A Collaborative Approach to Advancing Access and Equity in Rare Disease Clinical Trials

Life Sciences, Clinical Trials, Patient Recruitment & Retention, Drug Discovery & Development,
  • Thursday, February 29, 2024

Ensuring that all patients have equal access to participate in clinical trials across all geographies has become essential throughout the industry. Patient recruitment and retention remain one of the most challenging hurdles in clinical development, particularly for rare diseases. Implementing a patient-focused clinical trial design and fostering a collaborative approach between the Sponsor, CRO, sites, and advocacy groups can help lead to successful clinical trials.

Given the daily obstacles faced by patients with rare diseases, including late onset Pompe disease, it is critical to minimize patient burden while recruiting a committed group of patients. Once recruited into a clinical study, it becomes paramount to provide ongoing support by focusing on the needs and concerns of the patient. This enhances patient retention, facilitating the collection of vital data for primary endpoints, which ultimately leads to faster therapies to market to give the patient community a treatment option.

In this webinar, Medpace, a global full-service CRO, will discuss the importance of equity in patient-friendly rare disease trials. Experts will discuss strategies in creating a positive clinical trial experience through a collaborative approach between sites, the Sponsor, and the CRO.

Register now for the Rare Disease Day 2024 webinar to join the discussion with clinical trial experts on a collaborative approach to patient-friendly trials in rare disease.


Terence Eagleton, Medpace

Terence Eagleton, MB BS, BSc, Senior Medical Director, Medpace

Dr. Terence Eagleton is a UK-based physician who qualified from University College London Medical School, after which he trained as a general surgeon with a focus on trauma and critical patient care. He has subsequently worked as a senior pharmaceutical physician in the biopharma and CRO industries for over 20 years in global clinical research and medical affairs, in a wide variety of therapeutic areas. He has extensive experience and achievement in international orphan drug development across Phase I to IV, with a specific focus in clinical research with a variety of innovative therapeutic modalities. Dr. Eagleton is an affiliate member of the Faculty of Pharmaceutical Medicine (FPM) in the UK. He has authored original papers in peer-reviewed journals and established himself as an industry expert in rare diseases.

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Miaesha Campbell, Medpace

Miaesha Campbell, Senior Director, Patient Recruitment, Medpace

Miaesha Campbell is an accomplished professional with over 15 years of experience directing a global team of strategic partners, support staff and vendors for multimillion-dollar global marketing, patient recruitment and patient retention campaigns. This includes oversight of the planning and execution of programs with budgets ranging from $250,000 to $17 million. Miaesha graduated from California State University at Dominquez Hills in May 2002, earning a Bachelor of Arts degree in English and a Minor in Communications. Her early career was spent in public relations, and she was recruited to support the marketing of clinical trial programs in 2007.

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Kristin Black, Medpace

Kristin Black, Clinical Trial Manager, Medpace

Ms. Black started her career at Medpace in 2015 as a CRA, where she sharpened her strong leadership skills propelling her into the role of CTM. Kristin has significant experience working as a lead CRA and CTM in LSDs, cardiology, metabolic disorders, neurology, and rare disease trials. Kristin’s training and experience in rare disease, and proven track record of success building relationships, make her a natural fit as CTM.

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Nita Patel, Amicus Therapeutics

Nita Patel, RN, Executive Director, Patient Advocacy at Amicus Therapeutics

Nita is a registered nurse with experience in clinical research, obstetrics and genetic nursing. She has been working at Amicus since September 2011. Her expertise in lysosomal diseases was gained from managing a lysosomal treatment center for approximately 15 years at St. Peter’s University Hospital Institute for Genetic Medicine located in New Brunswick, NJ. Nita has also provided training and education for patients in renal failure receiving peritoneal dialysis, pre-natal teratology counseling and testing. Nita has also monitored clinical studies in lysosomal disorders; her passion is to advocate for patients and represent their voice to industry and drug development. Nita has been an Ambassador for Needy Meds; she continues to help patients with their needs to access the medical care they deserve. Since 2016, Nita has been a faculty member for Professional Patient Advocates in the Life Sciences (PPALS), she lectures on Patient Advocacy and “Return on Investment”.

Nita has completed midwifery training in London, UK; received Genetic Nurse Certification, Obstetric Nurse certification and Clinical Research certification.

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Who Should Attend?

VPs, Directors, Managers and Department Heads working within: 

  • Clinical Affairs 
  • Clinical Research 
  • Clinical Pharmacology 
  • Clinical Outsourcing 
  • Project Management 
  • Regulatory Affairs 
  • Medical Affairs 

What You Will Learn

Join Medpace experts in this upcoming webinar as they explore:

  • The importance of creating patient-friendly clinical trials in rare diseases
  • How to engage and support patients in rare disease clinical trials
  • Lessons learned in a successful Pompe clinical trial
  • Strategies for a successful collaborative approach between the Sponsor, CRO, and sites

Xtalks Partner


Medpace is a scientifically-driven, global, full-service clinical contract research organization (CRO) providing Phase I-IV clinical development services to the biotechnology, pharmaceutical and medical device industries. Medpace’s mission is to accelerate the global development of safe and effective medical therapeutics through its high-science and disciplined operating approach that leverages local regulatory and deep therapeutic expertise across all major areas including oncology, cardiology, metabolic disease, endocrinology, central nervous system and anti-viral and anti-infective.

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