Clinical Trials in Rare Diseases - Virtual Event

• 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

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.

• 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

• 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

• 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

• 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

• 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

• 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

• 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

• 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

• 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

• 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

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.

• 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

• 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

• 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

• 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

• 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

• 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

• 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

• 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?