Clinical Trials in Rare Diseases - Virtual Event
Virtual Event: Day 1, 9:00 GMT – Day 2, 9:00 EST
7th-8th December, 2020
Clinical Trials in Rare Diseases – Virtual Event
DAY 1|7th December 2020
DAY 2|8th December 2020
9:00 am OPENING KEYNOTE: Assessing the impact of COVID-19 on rare disease clinical trials and the outlook for 2021
- The impact of COVID-19 on simplifying clinical studies: are new study methods more patient-friendly?
- Methods to keep a trial running throughout the pandemic: home nursing, remote monitoring, decentralization and more
- Solutions to improve patient recruitment in the face of lockdowns and quarantines

Mimi Choon-Quinones
Founder and Chairman
Partners For Patients, PfP.NGO
9:20 am Live Q&A with Mimi Choon-Quinones

Mimi Choon-Quinones
Founder and Chairman
Partners For Patients, PfP.NGO
9:30 am The Importance of the Patient Perspective for Rare Disease Drug Development
The drug development process for new therapies for rare diseases faces enormous challenges. Efficiency is key to bringing novel therapies to market as soon as possible. Understanding the patient perspective for delivering optimal experiences in rare disease populations is critical for improving patient retention and trial completions. In this session, hear why it’s so important to include the perspectives of patients with rare disease when developing clinical trials. These perspectives include: The unique burden patients with rare diseases face in clinical trials Understanding the importance data plays for the patient in the trial Why the return of clinical study data to these these patients is so important In addition, learn how Medidata’s Patient Design Studios are utilizing the patient voice to make improvements in solution designs and are uncovering potential issues before they impact trials.

Alicia Staley
Senior Director, Patient Engagement
Medidata

Dave Bjork
Director of Community Relations
FRAXA Research Foundation
9:50 am Live Q&A with Medidata
10:00 am ROUNDTABLE: Outsourcing challenges: finding a CRO best suited for your rare disease study
- Weighing up the benefits of a small, specialized CRO vs a larger CRO
- Is therapeutic knowledge key when selecting a CRO?
- Streamlining the CRO selection process in order to ensure studies are conducted in a timely, cost-efficient manner

Jigar Chheda
Clinical Project Leader & Portfolio Lead, Rare Genetic Diseases and Rare Blood Disorders
Sanofi Genzyme
10:30 am Rare Disease Drug Development: A New Clinical Paradigm
- The rare disease patient experience
- Challenges of clinical research in rare diseases
- Current clinical research paradigm
- Adopting a patient-focused mindset
- Leveraging technology and innovation
- Bring Clinical trials directly to patients
Scott Schliebner, Senior Vice President, Center for Rare Diseases, PRA Health Sciences

Scott Schliebner
Senior Vice President, Center for Rare Diseases
PRA Health Sciences
10:50 am Live Q&A with Scott Schliebner
11:00 am Lunch and networking break
11:30 am CASE STUDY: Working with a rare disease in clinical development
- An overview of how Coronis Neuroscience is developing a drug to treat the ADNP syndrome
- FDA Orphan Drug status and how this has supported clinical development
- Next steps: challenges in moving from the preclinical stage into a clinical trial for a rare disease

Illana Gozes
Chief Science Officer
Coronis Neuroscience
11:50 am Live Q&A with Illana Gozes

Illana Gozes
Chief Science Officer
Coronis Neuroscience
12:00 pm Successful project delivery in rare indications: lessons from COVID-19
- Delivering Rare Disease Studies on Time on Budget
- Relationships with sites are critical success factor
- Lessons from COVID-19 studies
Dr Peter MacLennan, Chief Operating Officer, TCR Solutions
Dr Davy Yeung, Managing Director, TCR Solutions

Peter MacLennan
Chief Operating Officer
Tailored Clinical Resource Solutions LTD

Davy Yeung
Managing Director
Tailored Clinical Resource Solutions LTD
12:20 pm Live Q&A with TCR Solutions

Peter MacLennan
Chief Operating Officer
Tailored Clinical Resource Solutions LTD

Davy Yeung
Managing Director
Tailored Clinical Resource Solutions LTD
12:30 pm CASE STUDY: Precision personalised approach to autism related rare disorders

Dr. Lidia V. Gabis
MD MBA, Director, The Weinberg Child Development Center and Keshet Center for Autism
Safra Children's Hospital at Sheba, and Tel Aviv University, Israel
12:50 pm Live Q&A with Dr Lidia Gabis

Dr. Lidia V. Gabis
MD MBA, Director, The Weinberg Child Development Center and Keshet Center for Autism
Safra Children's Hospital at Sheba, and Tel Aviv University, Israel
1:00 pm Clinical Trials within Rare Diseases Post Covid-19
- The Rise of Small-Medium Size CROs specialising in Rare Diseases & Orphan Drugs.
- Rare Disease Clinical Trials Inhouse vs Outsourcing to Specialist CROs.
- Working from Home – The new normal post Covid-19

Asad Balal
International Head of Clinical Client Services - Life Sciences
Volt International
1:20 pm Live Q&A with Volt

Asad Balal
International Head of Clinical Client Services - Life Sciences
Volt International
1:30 pm FIRESIDE CHAT: Patient focus: how to keep the patient at the forefront of your clinical trial design
- A patient perspective on clinical trials: where are the main burdens to a patient with a rare disease, and what can sponsor companies do to make this easier?
- What is most important to patients in terms of making clinical trials easily accessible?
- The importance of clear, regular communication with patients throughout the trial process and how this can improve engagement and retention
- What should pharmaceutical companies take into account in order to ensure they are working with patients in an appropriate and sensitive way?
INTERVIEWER:
Wendy Erler, Vice President, Patient Experience/STAR & Patient Experience and Advocacy, Alexion Pharmaceuticals
INTERVIEWEE:
Jennifer McNary, Patient Advocate

Wendy Erler
Vice President, Patient Experience/STAR & Patient Experience and Advocacy
Alexion Pharmaceuticals

Jennifer McNary
Patient Advocate
2:15 pm PANEL DISCUSSION: Patients at the center of rare disease clinical trials
- Lessening the burden on patients participating in orphan drug trials
- How should pharmaceutical companies be working with patients and patient organizations in order to ensure studies are as patient-focused as possible?
- Involving patient advocacy groups in your study from the beginning in order to ensure the patient is always at the front of mind
- Emerging technologies: what new solutions are available in order to make clinical trials easier for patients?
- With virtual and decentralized trials becoming more popular in light of COVID-19, will this facilitate higher participation in clinical trials?
MODERATOR:
Ian Hodgson, Independent Expert
PANELLISTS:
Jill Dolgin, Executive Director, Patient Advocacy, AGTC
Karl-Johan Myren, Senior Director, Health Economics and Outcomes Research EMEAC, Alexion Pharmaceuticals
Jay Russak, Director, Clinical Development, Dicerna Pharmaceuticals

Jill Dolgin
Executive Director, Patient Advocacy
AGTC

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

Jay Russak
Director, Clinical Development
Dicerna Pharmaceuticals

Ian Hodgson
Head of Clinical Operations, Mereo BioPharma
2:00 pm Coffee and networking break
3:00 pm Drug development for rare childhood cancers

Cesare Spadoni
Founder and Chief Operating Officer
Oncoheroes Biosciences
3:20 pm Live Q&A with Cesare Spadoni

Cesare Spadoni
Founder and Chief Operating Officer
Oncoheroes Biosciences
3:30 pm CASE STUDY: Direct-to-patient shipments in orphan drug trials and how to navigate hurdles to deliver drugs safely and efficiently
- Ensuring instructions are clear and easy to use: the importance of communication with patients
- Navigating regulatory affairs when delivering drugs directly to patients in their homes
- Emerging technologies to make direct-to-patient shipping easier in a post-COVID-19 world

Suki Malhi
Senior Director, Clinical Affairs
Zogenix, Inc.
3:50 pm Live Q&A with Suki Malhi

Suki Malhi
Senior Director, Clinical Affairs
Zogenix, Inc.
4:00 pm CASE STUDY: Using data effectively in studies with smaller numbers of patients
- Ensuring data is accurate and efficient in situations where only one attempt at a trial is possible
- Optimal ways to analyze data in order to ensure regulatory guidelines are met even with smaller sample numbers and in single arm studies
- Strategies and solutions to maximise data obtained from orphan drug trials

Jay Russak
Director, Clinical Development
Dicerna Pharmaceuticals
4:20 pm Live Q&A with Jay Russak

Jay Russak
Director, Clinical Development
Dicerna Pharmaceuticals
4:30 pm An overview of the regulatory landscape in the United States for orphan drug clinical trials
- The changing regulatory pathway for rare diseases as the industry matures
- Navigating hurdles around data and evidence for studies with smaller numbers of patients
- The benefits of FDA Orphan Drug Status in the US and how this can help make your study more efficient
- Ensuring early access to drugs and how to make sure drugs are both safe and quickly available to the American market

Mukesh Kumar
Director Regulatory Affairs
ResQ Pharma
4:50 pm Live Q&A with Mukesh Kumar

Mukesh Kumar
Director Regulatory Affairs
ResQ Pharma
5:00 pm END OF DAY 1 – JOIN US TOMORROW FOR DAY 2!
8:45 am Registration
9:00 am CASE STUDY: Challenges associated with in vivo gene therapy

James Richardson
Country Medical Director, UK and Ireland
Sarepta Therapeutics
9:20 am Live Q&A with James Richardson

James Richardson
Country Medical Director, UK and Ireland
Sarepta Therapeutics
9:30 am Improving Access to Clinical Trials for Rare Disease Patients through Registries
New medicines are made possible by participation in clinical trials and patient recruitment is often the rate limiting step in bringing new therapies to market. Research has shown that patients are likely to participate when they are aware of clinical trial opportunities and are empowered with knowledge to optimize their engagement experiences. In rare diseases, these problems are further amplified. In this session, learn how Medidata is improving access to and optimizing experiences in clinical trials through myMedidata.

Kelly McKee
Senior Director, Patient Registries & Recruitment
Medidata

Dr. David Fajgenbaum
Co-founder & Executive Director
Castleman Disease Collaborative Network (CDNC)
9:50 am Live Q&A with Medidata
10:00 am PANEL DISCUSSION: Regulatory affairs in international markets: assessing the differences between conducting a rare disease study in the UK, Europe, the US and beyond
- Why is there an imbalance between ease of conducting rare disease trials between different regulatory markets?
- Designing your study to ensure your drug is swiftly approved across multiple international markets
- Justifying regulatory approval in situations with limited data and evidence
MODERATOR:
Mimi Choon-Quinones, PhD., MBA., LLM’21, Founder & Chairman of Board of Trustees, Partners for Patients, PfP.NGO
PANELLISTS:
Ricardo Hermosilla, Translational Medicine Leader, Roche
Mukesh Kumar, Chief Executive Officer, Akan Biosciences

Mimi Choon-Quinones
Founder and Chairman
Partners For Patients, PfP.NGO

Ricardo Hermosilla
Translational Medicine Leader
Roche

Mukesh Kumar
Director Regulatory Affairs
ResQ Pharma
10:45 am Coffee and networking break
11:00 am Rare Disease Drug Development: A New Clinical Paradigm
- The rare disease patient experience
- Challenges of clinical research in rare diseases
- Current clinical research paradigm
- Adopting a patient-focused mindset
- Leveraging technology and innovation
- Bring Clinical trials directly to patients
Scott Schliebner, Senior Vice President, Center for Rare Diseases, PRA Health Sciences

Scott Schliebner
Senior Vice President, Center for Rare Diseases
PRA Health Sciences
11:20 am Live Q&A with Scott Schliebner

Scott Schliebner
Senior Vice President, Center for Rare Diseases
PRA Health Sciences
11:13 am From bench to bedside: taking a drug from the lab to a clinic to treat Williams Syndrome
- Natural history studies and preparing for a rare disease trial
- Assessing unmet needs and long term outcomes for orphan drugs in situations with no alternative treatments
- Identifying endpoints when there is no precedence or pre-existing treatment for a rare or ultra-rare disease

Boaz Barak
Assistant Professor
Tel Aviv University
11:50 am Live Q&A with Boaz Barak

Boaz Barak
Assistant Professor
Tel Aviv University
12:00 pm Support for Rare Disease Site Staff through COVID-19 and beyond
- Safety of staff & patients: reducing outside personnel visits to site, engaging with home health, and ensuring adequate PPE
- Ensuring Studies are Convenient for Site Staff to Implement
- Simplifying Recruitment Communication Reduces Site Burden: how online pre-screening portals can help
- Leveraging, improving upon and forming deeper connections with advocacy group contacts & outreach
Kelly Millhaem, Sr. Clinical Trial Manager, Associate Director Clinical Operations, Medpace

Kelly Millhaem
Sr. Clinical Trial Manager, Associate Director Clinical Operations
Medpace
12:20 pm Live Q&A with Kelly Millhaem

Kelly Millhaem
Sr. Clinical Trial Manager, Associate Director Clinical Operations
Medpace
12:30 pm ROUNDTABLE DISCUSSION: Patient centricity and recruitment: overcoming challenges around access to rare disease trials
- How to identify populations and find patients for drug trials for rare and ultra-rare diseases
- Modifying inclusion criteria: how do to this without compromising the results of your study
- Ensuring studies are accessible to patients even when specialist knowledge centres are far away
- Recruiting in a timely manner in order to avoid delays to clinical trials

Wendy Erler
Vice President, Patient Experience/STAR & Patient Experience and Advocacy
Alexion Pharmaceuticals
1:00 pm Best practice for patient engagement throughout a rare disease study
- Are virtual trials a preferable alternative to long distance travel?
- How can the burden on patients be lifted to ensure access to trials is as easy as possible?
- The impact of emerging technologies on patient engagement throughout the trial process

Ricardo Hermosilla
Translational Medicine Leader
Roche
1:20 pm Live Q&A with Ricardo Hermosilla

Ricardo Hermosilla
Translational Medicine Leader
Roche
1:30 pm Rare Disease Studies - Data Challenges & Solutions
- Key data challenges around Rare Disease Studies
- Technology solutions for to support data challenges in these studies
- How a unified platform and centralized data can streamline the process
Andrew Schachter, Founder and CEO, Axiom Real-Time Metrics
Kaitlyn Townsley, Associate Director, Product Innovation, Axiom Real-Time Metrics

Andrew Schachter
Founder / CEO
Axiom Real-Time Metrics

Kaitlyn Townsey
Associate Director of Product Innovation
Axiom Real-Time Metrics
1:50 pm Live Q&A with Kaitlyn Townsley

Kaitlyn Townsey
Associate Director of Product Innovation
Axiom Real-Time Metrics
2:00 pm Patient Centric Approach in Clinical Trials

Anne Bruns
Director, Clinical Patient Advocacy
PTC Therapeutics

Vij Senthilnathan
Senior Manager, Clinical Operations
PTC Therapeutics
2:20 pm Live Q&A with Anne Bruns and Vij Sethilnathan

Anne Bruns
Director, Clinical Patient Advocacy
PTC Therapeutics

Vij Senthilnathan
Senior Manager, Clinical Operations
PTC Therapeutics
2:30 pm CLOSING KEYNOTE: Moving forward for rare disease studies: are decentralized and virtual trials a permanent solution?
- Getting your trial back on track post-COVID-19: are remote solutions the way forward?
- Weighing up the pros and cons of virtual and decentralized trials for rare disease studies
- Has COVID-19 marked an end for the need for regular site visits throughout clinical trials – or should we be looking at hybrid models for the future?

Dr. Ian Dr. Ian Hodgson
PhD, Clinical Advisor Consultant
Mereo BioPharma Group
2:50 pm Live Q&A with Ian Hodgson

Dr. Ian Dr. Ian Hodgson
PhD, Clinical Advisor Consultant
Mereo BioPharma Group
3:30 pm END OF CONFERENCE – THANKS FOR COMING!
SPONSORSHIP OPPORTUNITIES
To enquire about sponsorship opportunities for the conference, please contact:
Alexander O’Leary
Portfolio Director – OCT EMEA and Pharma Packaging & Labelling Global
Arena International Events Group
T: +44 207 936 6690
E: alexanderoleary@arena-international.com
SPEAKING OPPORTUNITIES
To enquire about speaking opportunities for the conference, please contact:
Louisa Manning
Programme Director
ZOOM PHONE: +44 (0) 207 661 0906
E: louisa.manning@arena-international.com