Patient-centric data – Real-World Evidence (RWE) and Real-World Data (RWD) – are becoming increasingly instrumental in the drug development process and for making health care decisions. This type of data not only informs the process of discovery of new indications, but drug development and clinical trials design. It can also be of value to monitor post-marketing drug safety and enable decision support in clinical practice. It is not surprising that science companies and medical organizations are increasingly focused on leveraging RWD and RWE data to better understand patient population use of drugs and the respective outcomes (e.g. responders vs. non-responders), and to accelerate and provide justification for clinical decision support.

New technologies gather and store huge amounts of health-related data RWD can come from a number of sources, for example:

• Electronic health records (EHRs)
• Claims and billing activities
• Product and disease registries
• Patient-generated data including in home-use settings and patient reported outcomes surveys
• Data gathered from other sources, et. wearables data collected via mobile devices, that can inform on health status

RWE is becoming actionable when powered by analytics, machine learning & artificial intelligence (AI)
RWE provides insight beyond traditional clinical trial data, adding the potential to link data from different sources. This is expected to translate into improved trial efficiency, identification of potential new indications, and generation of a real-world perspective on risks/benefits to make informed decisions beyond traditional clinical trials. RWE is impacting the health care market and has also been getting attention on the regulatory side. AI and information exchange needs to be kept secure in the context of managing, accessing, and extracting insights from RWE.

The FDA has recently published guidelines for submitting RWE and RWD for Regulatory Decision-Making
The FDA has been exploring ways to further support industry use of RWE as a part of clinical studies under the agency’s RWE framework released and an industry guidance for how to submit documents that use Real-World Data and Real-World Evidence to the FDA for drugs and biologics in May 2019. The draft guidance supports a range of proposals, from engaging with patient groups early in the drug development process, to re-enrolling patients from early-phase trials into later-phase trials. The degree of impact from the proposed changes will depend upon the clarity in the final guidance and the FDA’s willingness to enforce and/or incentivize adoption of these requirements with sponsors and research programs. While some have argued that RWD/RWE need to be more supportive of enhanced diversity in trial populations and the number of patients needed for clinical studies, others have encouraged the FDA to consider how RWE / RWD may be used to help provide information on effects of the drug in these broader patient populations.

“Calling stakeholders from across health care to meet at PMWC 2020 to get involved in this critical program with the FDA to leverage RWD and RWE to improve clinical development”.

The framework is aimed at evaluating the potential use of RWE for assessing a product’s effectiveness and focuses on increased stakeholder engagement to effectively leverage RWD and RWE for public health purposes. The FDA believes that the RWE framework will serve as a road map for increased incorporation of RWD and RWE into the regulatory paradigm.

“RWD sets are a critical component in creating a learning system that allows us to modify and improve treatment strategies based on RWE.” – Chris Cournoyer, former CEO of N-of-One, now strategic advisor to QIAGEN and Chair to PMWC 2020’s Track 4: ‘Molecular Profiling – From Research to Clinic’

PMWC Jan. 21-24, 2020 Silicon Valley has several exciting sessions scheduled that focus on clinical RWE data, the FDA’s perspective on RWD and RWE, as well as other relevant aspects the regulators are actively involved in and that empower the advancement of precision medicine.

Talks of relevance include:

1. FDA Perspective – Strategies for Modernizing Clinical Development: Modernizing clinical trials is a priority for the FDA. To take advantage of technological innovations, the FDA is also seeking new ways to modernize its approaches to accommodate novel opportunities.
a. Contributing speakers include:

i. Session chair Sean Khozin, Director, Information Exchange and Data Transformation (INFORMED), FDA

ii. Malvina Eydelman, Director, Office of Ophthalmic, Anesthesia, Respiratory, ENT and Dental Devices, FDA

iii. James Tcheng, Professor of Medicine, Professor of Informatics, Duke University

iv. Michael Waters, SHIELD Team Lead & IVD RWE Representative, FDA

2. Leveraging RWE To Drive Decisions: This session will focus on the various aspects of integrating RWE and RWD to support drug development and clinical decision support.
a. Contributing speakers include:

i. Jonathan Sheldon, SVP, Bioinformatics Business Area, QIAGEN Digital Insights

ii. Laura J. van’t Veer, Inventor, MammaPrint; Professor of Laboratory Medicine, Dir. Applied Genomics, UCSF Helen Diller Family Comprehensive Cancer Center

iii. Christopher P. Boone, VP, Global Medical Epidemiology & Big Data Analysis, Pfizer

3. Other Track 4 Sessions of Relevance:
a. Adoption of Molecular Profiling in the Clinical Setting – Chair: Chris Cournoyer, QIAGEN Digital Insights

b. Early Detection Progress and Challenges – Chair: Anne-Renee Harman, GRAIL

c. Molecular Tumor Boards – Evolution of Function and Practical Utility – Chair: Anna Berry, Swedish Cancer Institute

d. NGS Workflows For Clinical Diagnostics Applications – Chair: Noam Shomron, Tel Aviv University

e. Navigating Secondary Use of Genomic Data – Chair: Jennifer Cubino, BC Platforms

f. New Approaches to Clinical Trial Design and Enrollment – Chair: Steven Shak, Genomic Health

g. Regulatory & Reimbursement Risk/Reward in Clinical CDx – Chair: Hannah Murphy, Coalition for 21 Century Medicine

h. Uncovering Data through Cancer Genomic Profiling – Chair: Ellen Maloff, My Gene Counsel

See what other timely and critical topics PMWC 2020 Silicon Valley program has to offer and how you can take advantage of early registration to secure your place at this unique scientific gathering focused on precision medicine. Rates go up tomorrow!