Senior Director, Clinical Development

Role Summary:

The Senior Director, Clinical Development will have primary responsibility for the design and oversight of Duchenne Muscular Dystrophy (DMD) clinical studies within an integrated development strategy, including both in-house development and potential strategic partnerships to augment the internally developed pipeline. This position will provide comprehensive leadership of the development process from pre-IND through to NDA and Phase IV programs. The Senior Director, Clinical Development will be responsible and accountable for the development of the medical aspects of DMD studies within the clinical program. This role will be responsible for delivering quality and timely medical input and interpretation on specific program data in fulfilment of the program and study objectives, working in partnership with Regulatory Affairs and Pharmacovigilance in providing medical support for pharmacovigilance activities, responses to regulatory agency queries, and development. 

This role is based in Waltham, MA without the possibility of being fully remote.

Primary Responsibilities Include:

• Lead DMD Clinical Development study teams across planning, design, development, and implementation of clinical trials 

• Provide Clinical Development guidance for clinical programs, including strategy, planning, execution, and completion of clinical trials according to all applicable regulations and guidance 

• Develop and implement effective, timely, and cost-efficient clinical studies to establish the safety and efficacy of drug candidate compounds to achieve approval in the US and other countries 

• Develop knowledge of disease mechanisms to select biomarkers, pharmacodynamic endpoints, functional outcome measures, and patient stratification considerations  

• Actively contribute to Clinical Development and study team meetings to ensure close collaboration and alignment with key cross-functional stakeholders 

• Serve as study Medical Monitor and medical representative to investigators, advisors, and regulatory agencies, as needed 

• Coordinate with the Research group to assure smooth transition of development candidates/programs from research into development, including guidance on decisions that have clinical components and implications  

• Collaborate with Pharmacovigilance to manage timely reporting of safety signals to regulatory agencies  

• Collaborate with Clinical Operations to ensure all clinical study activities are completed in accordance with applicable regulations and guidance, maintaining the highest quality clinical programs, following GCP and ICH guidelines and the Company’s standard operating procedures  

• Maintain accountability for all relevant timelines, resources, and deliverables working closely with functional counterparts and colleagues on the integration of product and Clinical Development plans 

• Develop and contribute to key documents including protocols, investigator brochures, and informed consent forms  

• Develop and implement SOPs for clinical trials and related activities, as required  

• Review presentations and publications (regulatory submissions, original peer-reviewed articles, abstracts)  

• Select between outsourcing and in-house approaches for Clinical Development capabilities and work closely with the Clinical Operations team to implement choices  

• Manage, coordinate, and provide ongoing assessment, evaluation, and communication with other departments and third parties, such as CROs and external vendors, in order to ensure study objectives and timelines are accomplished  

• Ensure compliance with all applicable regulatory standards related to global clinical trials and interactions with physicians 

• Support the Vice President, DMD Clinical Development with strategic development and implementation of integrated development plans, and provide timely updates on clinical studies and relevant workstreams 

• Provide leadership and mentorship to Clinical Development team members, managing matrix and direct line reports 

• Interface with departments within and outside of R&D including Regulatory Affairs, Legal, Finance, and Medical Affairs  

• Identify, manage, and interface with external collaborators and key opinion leaders to create Clinical Development strategies for a novel class of therapeutics  

• Develop and maintain professional relationships with academic and community-based study sites  

Education and Skills Requirements:

• Doctor of Medicine (MD), Master of Science in Nursing (MSN) or Doctor of Nursing Practice (DNP) with a strong scientific background and a specialty in neurology or neuroscience training highly preferred  

• Minimum of 8 years of pharmaceutical industry experience  

• Experience leading a clinical development program(s) in Neurology/ Neuromuscular disorders  

• Experience with all aspects of designing, executing, monitoring, and managing clinical trials from inception to completion across all phases of development  

• Robust regulatory experience with Pre-INDs, INDs, CTAs, EOP2 meetings and NDA/BLAs/MAA, both US and ex-US  

• Proven track record of drug development with Phase 1 through 3 trial experience preferred  

• Experience managing significant accelerations and challenges in clinical programs  

• Strong knowledge of FDA and ICH regulations  

• Thorough knowledge of clinical medicine, clinical pharmacology and associated disciplines  

• Expert knowledge of Good Clinical Practice (GCP)  

• Experience in a small to mid-sized biotech company is preferred  

• Significant experience in communicating/presenting key/complex information to department/functional lead(s)/senior management 

• Readiness to “roll up the sleeves” to contribute to team efforts  

• Excellent writing skills with a track record of publications and polished presentation skills to influence internal and external audiences  

• Ability to develop creative approaches to processes, practices, services, business models, and strategy  

• Ability to translate basic science into a product development plan that will maximize the product’s commercial opportunity  

• Ability to work successfully in a matrix environment and effectively build consensus  

• Communication skills (written and verbal), which are equally effective internally and externally, with large and small audiences, and one-on-ones  

• Ability to effectively lead cross-functional meetings, and meetings with senior leaders to drive successful outcomes  

• Ability to collaborate with and inspire high-performance clinical development team  

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