Novel scientific techniques and technologies, including cell and gene therapies, mRNA, and AI/ML, offer exciting promise for improving treatment and outcomes, but require new thinking about regulatory and evidence generation frameworks. This talk will explore the policy perspective, looking at recent FDA moves and key areas for policy innovation, with a focus on data and evidence needs. It will include lessons learned on how researchers, technologists, and study sponsors can best engage with regulators to advance novel evidence generation methods and the breakthrough technologies that can improve patient care. 

As the industry hurtles down the path of digital transformation, the bulk of discussion has been on new tools and technologies and the interesting ways that the data produced from them can be analyzed, with occasional discussion of the need to develop data governance models. The data produced comes from human tissue – and yet there has been relatively no discussion about the development of human tissue policies and governance models. This presentation will examine the critical need for equivalent focus on development of policies governing the use of human tissue and recommended components of such a policy, to ensure that organizations position themselves for success. 

When companies share biospecimens and associated data with external entities, issues emerge of the conditions under which the materials and data were collected, and the regulatory frameworks of privacy that apply to sharing and downstream secondary uses of the associated data.   This session will explore the legal and ethical issues of provenance of human biospecimens and associated demographic phenotypic data, and how that provenance informs acceptable and ethical secondary use.

What is next after carefully implementing all the operational mechanisms to collect, process, annotate and store biospecimens? How to ensure biospecimens are utilized with legal and ethical and best practices?  Why would a governance structure for biospecimens be needed? When would this be beneficial? Navigating global regulations and internal expectation for research projects can be made easier with a few key principles regarding governance of utilizing biospecimens. 

The Cancer MoonshotSM Biobank (moonshotbiobank.cancer.gov) is a National Cancer Institute (NCI)-sponsored study that aims to accelerate cancer research through the collection of longitudinal blood and tissue biospecimens from cancer patients receiving standard of care therapy. The biospecimens, generally small biopsies, and accompanying medical data are being collected from community hospitals across US from diverse set of participant and are being made available to accelerate research progress in cancer. Evidence-based, well documented, and consistent procedures are being used to collect specimens of known quality. The Biobank will provide a networked biospecimen resource which will serve the goal of the Cancer Moonshot.

This presentation will discuss recent consent trends in the field of genomics in both the clinical and research settings. It will touch on lessons learned from the pandemic and look into the future when it comes to data sharing, interoperability, return of results and incidental findings and many more.

The Development path for cell therapies has distinct features as compared to historical therapies.  Early drug development is often focused on characterizing the Clinical Pharmacology of a drug therapy. In the world of cell therapies, many of these traditional investigations are quite unique.  For example, pharmacokinetics do not typically follow a first order enzymatic model.  Measuring therapeutic cells circulating in a patient requires methods to identify cells that have been engineered.  This talk will address the challenges with adequate and flexible sampling to support the development of cell therapies. 

CAR T cell therapies are one of the most exciting classes on immune therapies to reach the clinic in the last 20 years. Successfully developing a CAR T cell product requires a thoughtful clinical biomarker strategy that meets the needs of scientists, physicians, and regulators as they work to understand questions surrounding dose, safety, and efficacy. This presentation will share the factors that influenced our translational pharmacology approach for developing CRISPR-engineered allogeneic CAR T cells. I will discuss ways to improve speed, flexibility, and efficiency in the setting of dose escalation studies.

Patient-centric blood sampling, or the self-collection of samples remotely from clinical sites, has been increasingly adopted. This approach not only reduces patient burden and allows for more diverse participation in clinical trials but increases the efficiency and breadth of the trials themselves. Technology drives this shift, but the voice of the healthcare consumer has evolved and demanded this shift to remote participation, digital health devices, and convenient participation options.

The emergence of minimally invasive and patient friendly microsampling technologies can facilitate subject recruitment, improve retention, minimize blood volume and promote simplification of trial design and conduct. This talk will review utility of some of these technologies for clinical biomarkers and will cover the following key themes: Main drivers for biomarker patient centricity; key operational considerations for microsampling; and data interpretation and biomarker utility.