Medical Director – Clinical Trial Lead

Place in the Organization

uniQure is dedicated to bring innovative therapies or unmet medical needs to patients. In doing so each function in uniQure is directly or indirectly responsible for the lives of patients. This responsibility should be part of our day-to-day working life by (i) living up to high quality standards and (ii) by recognizing the sense of urgency in areas of high medical need.

The Medical Director will lead and oversee clinical trials based in the US. The successful candidate will be responsible for leading clinical trials, engaging with Key Opinion Leaders (KOLs) and stakeholders, and ensuring the successful execution and monitoring of ongoing trial activities, including safety and efficacy assessments. This role requires a deep understanding of trial execution, with a strong background in clinical research and drug development, preferably in neurology or rare disease.

Key result areas (major duties, accountabilities and responsibilities) 

Clinical Trial Leadership:

• Lead and oversee clinical trials focused, ensuring adherence to regulatory and ethical standards.
• Collaborate with the clinical development team to support study design, implement, and monitor clinical trial protocols, with a focus on safety, efficacy, and regulatory compliance.
• Provide expert guidance on patient selection criteria, dosing regimens, and endpoints.

Stakeholder Engagement:

• Develop and maintain strong relationships with Key Opinion Leaders (KOLs), clinical investigators, study coordinators, and other relevant stakeholders.
• Serve as a key point of contact for external partners, including clinical sites and contract research organizations (CROs) in terms of medical questions related to study readouts and assessments.
• Represent the company at scientific conferences, patient events and advisory boards.

Safety and Efficacy Monitoring:

• Oversee the monitoring of trial activities, including patient safety, adverse event reporting, and efficacy assessments.
• Support the study team in the review and interpretation of clinical trial data, providing insights and recommendations for trial progression.
• Collaborate with the Medical Safety and PV team (internal and external) to ensure timely reporting and resolution of safety issues.

Regulatory and Compliance:

• Ensure that all clinical trial activities comply with FDA regulations, Good Clinical Practice (GCP) guidelines, and other relevant regulatory requirements.
• Participate in the preparation of regulatory submissions, including clinical trial protocols amendments, and safety reports.

Team Collaboration:

• Work closely with cross-functional teams, including clinical operations, regulatory and medical affairs, medical safety and writing, and biostatistics, to ensure the successful execution of clinical trials.
• Educate and provide guidance to uniQure clinical staff and research team in terms of epilepsy relevant aspects and related assessments.

Qualifications & Skills

• MD degree with board certification, preferably in neurology.
• Minimum of 5 years of clinical experience, with a focus in neurology or rare disease preferred.
• Proven experience in leading and conducting Phase I and II clinical trials.
• Ability to travel to study sites, attend required meetings, and conferences.
• Excellent communication and interpersonal skills, with the ability to engage effectively with KOLs, clinical investigators, and regulatory authorities.
• Ability to interpret and analyze clinical trial data, with a focus on safety and efficacy.
• Knowledge of FDA regulations and guidelines for clinical trial, GCP guidelines, and clinical trial processes.
• Experience in medical writing and regulatory submissions is a plus.
• Experience in gene therapy trials is a plus.

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US Salary Range 205000 - 265000 USD