Outsourcing in Clinical Trials West Coast 2020

Donald Kellerman, VP Clinical Development and Medical Affairs, Zosano Pharma

This session will look more proactively at the protocol design phase, thinking through the various elements and planning ahead to build in that design upfront

• Another level of complexity: Discussing the implementation of biomarkers into a protocol whilst remaining agile
• Identifying key aspects of an agile protocol which is moving towards a patient-centric approach, such as home visits and the patient voice
• Real life example of a modified protocol based on feedback of patients and other assessments to reduce patient burden
• Machine learning, predicting enrollment and smart analytics- Utilizing data and tech sources to design a flexible recruitment strategy

• Pinpointing the technology-based root cause of inefficiency with the current approach to clinical research
• Exploring the 3 waves of digital transformation in clinical research: making full use of advanced tech such as AI
• What industry change is required in order to adopt better to new technologies?
• Microsoft’s role in transforming clinical research including how to further meet patient needs and reduce burden

• Accessing Australia’s ecosystem for early phase trials. A case study
• Accelerating patient recruitment in Asia for large complex trials
• The advantage of running global trials with regional CRO partners

Outsourcing & Clinical Operations

Working with niche CROs allows our study to be a priority because it’s a bigger piece of their pie… is it this simple?

• Exploring different approaches to finding niche CROs to ensure your partner can meet your specific needs
• Looking into unique ways niche vendors approach standard procedures such as enrolment and retention
• Identifying the issues that arise from high turnover rates in smaller CROs and putting a contingency plan in place to manage that risk
• Highlighting the advantages of niche CROs focussing on a specific indication and therefore having outreach in that patient community

• Identifying challenges in aligning timelines between clinical operations, supply and data management departments
• Highlighting the importance of getting input from all stakeholders during the planning phase to ensure timelines are realistic
• Defining a communication framework which enables departments to effectively report on milestones reached or missed, changing trial parameters
• Providing examples of how breaking down silos enables creative problem-solving with unforeseen challenges

JOIN ONE OF OUR BRAND NEW NETWORKING TABLES

Eat lunch with a like-minded group of industry colleagues by joining one of our lunchtime discussion groups on the topics below:

TABLE 1: Discussing the application of new technologies, such as AI and Blockchain, to clinical trials

TABLE 2: Overcoming common small company pain points

TABLE 3: C-level discussion: How to make your resources stretch further and reduce time-to-market

TABLE 4: What does ‘patient centricity’ mean to you?

TABLE 5: Innovative ways of improving CRO engagement and communication

CRO selection and planning ahead for Phase II: With such a crowed CRO landscape how can we differentiate and make a choice?

• Evaluating how to best gauge chemistry and compatibility during CRO selection to ensure you are acquiring a long-term partner
• Impressing the importance of a CRO who has done their homework and develops a comprehensive pitch
• Developing strategies to enable your vendor to become an extension of your in-house team

Thinking longer term to your phase II or III study which may require a CRO with global capabilities- Do they have a presence in Asia or Europe?

• Exploring who negotiates the budget: Sponsor and/or site?
• Pinpointing procedural costs plus Investigator/Coordinator costs as well as overhead
• How do procedural costs compare with insurance company costs?
• How does this process compare with other financial transactions?

This experiential session addresses the importance of aligning due diligence expectations between biopharma companies and investors (i.e., sell and buy side). Dr. Brownell facilitates the workshop to make it as fun as it is informative, current and memorable.

Attendees will also receive a report via email of the key points plus a due diligence request from actual investors giving a specific 2020 update.

Fireside Chat: Navigating the

 launch of international trials

and study start up from a small companies’ perspective to examine next steps

Clinical Innovation & Technology

Maximizing the use of wearable technologies for real time data entry: A more patient-centric approach?

• Exploring the benefits of direct patient input with devices and how best to incorporate them into trials
• Examining facets of your protocol design to gain further patient input on technologies during the trial
• Addressing compliance requirements and current FDA standards
• Exploring home monitoring technologies for patient data collection in a more timely manner

• Explore the driving forces behind the industrywide move to streamline information exchange in clinical trials
• Learn about modern technologies that improve how organizations work together across the clinical environment
• Discover new approaches to sharing trial information with study partners
• Learn best practices for driving greater efficiency and collaboration

Improving health outcomes with a world-class transportation platform

• Leverage Uber Health to help with retention and recruitment for clinical trials
• How Uber Health helps our partners to better serve those they care for
• The power of Uber Health for our most underserved populations (the elderly, low-income, at-risk)

Why are Machine Learning and AI techniques gaining traction in the life science industry?

• Recognising the ability of AI to keep track of the data we don’t collect
• Harnessing the power of AI to identify the most suitable sites using electronic health records
• Smart Analytics- utilizing databases to identifying which sites are enrolling the most suitable patients for your study
• Considering what processes can be automated through technology and AI to minimize human error

• Building a technical foundation to underpin improved business performance
• Exploring platform modernization approaches on GSK’s foundational infrastructure to simplify the system landscape
• Other initiatives around how GSK uses technology as a means to innovate our approach to engaging patients and sites

• Discussing current monitoring and site oversight model (FSP)
• Why in a small diagnostic start-up the model is fluctuating?
• Explore the impact of resources on outsourcing model
• Pinpointing the role of Clinical Compliance in outsourcing

• This session will address the ongoing challenge of onboarding in clinical trials and how we can improve trial design and innovation by utilizing patient voices
  • Patient voices at the concept stage
  • Why are we afraid of using patients and how can we start to combat this?
  • No one size fits all when it comes to patient advocacy groups
  • Innovative methods and technologies to improve trial design

Patient Engagement

LIVE DEBATE

‘Patient recruitment is most successful when taking the grass roots approach compared to investing in social media’ Do you agree or disagree?

With more being spent on tech but only 50% of studies recruiting on time, should we be spending millions on social media/digital recruitment? Or should we go back to basics and focus on human contact?

• Is the grass roots method the best way to increase awareness and hit start up timelines?
• FOR- Nothing beats human contact: Exploring the effectiveness of forming physician relationships for effective referral
• AGAINST- Social Media is the future: Adopting social media to increase interest in a patient pool
• How patient recruitment success can be determined at the site by understanding patient flow
• Public awareness: How meaningful conversations with patients can encourage them to spread the word

• Ensuring objectives are synchronised by sharing protocol insights with patients
• Identifying strategies for translating patient experience into protocol design to enhance engagement
• Collaborating with KOLs in patient populations for study development and setting up advisory boards
• Best approaches for building an advisory board with the specialities required for your indication

Gaining patient and caregiver insights into protocol development to reduce burden and boost enrollment and retention

• Converting the emotional perspective to a data driven patient voice to all stakeholders
• Working with Patient Advocacy Groups early to develop lay language for your trial and reach out to wider populations
• Discussing how to get patients involved in the trial design and further promote the study
• Putting patients at the steering wheel by inviting them to join advisory boards and examine recruitment strategies

• Discussing how an understanding of your patient population enhances participation and recruitment from diverse groups
• Thinking outside the box about what cultural aspects come into play during the protocol design
• Outlining the most relevant population for your study to identify what minorities to include
• Establishing relationships with sites to align objections and discuss strategies to reach minorities

SPOTLIGHT

This session will include 3 x 15min presentations to discuss whether a Strategic Partnership, FSP or Fully Outsourced model is the best option. The presenters will give case study examples including pros and cons for each model. We will end with an audience Q&A.

 Presentation 1: A Strategic Partnership with a CRO allows you to find a likeminded partner and establish a strong relationship leading to greater satisfaction and more effective studies.

Tentatively Confirmed: Jacqueline Mardell, VP Clinical Operations, Ascendis Pharma

Presentation 2: Investigating the trend of moving towards an FSP Outsourced Model as a solution to develop a controlled, flexible study

Presentation 3: Making the strategic decision as a small/virtual company to rely solely on a trial partner by adopting the Fully Outsourced Model

• Discussing why basket studies are so important
• Assessing how sponsors can rely on the right CROs to run the platform umbrella
• Overall adaptive trials successful

• Justifying the importance of putting together a governance committee and meeting schedule to address upcoming milestones and risk management
• Monitoring CRO engagement using high level metrics on start-up aspects to draw up timelines goals and assess performance
• Focussing in on inspection readiness to ensure correct documentation on CRO oversight and governance plans

• Reviewing FDA and EMA regulatory guidance to ensure compliance
• Discussion of study design options to ensure success in Phase I trials
• Highlighting tips and lessons learned on efficient and effective execution

• New methods to incorporate Asian data into Western Phase I protocols for maximum speed and efficiency
• Latest regulatory environment for Chinse and Japanese Phase I data requirements
• What data is needed to allow Asian countries to join global trials. When and where can these data be collected?

• Exploring the rapidly changing standards of care to develop a more flexible study design
• Handling additional operational safety precautions associated with immuno-oncology to ensure patient safety
• Evaluating long term feasibility of immuno-oncology trials and true cost factors associated to assess long term impact for reimbursement issues
• Can working closely with cluster busters help to identify more potential patients?

• When leading, managing and collaborating with others, technical skills won’t do it alone. It takes Emotional Intelligence to do this successfully
• The four core competencies of Emotional Intelligence include: Self-awareness, self-management, social awareness, and relationship management
• Uncovering the secrets to having a successful relationship with your vendors

• Unlocking best practices for negotiating budgets with new vendors and aligning trial timelines
• Getting into the granularity of the budget to properly understand what you’re paying for each visit
• Appreciating the importance of accurately forecasting protocol design costs
• Assessing tools which can help you decide on budgets giving holistic numbers around procedure costs
• Best practices for implementing site and vendor monitoring to ensure the study stays within a strict budget

Interactive roundtable sessions offer a unique opportunity to come together with your peers to share best practice and develop solutions to critical challenges facing the industry as a whole. Hosted by industry experts and each focused on a single issue, roundtables are an exciting, interactive way to build your personal network and learn from the experience and expertise of others.

Each roundtable session lasts for 45 minutes, and delegates may attend up to 2 roundtables.

• Considering a substitute to the traditional placebo group with a Synthetic Control Arm that using pre-existing data
• Highlighting the advantages of placebo arms being modelled on information previously collected such as real-world data and historical control data
• Uncovering how this model could enhance patient interest and enrolment as all patients will get the experimental therapy

• Understanding what ML is and is not
• Use cases for Machine Learning in Clinical Content Management
• Case examples of where ML is best designed to benefit your operations
• Conducting Global Studies with Machine Learning
• The future of Machine Learning and Clinical Operations

When do you need it? When to outsource? And what should be or shouldn’t be in an eTMF?

• Exploring the current trends in streamlining clinical trials with eTMF
• Identifying what internal resources you have to manage eTMF and what can be outsourced
• Allocating a team member with internal authority to be the TMF owner to ensure inspection readiness

IdentiRitu Vermafying how eTMF can quicken study start-up to improve overall trial efficiency

• When it comes to innovation, healthcare presents a paradox- This field can be described as both a hare and a tortoise
• While life-altering breakthroughs in medical treatments can come at a rapid pace, improvements to the way healthcare is actually delivered happen very slowly
• New technologies have the ability to challenge this paradigm, but unique problems exist that don’t occur in any other industry

• Patients will be more empowered
• Trials will become decentralized
• Technology will support this decentralization: Discussing wearables and telemedicine

Strategies for effective trial design

and securing funding

• • Identifying the key barriers to funding application approval for small, medium, and large sized medical device companies
  • Comparing the different funding resources available to start-up companies including angel investors and venture capital firms
  • Understand how regulatory approval affects reimbursement rate
  • Revealing key techniques for medical device and biotechnology companies to approach angel investors

• Details to be confirmed

Getting FDA approval for your trial 

• Insight based on real-life examples of good and bad clinical trial design from the FDA perspective
• Explaining why prioritizing patient-centricity and safety is crucial for FDA approval
• Summarizing key updates to 510(k) clearance guidance
• Looking at the relationship between the FDA’s Quality System Regulations and ISO 9001 and best practices for meeting both sets of guidelines

CASE STUDY:  Investigational device exemption 101

• Breaking down Investigational Device Exemption and different types of IDEs to understand a premarket approval application
• Discovering how Early Feasibility Studies IDE has been a game changer for the US device industry
• An in-depth look at how to successfully transition from EFS IDE to Pivotal IDE
• Innovative approaches that medical device companies can take to get EFS IDE approval

• Advantages of BYOD compared to supplying devices including patient preference and usability
• Assessing what choices are available when considering your BYOD outsourcing partner to ensure suitability for your trial
• Tackling cyber security risks with your BYOD partner to ensure processes are in place if devices get lost/stolen

Considering compatibility aspects to ensure devices have similar operating systems depending on what functions you require

Do you outsource in the US or outside the US?

In this keynote session our host will introduce the topic with a 15min presentation, which will be followed by an interactive panel discussion with both panellists and the audience

• Thinking about which location offers the most suitable trial site for your medical technology
• Looking at the logistical benefits of doing site selection and patient recruitment outside the US
• Considering the European Regulations on MDR and IVDR and how it affects the US market
• Highlighting how European privacy law implicates patient data collection and comparing it to US requirements
• Comparing financial costs of domestic versus international clinical trials to be fully informed of which option is best for your study

• Digital Therapeutics overview (specifically in Alzheimer’s/Dementia)
• Digital therapeutics in a global market (i.e. what it means outside the USA)
• What is happening on the science side and the corporate side
• Issues with digital therapeutics in the global market
• How these issues can be solved to allow digital therapeutics to flourish globally