Director, Clinical Research – Pompe Disease
The Sr. Dir./Director, Clinical Research, Pompe will be responsible for ensuring the best possible global development strategy and execution of the Pompe program in Amicus’ pipeline leading to successful submissions to Regulatory Authorities. This position will collaborate with multiple individuals within Amicus, most notably in Clinical Operations, Regulatory Affairs, Discovery Research, Program Management and Medical Affairs to execute this strategy. This position will have the experience, flexibility and maturity to adapt his/her skills effectively in a variety of diverse tasks related to clinical development of the company’s products, from generating strategy to every aspect of drug development, including developing protocols, medical monitoring, analyzing data, reporting study results, collaborating with external experts and Key Opinion Leaders in Pompe disease, and interacting with FDA, EMA and other regulatory agencies.
The Sr. Director/Director, Clinical Research – Pompe Disease is responsible for:
- Leading the clinical development strategy for the Pompe program and leading the clinical development plan preparation.
- Lead role in executing the development strategy for the Pompe program from early phase studies up to the NDA and beyond, on a global basis.
- Play a significant leadership role, as well as provide hands on tactical work, in project teams and work in collaboration with relevant team members and peers to ensure the highest level of execution for the Pompe clinical development program.
- Clinical Study Design and Execution:
- Develop, author and execute on Pompe clinical protocols working with external Pompe Key Opinion Leaders, Principal Investigators, internal and external scientific, pharmacology and regulatory teams.
- Work with Clinical Trial Investigators and clinical research organizations, to collaborate on trial site selection, training and ongoing conduct of the study.
- Act as Pompe Program lead medical monitor, conduct site visits as needed, have detailed understanding of patient level data including safety and efficacy data, and data review during the study.
- Ensure that studies are performed with the highest quality. Perform data review and analysis, presentations (oral and written) of clinical data, authoring and editing of clinical study reports.
- Successfully interact with Pompe clinical trial investigators, experts and other external partners in a manner consistent with Amicus’ beliefs.
- Participate in preparation of regulatory documents (e.g. Investigator Brochures, CSRs, Briefing documents) and participate in face-to-face meetings with the regulatory agencies.
- Maintain highest level of medical science and clinical development expertise in Amicus’ fields of interest (Pompe and other relevant rare and orphan diseases).
- Collaborate on business development activities in support of Pompe or other programs
- Perform all responsibilities in compliance with company policies, SOP and guidelines.
- Medical Doctor’s (MD) degree and post residency training is required.
- Completion of a residency in either pediatrics or internal medicine and fellowship in a related subspecialties area (e.g. neurology, genetics).
- Significant experience practicing medicine is a plus.
- A combination of credentials and personal attributes including medical training, reputation, clinical and industry drug development experience (preferably in rare diseases) and integrity are necessary for success in this role.
- A minimum experience of 5-7 years in industry is required.
- A drug development professional with experience derived from a career in a major pharmaceutical company, or in a significant biotechnology or biopharmaceutical company environment, including a key role in the submission of a successful NDA/BLA or MAA.
- Experience with rare and/or orphan diseases is highly desired. Knowledge of lysosomal storage disorders a significant plus.
- Experience in drug development in pediatric populations is a significant plus.
- Experience on teams with early and late stage development products.
- Experience leading Medical Advisory Boards.
- Strong project management skills, business savvy and business development experience is desirable.
- Flexibility and adaptability, able to thrive in small biotechnology company environment.
- Excellent oral and written communication skills.
- Strong interpersonal skills, a collaborator/ team player, open minded to the diverse opinions of others.
- Ability to produce highest quality and timely clinical development work product/ documents with limited direction.
- Hands-on performer, ensuring strong execution of clinical trials and other drug development activities.
- A superior work ethic and a strong personal desire for success. He or she must also possess a strong sense of personal and professional ethics.
- A good balance of confidence and humility.
- Must be passionate about developing treatments for rare/orphan diseases.
- Both domestic and international up to 25-30 % of time
To Apply to this job Please go to website address given in Source below