Real World Evidence (RWE) and Emerging Trends in Clinical Trial Subject Recruitment

May 3, 2024 @ 1:30 pm 4:00 pm

Hybrid: In-person (Boston) and Virtual (Zoom)

Meeting of The Research, Development, and Regulatory Roundtable (R3). Meetings are open to R3 Sponsors. To learn more about how to become an R3 Sponsor, click here.

Topics:

  • Recent Developments in FDA Guidance on Real World Evidence
  • Subject-Directed Recruitment Materials and Testimonials

Abstract: Real World Evidence and Emerging Trends in Clinical Trial Subject Recruitment

Real World Evidence

In recent years, real-world evidence (“RWE”) has increasingly been incorporated into product development programs, spurred by greater access to RWE, legislative changes, and significant policy development at FDA. The use of RWE raises novel opportunities, as well as new challenges for regulators, clinical trial sponsors, and the life sciences and healthcare industry to set common standards for RWE to ensure it can be relied upon with confidence in regulatory decision-making.  

During 2023, FDA released two draft and three final guidance documents concerning RWE.  These included the following:

  •  Considerations for the Design and Conduct of Externally Controlled Trials for Drug and Biological Products (Draft, February 2023)
  • Considerations for the Use of Real-World Data and Real-World Evidence to Support Regulatory Decision-Making for Drug and Biological Products (Final, August 2023)
  • Use of Real-World Evidence to Support Regulatory Decision-Marking for Medical Devices (Draft, December 2023)
  • Data Standards for Drug and Biological Product Submissions Containing Real World Data (Final, December 2023)
  • Real-World Data:  Assessing Registries to Support Regulatory Decision-Making for Drug and Biological Products (Final, December 2023) 

This session will provide an overview of key concepts from these guidance documents, focusing in particular on how they intersect with FDA’s existing regulations on clinical investigations and how they may change the regulatory landscape.  In addition to focusing on these recent FDA guidance documents, the speakers will address the application of other U.S. regulatory regimes to RWE activities, including the Common Rule, the Information Blocking Rule, and HIPAA and other data privacy laws.

Clinical Trial Subject Recruitment Trends

Clinical trial sponsors are increasingly exploring different techniques to attract potential subjects to clinical trials, particularly in the context of rare disease studies for which subjects may be difficult to find.  These techniques have included the following:

  •  Use of testimonials featuring active clinical trial subjects in clinical trial advertisements
  •  Inclusion of detailed scientific information gathered in pre-clinical or Phase 1 studies in subject-directed recruitment materials
  • Partnerships with electronic health record vendors to identify potential trial sites or subjects

Each of these techniques raise novel regulatory questions, including concerns about pre-approval promotion, data bias, need for institutional review board review and approval, and data privacy.  

This session will analyze these trends and the attendant regulatory considerations for each.

(1) Laboratory Developed Tests; (2) Limitations on Sharing Data with China and Other Countries of Concern

October 10, 2024 @ 1:00 pm 3:30 pm

Hybrid: In-person (Boston) and Virtual (Zoom)

This is a meeting of The Research, Development, and Regulatory Roundtable (R3) and is open to sponsors. Click here to learn more about the Research, Development, and Regulatory Roundtable (R3) and how to become a sponsor.

Topic: (1) Laboratory Developed Tests; (2) Limitations on Sharing Data with China and Other Countries of Concern

Expanded Access (Compassionate Use) for Drugs and Devices

March 22, 2024 @ 9:00 am 11:30 am

Hybrid: In-person (Boston) and Virtual (Zoom)

Topic: Expanded Access (Compassionate Use) for Drugs and Devices

Abstract:

The MRCT Center of Brigham and Women’s Hospital and Harvard and Ropes & Gray, LLP are pleased to announce the first meeting of the Research, Development, & Regulatory Roundtable (R3) in 2024. This session will focus on issues surrounding regulatory pathways for Expanded Access and/or Compassionate Use of drugs and devices.

While clinical research continues to yield medical advancements, there remain no approved therapies for a multitude of serious and life-threatening disorders. In some instances, however, investigational treatments show promise while being evaluated in clinical studies, prompting both patients and physicians to seek access to those investigational treatments, even though such treatments have yet to clear the relevant regulatory checkpoints to be brought to market. Several major markets around the globe, however, provide pathways for patients to receive these as-yet-unapproved investigational treatments outside the context of a clinical trial. Depending on the specific market in question, these regulatory pathways go by different names and necessitate different requirements for patients and their treating clinicians both before and after gaining access to investigational treatments.

In this session, the speakers will deliver prepared remarks to deconvolute these pathways – the key terminology, the relevant statutory and regulatory language, and the key differences between one program and the next. While the primary foci of the R3 will remain the U.S. and European markets at this session, references to programs in non-U.S. and non-European markets will also be included. As always, this session will occur under Chatham House Rule and attendees are thus encouraged to participate freely in discussions of specific, real-world challenges with our invited experts.

Research, Development, and Regulatory Roundtable (R3) meetings are open to sponsors. To learn more about R3 and how to become a sponsor, click here.

Update on the GDPR & Ensuring Integrity of Clinical Trial Data

December 13, 2023 @ 2:00 pm 4:30 pm

Hybrid: In-person (Boston) and Virtual (Zoom)

Topics: 1) Update on the GDPR, including discussion of the EU/US data privacy framework; 2) Considerations for ensuring accurate clinical trials data

OPEN TO THE PUBLIC

GDPR: This session will involve a panel discussion exploring developments occurring in the past year related to the effect of the European Union’s privacy law, the General Data Protection Regulation (“GDPR”), on clinical research. GDPR was the topic of a one-day meeting held in conjunction with the MRCT Center annual meeting in December 2022, and the intent of this panel is to explore what has changed in the intervening 12-month period. Topics to be addressed will include the Trans-Atlantic Data Privacy Framework, the UK-US Data Bridge, ongoing litigation in the EU regarding the definition of anonymization, and the latest relevant European Data Protection Board guidance. The panel will include representatives from leading life sciences companies who advise on data privacy issues as well as a representative of the U.S. Department of Commerce who worked on the Trans-Atlantic Data Privacy Framework.

Ensuring Integrity of Clinical Trial Data: Clinical trials are most often conducted at sites contracted to perform clinical trials. Historically, trials have most often been sited at organized clinics or hospitals, which have reliable systems of ensuring quality of care and creating and maintaining accurate medical records. Yet in recent years, clinical trials have been sited at stand-alone, for-profit research sites that lack the quality assurance infrastructure of clinics or even physician group practices; and in the wake of COVID, clinical trials have often become “remote” or “decentralized.” This session will explore the challenges in this new clinical trial environment to ensuring accurate recording and retention of data, and whether and how monitoring processes should be revised and enhanced.


This meeting is part of the MRCT Center’s 2023 Annual Meeting in Boston on December 13-14 and is open to all registrants at no cost. Guests are welcome to attend the entire program or individual sessions. While in-person attendance is encouraged, virtual participation is welcome.

Click here to register.

Click here for the full schedule and FAQs.

Deliverables

Part 2: The Regulation of Research Involving Cannabis

September 20, 2022 @ 2:00 pm 4:00 pm

Virtual Event

As more states move to legalize marijuana, the regulatory landscape is becoming increasingly complex. The number of sponsors and researchers inquiring about these regulations has also increased. Clinical research involving cannabis is subject to both federal and state regulations. Key regulatory frameworks include DEA regulations on the use of controlled substances in research studies, FDA IND regulations, and state law. Proposed federal legislation may also affect the conduct of research involving cannabis. David Peloquin, MRCT Center Senior Advisor and Partner at Ropes & Gray LLP, provided an overview of DEA and FDA regulations relating to research involving cannabis, reviewed recent legislative developments, and discussed state-level policies and challenges using New York state as an example.

Deliverables

Meeting Summary

Released on: September 20, 2022

Topics: 1) Legal and Regulatory Perspectives of For-Profit Research Sites, and 2) The Regulation of Research Involving Cannabis

Application of General Data Protection Regulations to Research: Legal, Practical, and Strategic Implications

July 30, 2018 @ 8:30 am 12:30 pm

Ropes & Gray, Boston, MA

800 Boylston Street
Boston, Massachusetts 02199 United States

Convening representatives from academia, industry, and government, this conference explored the impact of the EU GDPR on human subjects research, highlighting the challenges posed by the GDPR to clinical research, biobanking and databanking, and big data research, particularly in light of the limited guidance available to date from EU and EU member state authorities.

Deliverables

Meeting Summary

Released on: July 30, 2018

Topic: Application of the General Data Protection Regulation (GDPR) to Research: Legal, Practical and Strategic Implications

Expanded Access: Practical, Legal, and Regulatory Considerations

November 8, 2018 @ 8:30 am 4:00 pm

Ropes & Gray, Boston, MA

800 Boylston Street
Boston, Massachusetts 02199 United States

Attendees discussed the advantages and drawbacks of the two pathways by which patients may now request access to investigational therapies. Among other things, we considered the impact of each avenue on long-term drug development, the resources necessary for handling requests in a timely and consistent manner, and the optimal contents of organizational policies, considering the perspectives of both pharmaceutical companies and large academic healthcare facilities.

Deliverables

Meeting Summary

Released on: November 8, 2018

Topic: Right to Try vs. Expanded Access: Practical, Legal, and Regulatory Considerations.