Clinical Trials In Rare Diseases Conference 2021
DAY 1|14th September 2021
8:58 am Chair’s opening remarks
9:00 am Rare disease in clinical development: learnings from development of Lumevoq Gene Therapy in Leber Hereditary Optic Neuropathy (LHON)
- 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?
Chief Medical Officer
9:30 am Featured sponsor presentation
10:00 am 5 considerations needed for a successful clinical trial in rare diseases
- Do your homework: Study similar trial cases to prevent any early obstacles
- How to manage progress with insufficient data from previous trials?
- Having a medical directory is essential for to determine the right patient
- Recognising suitable vendors in advance to avoid disappointment
VP of Clinical Development
10:30 am Available for sponsor
11:00 am Is this biomarker ready for a trial?
- Motivation for clinical biomarkers (use case)
- Technical expectations for a clinical biomarker
- Case study evaluating a fully validated assay for trial utility
Science Director, Clinical Research
11:30 am Available for sponsor
12:00 pm Embracing disruption in pharma: is COVID-19 a catalyst for lasting change?
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.
Global Head and Executive Vice President of Healthcare Operations and Strategy
12:30 pm Available for sponsor
1:00 pm CASE STUDY: The ADNP Syndrome: drug development to treat rare diseases
- 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
Chief Science Officer
1:30 pm BREAK – VISIT EXHIBITION HALL Sessions resume at 9am US Eastern Time/3pm EU time
US – TIMES IN US EASTERN TIME
8:55 am Chair’s opening remarks
9:00 am Running a decentralized trial for a rare disease study
- 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
Vice President, Patient Affairs and Advocacy
9:30 am Featured sponsor presentation
10:00 am PANEL DISCUSSION: Post COVID-19: Incorporating flexibility into rare disease clinical trials from the beginning
- 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
Anka Ehrhardt, Science Director, Clinical Research, CHDI Foundation
Karl-Johan Myren, Senior Director, Health Economics and Outcomes Research EMEAC, Alexion Pharmaceuticals
Steven D Cohen, M.D., Executive Director, Search & Evaluation in Specialty Medicine, Global Business Development, Translational Medicine in Specialty Medicine & Cancer Enterprise, Daiichi Sankyo
Senior representative, Healx
10:45 am Available for sponsor
11:15 am CASE STUDY: Patient recruitment and retention in a rare disease setting
- 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
- Engagement: maintaining human contact even in a decentralized trial
Director, Patient Enrollment
11:45 am Available for sponsor
12:15 pm Strategies for optimization of clinical trial preparation
- 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
Executive Director, Clinical Research Unit (CRU)
Montreal Neurological Institute
12:45 pm Available for sponsor
1:15 pm PANEL DISCUSSION: Working effectively with patients and advocacy groups to ensure your study is accessible and of maximum benefit to patients
- 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?
Senior representative, GlobalData
Jill Dolgin, Executive Director, Patient Advocacy, AGTC
Stephanie Kallay, Director, Patient Advocacy and Outreach, Crinetics Pharmaceuticals
2:00 pm Available for sponsor
2:30 pm Real world data and the utilization to tell stories to payers in rare diseases
- Limitations and challenges when collecting real world data for rare diseases and methods to overcome these
- Using a combination of data from different sources in order to improve reliability and accuracy during a rare disease study
Chief Executive Officer
3:00 pm END OF CONFERENCE
To enquire about sponsorship opportunities for the conference, please contact:
Portfolio Director – OCT EMEA and Pharma Packaging & Labelling Global
Arena International Events Group
T: +44 207 936 6690
To enquire about speaking opportunities for the conference, please contact:
ZOOM PHONE: +44 (0) 207 661 0906