Clinical Trials In Rare Diseases Conference 2021

• Overview: key aspects of Lumevoq Gene Therapy development
• Regulatory pathways and challenges around documentation and working with regulatory authorities
• Recruiting and following up with patients in order to maximise patient numbers and minimise attrition throughout the study
• Key learnings and takeaways: what do you need to know when running a gene therapy study?

• APACs Rare Disease landscape
• APAC recruitment success for rare disease programs
• What to look for in a Rare Disease CRO partner
• Rare Disease program strategies

• Collate the available evidence base carefully
• Do your homework: Study similar trial cases to prevent any early obstacles
• Consider: How to manage progress with insufficient data from previous trials?
• Having a disease progression perspective is essential for to determine the right patient and time of intervention
• Recognising suitable vendors in advance to avoid disappointment

• Challenges with the traditional drug development paradigm in rare disease
• Use of external controls in clinical trials
• Sources of external control data
• Regulatory guidance
• Case studies

• Motivation for clinical biomarkers (use case)
• Technical expectations for a clinical biomarker
• Case study evaluating a fully validated assay for trial utility

• Why clinical teams are keeping and expanding their virtual engagement strategies
• How adopting a hybrid engagement strategy increases diversity, maximises patient engagement and results in deeper, more actionable insights
• Industry case studies: how clinical teams are using hybrid engagement to improve study design, training, and execution

We welcome Bonnie Bain from GlobalData to present her research into the state of the Biopharmaceutical Industry in 2021. Including:

• Identification of key themes that were triggered by COVID-19 and how they are shaping the biopharmaceutical industry – both today and into the future
• Key emerging technologies and disrupters
• Industry, regulatory and macro-economic factors affecting pharma in 2021

Don’t miss this talk; it is crucial for the biopharmaceutical industry to recognize the factors shaping the clinical trial landscape in order to maximize their future success.

• Assessing the benefits of repurposing over creating drugs from scratch
• What additional regulatory challenges are created when re-using an existing vs. a new drug to treat a rare disease?
• Sourcing funding and investment: financial implications of drug repurposing

Sessions resume at 9am US Eastern Time/3pm EU time

• Challenges of clinical research for rare diseases
• Opportunities and challenges around recruitment: how to ensure studies are as accessible as possible to patients with rare diseases
• Is retention and trial completion easier in a decentralized model?
• Home nursing options where complete remote monitoring is not possible
• Collecting data remotely and navigating hurdles around data and evidence for a study with a smaller number of patients

• Challenges to clinical trial participation for Rare Disease patients and families
• How DCT approaches can help to reduce participant and family burden
• Which DCT approaches may be most relevant in rare disease clinical studies
• How DCT elements can be implemented across the rare drug development pathway

• What suits your patients best? Designing a model that is flexible and appropriate for your patients
• Choosing a CRO who is comfortable with rare disease and orphan drug studies
• Creating your trial model: weighing up the costs and benefits of direct to patient, home nursing, fully remote monitoring and onsite visits
• Budget implications for a decentralized rare disease trial
• What are the priorities for patients participating in a rare disease study, and is it possible to make your study work for all patients?
• Assessing new technologies that are available to make your studies more efficient

MODERATOR:

Anka Ehrhardt, Science Director, Clinical Research, CHDI Foundation

PANELLISTS:

Karl-Johan Myren, Senior Director, Health Economics and Outcomes Research EMEAC, Alexion Pharmaceuticals

Steven E Cohen, M.D., Executive Director, Search & Evaluation in Specialty Medicine, Global Business Development, Translational Medicine in Specialty Medicine & Cancer Enterprise, Daiichi Sankyo

• APACs Rare Disease landscape
• APAC recruitment success for rare disease programs
• What to look for in a Rare Disease CRO partner
• Rare Disease program strategies

• How can we help patients mobilize to give a voice to their disease?
• Is decentralization the best solution to ensure accessibility to clinical trials, or do patients still want physical contact throughout studies?
• The importance of patient advocacy groups and listening to patient voices when designing your study
• What are the main barriers and burdens that prevent patients from participating in clinical trials, and how can pharma and biotech sponsors overcome these?

MODERATOR

Revati Tatake, Global Director of Databases and Analytics, GlobalData

PANELLISTS:

Jill Dolgin, Executive Director, Patient Advocacy, AGTC

Stephanie Kallay, Director, Patient Advocacy and Outreach, Crinetics Pharmaceuticals

Wendy Erler, Vice President, Global Head Patient Experience, STAR and Patient Advocacy, Alexion Pharmaceuticals

KimberLee Heidmann, Vice President, Patient Services, Scout Clinical

Ahmad Namvargolian, Chief Executive Officer and Co Founder, Care Access

• Challenges with the traditional drug development paradigm
• Current clinical trial landscape
• Including patient feedback and the patient perspective
• A toolkit for developing patient-focused clinical trials
• Outline for a 21st century clinical development paradigm

• Choosing vendors and partners: how to select the best CRO, CMO and site for your study
• Regulatory affairs and study design for a rare disease clinical trial: overcoming common challenges
• Simple methods optimizing your supply chain management
• Patient recruitment and engagement: how to maximize numbers of patients in your study

• Identifying enrollment challenges and opportunities of Rare Disease Studies
• Technology solutions that can create engagement
• How a unified platform and centralized data can streamline the process

Dawn Weagle, Associate Director, eClinical & Project Management, Axiom Real-Time Metrics

Brian Dempster, Senior Director, Global Clinical Management, Axiom Real-Time Metrics

• Recruitment: finding patients for your trial
• What extra factors should you take into account when recruitment and advertising is mostly online?
• Understanding attrition in a virtual setting: what to expect

• Landscape of clinical trials in rare diseases in the past 10 years
• Enrollment challenges for rare diseases clinical trials
• How has COVID-19 impacted clinical trials in rare diseases?
• Movement towards using decentralized trial model

Revati Tatake, Global Director of Databases and Analytics, GlobalData