A world leader in developing and commercializing innovative biopharmaceuticals for rare diseases driven by genetic causes. Throughout, we’ve worked tirelessly to make a difference by pursuing bold science while respecting, educating, and connecting with patients. Through our expertise in genetics and molecular biology, we have been able to develop targeted therapies that address the root cause of the exact conditions we seek to treat.

Our discoveries have led us to countless breakthroughs, best-in-class treatments and many ‘firsts’ in the category. We are grateful to able to better the lives of those struggling with genetic diseases. As we look forward, we strive to make an even greater impact by applying the same science-driven, patient-forward approach to a broader range of genetic disorders.

RESPONSIBILITIES

Global Regulatory Affairs (GRA) is responsible for obtaining approval for new products and ensuring that approval is maintained throughout the product lifecycle. GRA serves as the interface between the regulatory authorities and the program teams aiming to ensure that the project plan correctly anticipates what the regulatory authority will require before approving the product. It is the responsibility of GRA to keep abreast of current legislation, policies, guidelines and other regulatory intelligence. The RA department plays an important role in giving advice to the program teams on how best to interpret the regulatory legislation and guidelines

The Manager supports Global Reg Teams in the development and execution of global nonclinical / clinical regulatory strategies through a program’s lifecycle. (Open to position being Remote)

Responsibilities may vary depending upon product regulatory lifecycle and may include the leadership of selected activities which will be delegated by the Regulatory Nonclinical/ Clinical Lead and may include the following:

Development Stage Product

• Support the development of the nonclinical / clinical regulatory strategy and plan.
• Assist with selected planning, document development and meeting execution activities in preparation for HA meetings / interactions.
• Participate in Study Execution Teams for assigned products and identify relevant regulatory considerations for discussion with RNC Lead.
• Collaborate with CROs that are contracted to perform regulatory submissions/HA interactions for global clinical trials.
• With oversight by RNC lead, develop and coordinate the core nonclinical / clinical content of regulatory submissions.
• Responsible for ensuring high quality nonclin/clinical content that adheres to regulations and guidance's.
• Lead the maintenance of IND/CTAs throughout the life of the studies (e.g., annual reports)
• Assist with the preparation of HA submission packages including but not limited to compiling relevant materials (Protocols, Annual Reports, Briefing Books, etc.) and completing regulatory documents/forms for internal review.
• Collaborate with Reg PM to create and align submission timelines,
• Collaborate with Reg Ops to provide final documents and QC published outputs for submissions to HA.
• Oversee the collaboration of Nonclin/clin with Reg Ops to provide final documents and QC published outputs for submissions to HA.
• Organize and maintain the administrative, nonclinical, and clinical portions of IND’s, NDA’s and BLA’s.
• Oversee the archiving of HA submissions and correspondence

Commercial Stage Product

• Lead the development of updates to the clinical and nonclinical sections of the core dossier used for INTL marketing applications.
• Provide support to RNC lead on development of nonclinical / clinical portions of required product lifecycle maintenance regulatory submissions, examples are: clinical or nonclinical supplements to approved US product dossiers and any required annual reports, license renewals, supplemental filings.
• Coordinate necessary interactions with INTL in support of INTL MA filings. Example include: meetings to provide RNC guidance/input on agency interaction, submissions, RTQs.

General

• Attend relevant functional area and project team meetings.
• Review, understand and stay up to date on regulations, guidelines relevant to responsibilities
• Review documents (SOPs, protocols and reports) related to Clinical or Nonclinical studies as necessary
• Ensure that the PM timelines and tracker accurately reflect submission plans

Skills

Decision Making and Problem Solving, including an understanding of decision-making models and methodology

Influence, Cross-Functional Collaboration and Organizational Awareness, including demonstrating professional maturity, including understanding own strengths and receptivity to feedback

Agility and Proactivity, including an ability to anticipate risks or issues to program or business and take the necessary steps to mitigate or address

Leadership, such as skill in clearly communicating objectives, goals, and expectations to others

Communication, including an ability to provide context and articulate key messages effectively and with appropriate level of detail

Strategic Thinking and Planning, such an ability to set short-term and longer-term goals for individual objectives and task completion

Regulatory Strategy, Frameworks and Processes: Demonstrates basic understanding of regulations, guidances and pathways including when and how to file routine submissions; asks appropriate question to clarify / validate understanding

Health Authority Engagement: Knows basics of how relevant health authorities function, their general structure and the key personnel

Scientific Writing for Regulatory Submissions: Demonstrates basic knowledge of content guidelines and structures (sections, etc.) for writing routine regulatory submissions (i.e., annual reports)

Drug Development and Study Design: Understands the objectives/purpose of each drug development phase and the related objectives and regulatory requirements, including as it relates to rare disease development

Scientific and Medical Knowledge: Understands the implications of competitive product developments on therapeutic area regulatory strategies / plans

Education/Experience

• Degree in health or life sciences, including chemistry, molecular biology, or similar. PhD preferred, Masters/Bachelors acceptable with relevant experience
• 4 + year experience with PhD; 6+ years with Master's or bachelor’s degrees
• Nonclinical / Clinical Regulatory Affairs experience preferred