Welcome to the Duke CTSI Virtual Town Hall

January 25, 2017

Hosted by Ebony Boulware, MD, MPHDirector, Duke Clinical & Translational Science Institute

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Today’s topic: Perspectives on the future of clinical research: updates from Partnering for Cures 2016

Adrian Hernandez, MD - Director, Health Services Outcomes Research, DCRI

Jamie Roberts, Senior Project Manager, Clinical Trials Transformation Initiative (CTTI)

Bray Patrick-Lake, Director of Stakeholder Engagement, CTSI

What is…

Action tank determined to remove barriers to medical progress, whose one goal is to save lives by speeding up and improving the medical research system.

Annual conference convening leaders with the experience, creativity, and motivation needed to transform the medical research system and reduce the time and cost of getting new therapies from discovery to patients.

Key themes: We know that we need to get to faster cures…

• Create a culture of meaningful engagement with patients• Continue breaking down silos in order to facilitate collaboration and

data sharing• Enable the disruptive innovation that incentivizes novel models and

mechanisms

More than a seat at the table: Creating a culture of patient engagement

• More must be done to integrate patients into the fabric of the research • Cultural, institutional, regulatory, and privacy frameworks surrounding

patient data are prohibiting full patient engagement• Need to expand the existing set of decision-making tools to include

patient preferences, because they are not always reflected in the controls built into clinical trials• Trials need to be designed around patient needs

"Finding the right trial for the patient, rather than the right patient for the trial.”

Tools of Engagement: How patient-centricity can advance product development• Data collection does not equal partnership

• Partnership: never needing to ask “what would patients want” because they’re already in the room with you

• FDA is actively soliciting the patient voice• PROs may not define the efficacy of an agent but can define how to determine the efficacy of an agent

and for which patients• In RCTs, PROs can help establish what is: safety, efficacy, risk, benefit, based on the needs of the

community

• There’s a need to educate:• Regulators: importance of PROs• Payers: needs being met• Developers: needs unmet

• Engagement should be relational, not transactional• Start with the goal in mind, not the reasons it will be hard

Big Science: Does more mean a better future?

• Data interoperability and sharing are imperative• Funders need to require and help investigators to release data to the public, noting

that “your one dollar will go that much further if more eyes are on the data you just funded.”

• A “national data fabric” needs to be established in order to reduce costs associated with conducting trials.

• HHS final rule on trial reporting: Effective January 18, 2017, researchers are required to present summary data on ClinicalTrials.gov 12 months after collecting the last data point; absent that, they will lose NIH funding and must pay the FDA $10,000/day.

• Current issue in the healthcare system is that clinicians do not have the time to discuss clinical trial participation with patients, thus impeding large-scale and diverse clinical trial participation. We must create and incentivize a culture of research!

From Avalanche to Aha!Success Stories in Collecting Patient Data for Research• Data collection and sharing is not a technology problem, it’s a social

one• We must work harder to get data from the most vulnerable of us• Standards will develop when people start moving data around• Data sharing must become frictionless• Patients want a good product – we need to make the data useful and

delightful to them

R&D Remodel: Building trust & changing the culture in research

Barriers• Traditionalism (we’ve always done it this way), lack of data sharing

(between scientists, between scientists and patients), lack of diversity (in science and in participation)• Failure that is too punishing for investors and leads to risk aversion

and decreased innovation• Open sharing of data, reagents and resources requires a significant

investment and is the first thing cut when budgets get tight• Impact mindset rather than an incremental progress mindset

Dealing with Megawatt Scientists: Big Talkers, Control Freaks, and DemigodsA conversation with Dr. Francis Collins• When building teams and “Partnering for Cures,” we can’t ignore personality • ‘Do your homework’ and get to know your research colleagues personally so that

you’re comfortable making proposals. • Scientists get involved in research to do good, augment knowledge, and help people –

ultimately, this greater good is more powerful than individual ego, but the culture of science and research needs to recognize this

• The goal of mentoring is not for a trainee to be a clone of the mentor, but for the mentor to communicate with the trainee and keep the trainee’s interests in mind

• We need more diversity in the scientific world – he called upon the field to bring in more women and minority groups. Teams are more productive when they’re diverse, and future leaders should be those individuals well-versed in all interests and backgrounds.

Backing the next breakthrough: New models for investing in biomedicine• Funding is constant but the # of investigators applying has dramatically increased, so

fewer resources/investigator are available• Most cash flows to a small number of companies at later stages in drug development• Unique novel therapies won’t make it into real-world clinical practice if all funding and

focus is going toward established drug classes and drug franchises; e.g. diabetes and obesity

• New models are blended• Nonprofit organizations such as philanthropies, universities and research institutes are

pursuing financing models that will help sustain research interests without continuous fundraising

• Vison of the future: universities will increasingly acquire internal capabilities for early-stage drug development and commercialization

Backing the next breakthrough: New models for investing in biomedicineOn obstacles in funding in the life sciences:

• Peter Schultz, PhD (Scripps): This system is unfortunately dis-incentivizing the best young scientists. This is a challenge, and why we need to create new models for sustainable research funding

• David Steinberg (PureTech): Quantitatively, funding has picked up in recent years. Commercial models are becoming important at earlier stages. Therefore, just having great science at the early stage isn’t going to be enough to differentiate one idea from the next

On ways to hasten the drug development and approval timeline: • Schultz: CT design is critical to timeline acceleration. As we learn more about biology basic

researchers want to have an impact on medicine. But they may not understand the best way to prove the clinical utility of a product. Looping clinicians in is very important for this translation and the design of CTs

Adaptive Trials, Platform Trials, Master Protocols and Beyond: Pathways to Transformation - or Not?• Much interest in and excitement about the potential of innovative trial designs, such as

adaptive trials and platform trials, to make the long and expensive CT process more efficient and effective.

• FDA likes several things about such trials:• Patient focus, ability to screen patients by genomic profile and ability to make trials more accessible to patients. • Opportunity to ask multiple questions and test multiple treatments in the same trial.• Ability to scale, enhance quality with reliable results

• Design a trial around patients with a disease and not around a specific product – more efficient and better for patients

• LungMAP platform: 700-site network (half the patients enrolled at nonacademic medical centers) studying multiple drugs in multiple sub-studies, over a single trial meant to answer a single question• Must ensure all stakeholder voices involved in network development• Industry: Trial designs must have statistical significance that fulfill regulatory requirements• FDA was critical in providing counsel on that score and laying out the path forward.

Mixing Policy and Politics: Opportunities and Challenges for our President-Elect• Trump’s healthcare policies were not laid out in great detail during his campaign

• Former FDA Commissioner Dr. Andrew von Eschenbach interpreted this to mean that his administration will be open to innovative ideas that will create opportunities for investment in healthcare that also benefit financial markets.

• There should be careful consideration and engagement with all stakeholders before the new administration acts.• The administration should be clear and consistent when communicating policy changes.

• Need continued support for research funding at the NIH and the FDA user fee agreements that were negotiated with, and strongly supported by, the industry throughout 2016. These fee agreements will need to be enacted into law in 2017.

• Need to recognize the global nature of the biomedical innovation system, both in basic science and in the biopharmaceutical industry

• The 21st Century Cures Act aims to accelerate US biomedical innovation

NIH Collaboratory, Jan 16, 2016

https://www.nihcollaboratory.org/Pages/GR-Slides-01-06-17.pdf

What Can We (at Duke) Do?

We the People need to …• Engage patients as a driving force in improving care and also for the

research enterprise• Establish a collaboration framework built on trust and sharing first –

pay it forward by sharing data • Embed and invest in engagement across the research spectrum from

the beginning• Just Do It!• “We’ve been talking about this for awhile… It’s time to get married.”

Sharon Terry

Duke CTSI Virtual Town Hall – January 2017

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Virtual Town Hall January 2017