Insights (RWI), Quintiles IMS

“Big data is getting bigger and bigger!”


  • Scope and applications and source of RWE
  • What is RWE?
  • RCT+RWE: Strong evidence
  • Evidence platforms

Ms. Goyal started the session by giving a brief of scope, sources and applications of RWE. It’s a well known fact that RCTs are the gold standard for evidence. Ms. Goyal opined that, when RCTs and RWEs are done together, they can strengthen the evidence. “Data is the new oil”; meaning, the vast and humongous healthcare data is still being discovered and if it is channelized and harnessed properly, it can entirely change the face of all the associated stakeholders. Furthermore, she also spoke about various RWE data sources which may include HCPs, electronic medical records (EMRs), registries, pharmacies, social media, lab data, and so on. Companies can collaboratively work on channelizing and harnessing the available data and put it to maximum use by using analytical tools, publications, patient analytics, consultations, KOL engagement or own in-built databases. She further mentioned that RWE cuts through all stages of clinical drug development till drug delivery and it can be analyzed in parallel with the clinical trial. In RWE, payers and providers work together to generate substantial evidence beneficial for the patients. Ms. Goyal further pointed out the key challenges for conducting RWE studies, viz. no set guidelines, no transparency or reproducibility and limitations of bias. She also explained the 3 A’s of the drug for patients: Approvals, Accessibility, and Affordability. To add, not only primary but secondary data are also important in RWE, viz. assumptions, modeling studies, budget impact analysis, systematic reviews and meta-analyses, HTAs and so on. In the end, Ms. Goyal also threw some light on the position of RWE in India along with the current challenges and probable solutions to overcome those challenges.

Ms. Amita Bhave
Head- Regulatory Affairs Global Drug Development, Novartis

“RWE is important in regulatory decision making”


  • RWE guidelines and regulatory scenario around RWE research in regulated countries
  • Current position of RWE in the western regulated markets
  • Importance of RWE in decision making

Ms. Bhave shared some insights on the status of RWE in the regulated countries, e.g. US (USFDA), Europe (EMA). These countries have some regulations in place for the evidence from real world (E.g. PDUFA- Prescription drug user-free drug act by USFDA for RWE activities, 6th version of this act to be enacted in October, 2017). In the near future, USFDA will be conducting pilot studies and publishing guidance on how RWE can prove the safety and effectiveness in regulatory submissions or can contribute in the decision making processes. Additionally, Cures Act is in place which will be drafting a framework for RWE activities. Furthermore, USFDA will be conducting RWE workshops that would discuss the role of RWE in decision making along with the ways to overcome challenges faced by RWE activities as of now and bridging the gaps in data collection. EMA also conducted two workshops recently viz. patient registry workshop and regulatory data workshop – both focusing on the post-authorization area. EMA also describes a lot of adaptive pathways in regards with the RWE data. Ms Bhave also highlighted the outcomes of the Big Data Workshop by EMA and possibilities of regulatory tools for analysis of RWE data in order to incorporate it in the decision making, thereby promoting the use of RWE data. Although, very little data available on the RWE regulations in UK or Japan and challenges still exist with respect to its use in regulatory approvals or decision making processes.

Dr. C S Pramesh
Chief, Thoracic Surgery Professor, Dep’t of Surgical Oncology, Tata Memorial Center

“Population based research has great potential to reduce mortality in cancer or for that matter, any disease”


  • How RWE can be used in decision making processes
  • “Getting the right treatment to the right patient at the right time”

Dr. Pramesh, who is an eminent name in the current cancer research in Mumbai, opined on some advantages as well as disadvantages of RCTs. Reasons being, 1) RCTs are not generalizable as they have eligibility-inclusion/exclusion criteria; 2) they are not large enough to identify everything to be identified about a new treatment that is going to be launched in the market; 3) RCTs lack external validity; 4) They ignore variability in care delivery. He further shared the initiatives implemented by Tata Memorial Center (TMC) like, 1) National Tumor Board that fosters anonymized data sharing on a secure web-based platform in order to reach out to cancer patients and providing easy care; or 2) Navya which is a service for cancer patients to obtain second opinion. Dr Pramesh even shared some case studies where RWE has been implemented in the research that has shown noticeable results. He also opined that about 2/3rd of the patient population may not benefit out of the treatment which it is actually labeled for in the real world practice.

Dr. Jitender Sharma
Founder and CEO, Andhra MedTech Zone (AMTZ)


  • RWE in addressing the efficacy and effectiveness gap
  • Use of HTA and related tools

Dr. Jitendar Sharma addressed the importance of promoting RWE in India by way of looking after the efficacy and effectiveness gap. He suggested ways of doing so with the help of HTA and budget impact analysis. He further opined that evidence can be too logical and one must go beyond the logic and prove the efficacy or feasibility of a drug/treatment for the patients, which is the general concept behind ‘value-based medicine’.

Panel Discussion – I

The panel discussed at length about the need for RWE in the Indian scenario and the challenges that could be faced by while implementing it. The panel unanimously opined that data sources could be a big challenge; therefore data collection should be done in an organized manner. In case of execution, the challenges exist with regards to the lack of awareness about designing a RWE protocol among the industry professionals. Value-based medicine is a new term and it needs to be promoted. At the same time, affordability of the treatment is necessary. While the external regulatory environment is quite strong, the same needs to be available in India to adapt to the RWE world and also to bring the RWE into practice. To add to it, a structured framework and capability building are also need to be in place in order to overcome challenges. More and more companies are considering patient support programs or disease/wellness management program to gain data directly from patients in order to analyze it and use it as evidence. Furthermore, lack of Indian data can be attributed to the lack of logistics and to tackle this, safe data sharing on a secure platform needs to be promoted along with tools that will be comprehensible for patients. Also, equipping the HCPs to use the language that patients would understand will also be helpful.

Ms. Suneela Thatte [Chairperson: Health Economics and Outcomes Research]
Co-Chair, Medical & Regulatory Work Group, OPPI; Vice President, Global Operations, Quintiles IMS, India

“Similarity between RCTs and RWE”


  • Supporting the drug to be launched in the market having RCT data with RWE, making it available to the end-user

Ms. Thatte, while setting off the context for session 2, stressed on the importance of merging RWE data along with that of RCT in the drug development process itself. This will make the drug to strongly hold the place in market, along with the data from real world patients (beyond the eligibility or inclusion/exclusion criteria of an RCT).

Mr. Mahendra Rai
President-Elect, ISPOR Mumbai; Delivery-head, Market Access, HEOR, PRMA, RWE), Tata Consultancy Services

“The guidelines for RWE are in place and being reviewed’”


  • An overview of health economics and outcomes research
  • Introduction to healthcare costs and analysis
  • HTA in India

Mr. Rai introduced the audience to the concepts of health economics, healthcare costs and healthcare perspectives that are a part of health economic evaluations. The costs and perspectives form an important part of any economic evaluation.

Dr. Amit Dang
President- ISPOR Mumbai; Founder and CEO, MarksMan Healthcare Solutions LLP

“Though patient involvement is necessary today, ‘over-empowerment’ of patients can be harmful for them”


  • Comparative effectiveness research (CER) – Methods and applications
  • An Indian perspective towards CER at a very nascent stage

Dr. Dang stressed on the importance of patient centeredness and also threw some light on patient preference studies/patient centric clinical trials/patient advocacy. This kind of patient involvement is the future of Indian clinical trials, where patients will be involved right from the protocol design stage. He further added that there needs to be a constant flow of information from physicians. CER compares the effectiveness of two or more drugs or medical devices/services; however, it involves patient centric healthcare. HCPs need to keep up with the pace and keep themselves updates because today’s patients can easily access the internet and vast information available online. Dr Dang further explained about the stakeholders (patients, payer, provider, government and pharma industry) and their role in the RWE area. He also explained the steps involved in the CER, viz. gap identification, evidence generation by means of pragmatic studies (more observational studies), and evidence synthesis with the help of systematic literature reviews and meta-analyses. Furthermore, Dr Dang discussed about the impact of CER on RWE.

Panel Discussion – II

The panel discussed about ways to adopt for the development of RWE framework. The panel members unanimously agreed upon the need for involving the regulatory bodies and government to ease out the process of using RWE studies as constructive evidence. More epidemiological studies are required to generate RWE data. Monitoring the recent trends during data collection is also a necessary step in order to utilize those during drug development phase. The panel also discussed about the role of academia in development of RWE framework. Finally, a couple of important suggestions came up during the panel discussion that could possibly form some sort of a basis for the RWE framework. One option is to build a narrative to submit to the government in order to supplement the evidence generated from RCTs with the RWE data. Second approach is to make policy for rare diseases, like the one launched for cancer; which will definitely be of some help in using the real world data in the clinical setting.

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