In this three-part series of discussions with top experts in clinical development for rare disease and orphan indications, Medpace, along with industry partners, will address some of the challenges, opportunities, and considerations for conducting research in this dynamic and complex area of research. The series is targeted to Sponsors who are developing therapies for rare diseases with a goal of providing insights and a forum for discussion into three of the key areas of trial design and execution that can impact the speed, efficiency and overall success of the trial.
Part 1: Rare Disease Clinical Research – Spotlight on the Patient and Caregiver
In this webinar, Medpace partners with the Batten Disease Support and Research Association to discuss the need to educate patients and their caregivers on the importance and increase of clinical trials aimed at finding effective therapies and cures for rare diseases. This includes identifying the appropriate communication pathways and using them to develop effective outreach strategies aimed toward providing caregivers and patients with what they need to find and enroll in clinical studies.
It is important that sponsors leverage different methods of study awareness such as broad-based communications to reach patients. These methods may include sending letters, emails and newsletters to patient advocacy groups’ member databases, and creating websites and social media posts to post study information. Additionally, hosting informational meetings to walk through study participation and providing educational support materials for a greater understanding of the study are useful strategies for recruiting patients.
Once recruited into a clinical study, it then becomes paramount to provide ongoing support and reassurance by focusing on the needs and concerns of the patient and caregivers. This type of tailored support will lead to greater retention on studies, which leads to the collection of needed data for primary endpoints and then ultimately faster therapies to market.
Join this free webinar to learn about:
- How contract research organizations and sponsors can work collaboratively with advocacy groups to raise awareness, train and support patients and caregivers
- Patient-centricity – how to make the buzz word a reality
- Tools and tactics – what works and what doesn’t
- Special considerations for pediatrics and young adults
Part 2: Rare Disease Clinical Research – A Deep Dive into Regulatory Strategies and Considerations
Regulatory bodies have demonstrated flexibility in applying the standards of safety and efficacy to rare disease trials given the unique challenges that drug sponsors face. Developing a sound strategy to navigate the regulatory landscape for rare diseases can literally save (or cost) a sponsor months, or even years, of development. That translates into lives saved and quality of life for those suffering with the disease, and financial gains or losses as well as a competitive advantage for the drug sponsor. In this free webinar, Medpace’s global regulatory experts will discuss some of the key considerations for accelerating development.
Topics will include:
- Which way should we go? A deep dive into the available designations and regulatory pathways that can be used to expedite and accelerate treatments for rare diseases in the US and EU including: orphan drug designation, fast track designation, breakthrough designation, rare pediatric disease vouchers, PRIME and accelerated and conditional approvals.
- What is required and how do we prepare? An in-depth assessment of the requirements for these designations and accelerated or expedited pathways, how to prepare your application and what to expect when you’re granted a designation or an expedited or accelerated pathway, as well as considerations around best-timing for submission will be provided from a global perspective. The presenters will also describe how to prepare for meetings with the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA), and what to expect when engaging with regulatory authorities, particularly in a face-to-face meeting.
- With or without pediatric populations? The pros and cons of including pediatric patients in adult trials for rare diseases will be presented, including suggestions for when it is time to engage regulators in discussions about including pediatrics patients earlier in the clinical development program. The webinar will wrap up with an overview of requirements for pediatric plans in the US and EU, and how these plans fit into the overall development program for rare diseases.
Part 3: Rare Disease Clinical Development – Strategies for Ensuring Endpoint Integrity
Because clinical trial endpoints for rare diseases are often not well defined, consistency of endpoint assessments across study sites and over time are critical to ensuring data integrity. Having a cohesive training strategy that includes conducting pre-study evaluation and training, as well as ongoing review of clinical evaluator (CE) performance and adherence to standardised procedures is an important contributor to success.
In this free webinar, Medpace partners with Michelle Eagle of ATOM International, a provider of CE training for clinical trials across the world, to discuss approaches and steps that can be taken to ensure quality and integrity. For example, neuromuscular studies incorporate training of clinical evaluators at investigator meetings, support video-recording of patient assessments at study visits to review for quality control and perform periodic refresher training throughout the course of a study.
Topics will include:
- What does a comprehensive training program look like?
- How natural history data can inform endpoint selection (real-word examples)
- Rating assessment considerations and review (video QC and clinical assessment reliability)
- The role of data management – cleaning and coding of data to create structured output for review and reliability
Learn more about Medpace’s clinical research experience in rare disease and orphan indications here.