Conferences

"What does the general public not understand the most about clinical trials?" is the question we posed to three industry professionals. And their answers span only a few of the challenges that remain in patient education and knowledge about the clinical research enterprise. 

In this video, Kenneth Getz, Executive Director and Professor, Tufts Center for the Study of Drug Development and Founder, Board Chair, CISCRP; Janice Chang, CEO of TransCelerate BioPharma, and Greg Ball, Safety Data Scientist and founder, ASAP Process Consulting present their views on the topic. 

Videos

"What does the general public not understand the most about clinical trials?" is the question we posed to three industry professionals at DIA 2023.

Public Misunderstandings Around Clinical Trials

At this DIA 2023 session, “The Other Side of the Table: Drug Development Professionals Share Their Experiences Participating as Patients in Trials” the following participants from the industry spoke about their own journey in clinical trials: Peter DiBiaso, Senior Vice President, Clinical Solutions and Strategic Partnerships, WCG Clinical;Maria Florez, Research Consultant, Tufts Center for the Study of Drug Development; Sheila Mahoney Jewels, Drug Development Multi-Functionalist, LifeScieHub and Amber Whaley, Lead Regulatory Chair, WCG

The biggest issue that came the fore during the panel discussion was problems with informed consent.

Whaley explained that when she received the informed consent, she felt that people could be vulnerable to therapeutic misconceptions. Therapeutic misconceptions are inappropriate assumptions by research participants that decisions about their care are being made solely with their benefit in mind.

Also, Whaley said that trials should be as accessible as possible and should include the availability of alternative forms of consent. And, of the three consent forms she needed to sign for the COVID-19 trial she was participating in, only one was provided in advance. “There needs to be improvement there,” she said.

Florez, who was in a hybrid trial, with more decentralization than not for a one-year study, noted the eConsent was used remotely, but the jargon and overly long form and the language used should be something that that should be changed.

Jewels believes that part of the informed consent struggle is related to the fact that material gets “thrown” over to legal departments. She said, “If all legal departments were required to be in a clinical trial, we would not have the informed consent forms we have.”

As mentioned, Florez was in a primarily remote trial, and the interaction with the study staff was remote, as was the data entered regarding monitoring her own physical state was remotely into a system. As noted, she entered data remotely into the system, however, she found the interface or design was not easy or required too many log-ins.

From her vantage point at Tufts, she said it is found in their research that the site burden when dealing with patient calls regarding technology issue is difficult. “Some sites are having to create new positions to tech-support these patients,” she said.

Whaley needed to supply health questionnaires once a week, remotely. She had minimal technology issues as they loaded her personal iPhone to manage the data. But she wondered, “They didn’t provide phones, so could those participants without a phone participate?”

DiBiaso noted that follow up was poor and he never saw an outcome on the study he was in. He said, “There should be a level of transparency with the data.”

Florez agreed that it important for patient to have access to their data, however, she also thought it would be important ask the physicians if they want the patient to receive the data.

Whaley said that her being in a COVID trial was a unique case and with the large amounts of data that openly shared and covered in the media. While Whaley did not receive results and wasn’t sure if she received the therapy or placebo, participants were allowed to be unblinded if they wanted to get the vaccine to discover if they were able to get it or were already treated.

Final thoughts from the DiBiasio on thinking of participants means advanced moves such as challenging teams on exclusion/inclusion criteria and work toward less endpoints in the protocols. And then some simple acknowledgments, such as giving them thank you cards, or making sure they had ample parking.

As a close, an audience member suggested using the Gratitude Toolkit available from TransCelerate as a great resource to help provide that patient acknowledgment. 

https://www.transceleratebiopharmainc.com/assets/patientexperience/gratitude-toolkit/

REFERENCE: “The Other Side of the Table: Drug Development Professionals Share Their Experiences Participating as Patients in Trials”, DIA 2023, Boston. June 29, 2023.

Articles

DIA 2023 panel discussed personal experiences in clinical trials and offer suggestions for improvements to focus on the participants. 

When Clinical Trial Professionals Participate in a Study

In an interview at the 2023 DIA annual meeting with ACT editor, Andy Studna, Jaydev Thakkar, chief operating officer, Biofourmis, disucsses how Biofourmis got involved with CancerX and is looking to work on leveraging digital solutions to expand care access in oncology.

Jaydev Thakkar, chief operating officer, Biofourmis discusses how Biofourmis got involved with the CancerX Initiative at the 2023 DIA annual meeting in Boston, MA.

DIA 2023: How Did Biofourmis Get Involved with CancerX?

In an interview at the 2023 DIA annual meeting with ACT editor, Andy Studna, Jaydev Thakkar, chief operating officer, Biofourmis, disucsses how Biofourmis is working with CancerX to address the financial burden cancer treatment can place on patients.

Jaydev Thakkar, chief operating officer, Biofourmis discusses the work his organization is doing within the CancerX initiative at the 2023 DIA annual meeting in Boston, MA.

DIA 2023: What is Biofourmis Currently Working on with CancerX?

In an interview at the 2023 DIA annual meeting with ACT editor, Andy Studna, Jaydev Thakkar, chief operating officer, Biofourmis, gives an overview of the CancerX initiative and discusses how it was started. 

Jaydev Thakkar, chief operating officer, Biofourmis gives an overview of the CancerX initiative at the 2023 DIA annual meeting in Boston, MA.

DIA 2023: What is the CancerX Initiative?

In an interview at the 2023 DIA annual meeting with ACT editor, Andy Studna, Hilde Vanaken, PhD, head, European Forum GCP (EFGCP) eConsent Initiative, and head, TCS Industry Leaders gives an overview of the EFGCP eConsent Initiative and discusses how she started it and what it is currently working on. 

Hilde Vanaken, PhD, head, European Forum GCP (EFGCP) eConsent Initiative, and head, TCS Industry Leaders gives an overview of the EFGCP eConsent Initiative at the 2023 DIA annual meeting in Boston, MA.

DIA 2023: What is the European Forum GCP eConsent Initiative?

In an interview at the 2023 DIA annual meeting with ACT editor, Andy Studna, Hilde Vanaken, PhD, head, European Forum GCP (EFGCP) eConsent Initiative, and head, TCS Industry Leaders discusses what eConsent is all about and common misunderstandings surrounding it. She also touches on an article she recently authored for 

Hilde Vanaken, PhD, head, European Forum GCP (EFGCP) eConsent Initiative, and head, TCS Industry Leaders gives an overview of eConsent at the 2023 DIA annual meeting in Boston, MA.

DIA 2023: Understanding eConsent

In an interview at the 2023 DIA annual meeting with ACT editor, Andy Studna, Hilde Vanaken, PhD, head, European Forum GCP (EFGCP) eConsent Initiative, and head, TCS Industry Leaders discusses where industry is currently at with increasing adoption of eConsent as well as takeaways from a session she spoke at with FDA and MHRA.

Hilde Vanaken, PhD, head, European Forum GCP (EFGCP) eConsent Initiative, and head, TCS Industry Leaders discusses takeaways from a 2023 DIA session she spoke at on eConsent.

DIA 2023: Where Does Industry Currently Stand with eConsent?

Session chair Ryan Hoshi, PhD, MBA, MSc, Director, Regulatory Policy and Intelligence, AbbVie, opened the session laying the current complexities of medical devices, with the emergence of AI or device technologies themselves, and how the sponsors and regulators interact. The session brought forth many global barriers for both parties.

Melissa Torres, MS, Associate Director for International Affairs, CDRH, opened up explaining what 

International Medical Device Regulators Forum (IMDRF) 

actually is. She explained that IMDRF is “like ICH for the device side.”

IMDRF was established in October 2011, with the goal to accelerate international medical device regulatory harmonization and convergence. It includes 11 management committee members, as well as industry observers including WHO, Argentina, Suisse Medic, and an affiliate membership South Africa. The affiliate membership is for countries that want to participate, who’s regulatory sophistication in this area may not be high but they still want to engage in IMDRF.

Lesley Maloney, PharmD, Global Regulatory Policy Lead - Digital Health and Integrated Solutions, Genentech, A Member of the Roche Group, who was most recently with their diagnostics arm, described the key differences between IMDRF and ICH. “The key difference, and or criticism, is the level of engagement IMDRF has with industry. At ICH, it’s regulator and pharma together, and ICH is more mature,” she said. She says that IMDRF is just regulators, and not a co-creation of technical guidelines. She did concede that ICH has more resources, and training on their guidances, while the IMDRF does not, so it’s up to the regulators of each country to interpret the guidance.

Augusto Geyer, MSc, Head of Medical Devices Office, ANVISA, the Brazilian Health Regulatory Agency, noted that IMDRF is important to ANVISA. He explained that IMDRF provides the basis of for their regulations in Brazil, and their recommendations are also used by ANVISA to convince other regulators in South America to adopt ANVISA recommendations. He said, “In Brazil, most of the products are imported, so we realized a program with ANVISA instruction could be used on the registrations from other regulators, so idea is to have shortage of time for products to reach the market…at almost 35% to 40% faster.”

Of the many emerging technology products that are available, the audience was polled on the most impactful topic. They were, in order, combination products; Software as a Medical Device (SaMD); digital health technology in clinical trials; companion diagnostics; AI/ML and cybersecurity.

Daniel Yoon, Acting Manager, International Affairs, Medical Devices Directorate, Health Canada, Canada said that both the SaMD and cybersecurity working groups at IMDRF are co-chaired by Canada and the United States. He noted that the general principles and considerations in cybersecurity were released in 2020, along with two more in early 2023. One of these is cybersecurity considerations for legacy medical devices. Yoon said, “The ones on the market may not be up-to-date, wo how do you address these. As well as the software components in medical device and how to address that.”

Yoon also noted that there are four IMDRF documents on SaMD, all which were released between 2013 and 2017. The goal is to update those documents within 10 years, to address the many advances, and making sure they are updated and if the prior recommendations still “hold water.”

Maloney responded to a combination products audience question that there are less harmonized definitions for combination products globally, and then further from jurisdiction to jurisdiction. “How do we tackle? Whose purview is it? IMDRF or ICH? Device or drug? These are challenges that regulators have not pursued and we do need to get to a common definition of device and drugs in combination products.”

The greatest lament overall from the panel was this lack of harmonization from this rapidly changing are of devices. Some say that regulators need to be in the same room..the EMA and IDVR; there are conversations with CTTI about basic lexicon/terminology needs to be discussed, how do ICH and IMDRF interact and how to get industry more involved on the device side.

Yoon said, “It’s a lot easier when industry can be evolved. At IMDRF, closed working groups are for only regulators. Both of the cybersecurity and SaMD were open working groups so [industry] did participate in those documents.”

As for the FDA and IMDRF relationship, Torres said that they were able to get fives FTEs to show its accountability on how in FDA we have implemented IMDRF guidances. And then FDA will provide updates on implementation.

 International Medical Device Regulators Forum (IMDRF) and Technological Innovation: How International Medical Device Harmonization is Impacting Drug Strategy, DIA Boston, June 29, 2023.

Session at DIA discusses the International Medical Device Regulators Forum (IMDRF), its importance and how harmonization could be improved.


DIA 2023: Medical Device Regulators and Harmonization

In the FDA Rare Disease Town Hall at DIA 2023, Chair James Valentine, Director for Hyman, Phelps & McNamara, PC, laid the foundation for FDA rare disease therapeutic approvals and then introduced the FDA panelists for the session-- Celia M. Witten, MD, PhD, Deputy Director, Office of the Center Director, CBER, FDA, and Janet Maynard, MD, MHS, Director, Office of Rare Diseases, Pediatrics, Urologic & Reproductive Medicine.

Maynard began stating that of the 37 NME approvals for 2022, 20 were rare diseases. She updated the audience on the CDER-wide effort to connect the new drugs office, office of the Center Director and translational science. That is the 

Accelerating Rare disease Cures (ARC) Program

, which is now a year old. Maynard encouraged the audience to sign-up for its CDER Rare Disease enewsletter, which is now up to 10K subscribers.

Three notable initiatives in the rare disease space are:

Accelerating Access to Critical Therapies for ALS Act – ACT for ALS.

 This is a public-private partnership that requires, according to the website, “FDA to publish and implement a five-year action plan to foster drug development and facilitate access to investigational drugs for ALS and other rare neurodegenerative diseases.”

RDEA Rare Disease Endpoint Advancement (RDEA) Pilot Program

As part of PDUFA VII, the joint CBER, CDER pilot will develop novel endpoint efficacy development for drugs that treat rare diseases. Maynard encouraged sponsors to apply to the pilot, which will allow them to collaborate with the FDA in this effort.

The Learning and Education to Advance and Empower Rare Disease Drug Developers or LEADER 3D

, is a collaboration of stakeholders that will seek to understand challenges in developing these drugs, including the design of clinical trials, and where knowledge gaps. exist. The goal is to then create or expand educational resources for stakeholders.

Moving to CBER, Witten noted the general trend is cell and gene therapy development, away from the plasma-derived products, which were the primary therapeutics reviewed by CBER in prior years.

She said that the agency and industry has growing experience in CAR-T cells, and “although quite novel, the length of time for clinical development has decreased over the years because the [scientific] community has learned.” While the science has increased, she did note that the CMC processes have yet to catch up.

 has been launched to keep pace with clinical development. CBER will select nine proposals from sponsors to work with in this pilot with CBER. 

Witten said that a meeting of the Clinical Trials Patient Experience and 

Patient Engagement and Regenerative Medicine

 meeting was held in April 2023 and was very interesting. The FDA will issue a transcript of the recording soon. 

REFERENCE: FDA Rare Disease Town Hall, DIA Boston, June 28, 2023.

CDER and CBER leaders update initiatives in the rare disease space within the past year. 

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