Agios Fellowships

About Clinical Development, Clinical Scientist Fellowship

Clinical development is responsible for the architecture of clinical trial concepts, protocol development, study start up, study execution, data review and cleaning, clinical interpretation of data, publication contributions, and serves as a clinical resource for regulatory filings and queries from authorities and sites. Within clinical development, clinical scientists play a role on the clinical trial working group (CTWG). They work to help align study goals and strategy within the clinical trial working group, , serve as a resource for investigators on patient eligibility, safety, and protocol questions, and partner cross-functionally with early or late-stage clinical development, medical, and commercial teams.

Clinical Development, Clinical Scientist Fellowship Goals and Objectives

The fellow will gain hands-on knowledge of US and Global clinical development at Agios. The fellow will gain experience in clinical trial-related activities within the cross-functional clinical trial working group and gain an extensive understanding of the responsibilities of a clinical scientist. The fellow will:

• Develop a strong clinical development knowledge base and be able to apply their skills practically.
• Obtain well-rounded knowledge of country or region-specific clinical trial processes.
• Cultivate strong communication and project management skills.
• Collaborate cross-functionally with various departments within the company and with key external collaborators as a partner in the product development process.

Clinical Development, Clinical Scientist Program Design

The fellow will join our dynamic Clinical and Translational Development (CTD) function in the Research and Development organization at Agios and will be given a plethora of opportunities to learn the business of rare disease drug development. Agios already has a network of MCPHS PharmD Fellows, who are seen as key contributors to their respective functions and have shown the positive impact that they can have on the company. The clinical and translational development (CTD) function and clinical scientist team is excited to welcome their first fellow to contribute to the success of the organization and learn from our enthusiastic and diverse group of researchers.

Year 1: Clinical Development

• Learn the purpose and goals of a clinical scientist across different phases of clinical development.
• Gain an understanding of the structure and function of a clinical trial and the roles and responsibilities of a clinical scientist within study start up, execution, and closeout.
• Collaborate with key team members and functions who are part of clinical trial working groups at Agios, such as Clinical Operations, Regulatory Affairs, Data Management, and Safety for assigned trial(s).
• Become familiar with clinical trials being run at Agios (Phase 1-3, Adult and Pediatric, and clinical pharmacology).
• Independently review contracts, study documents, clinical data and protocol deviations for assigned trial(s).
• Perform initial review of key study documents.
• Develop a working knowledge of GCP and ICH.

Year 2: Clinical Development

• Contribute to initial authoring and/or review of key study documents (Protocol, Informed Consent Form (ICF), Investigator Brochure (IB), Lab Manual, etc.).
• Respond to site queries and regulatory authority queries in conjunction with clinical scientist (CS) 
• Contribute to clinical data review activities across an indication/molecule.
• Assist in development  of clinical data review standards and timelines with cross-functional teams.
• Play a role in preparing for functional review of program milestones and data supporting milestones such as interim analyses, database locks, publications, clinical advisory boards and clinical study reports (CSR), etc. as needed for assigned trial(s).
• Serve as a clinical contributor for other relevant activities (analysis plans, filing, data monitoring committee, clinical advisory boards, etc.) as needed for assigned trial(s).

About Clinical Supply Chain

The Clinical Supply Chain group at Agios develops the strategies for managing all aspects of drug supply for investigational studies and expanded access programs. Responsible for end-to-end supply chain management, Agios’ Clinical Supply team members collaborate across a multitude of departments in drug development and oversee activities between drug product manufacturing and dispensation to patients at the clinic. These activities include determination of drug demand forecasts, development of packaging and labeling supply plans, facilitation of manufacturing with external vendors, setting up global distribution strategies, and development of risk mitigation plans. As an integral part of clinical program and study teams, Clinical Supply members also contribute to clinical study protocols, provide guidance on patient centric packaging design, participate in the build of randomization and trial supply management (RTSM) systems, and develop the masking strategy for blinded studies. Supporting multiple quickly growing programs, the group utilizes industry expertise and explores cutting-edge technology to continuously improve the way Agios delivers medications to patients.

Clinical Supply Chain Fellowship Goals and Objectives

The fellow will gain hands-on knowledge of Clinical Supply Chain activities within the Technical Operations organization and gain an extensive understanding of the drug development process.

Objectives include:

• Develop end-to-end clinical supply chain knowledge and be able to apply their skills practically to drug supply management at the clinical study and program level
• Cultivate strong communication, leadership, and project management skills
• Collaborate cross-functionally with various departments within the company as a partner in the drug development process
• Recognize common supply challenges and act proactively to ensure study demand is met throughout the trial process
• Effectively coordinate activities and communication with vendors within an external manufacturing and logistics network

Clinical Supply Chain Program Design

As an integrated member of the Clinical Supply Chain team at Agios, the fellow will be provided with a variety of opportunities for growth and development within the genetically defined disease (GDD) portfolio. Working in a future-oriented and improvement-focused environment, the fellow will gain hands-on clinical supply chain experiences to further enhance their professional knowledge. Assuming increasing responsibility over the two years, the fellow will obtain a deep comprehension of clinical supply chain’s role, responsibilities, and day-to-day tasks as part of a cross-functional team to support global drug development.

Through the afforded mentorship, leadership, and empowerment of the fellowship team at Agios, the fellow will have access to the following responsibilities:

Clinical Supply Chain Year 1

• Integrate into the Clinical Supply Chain/Technical Operations teams
• Learn how to interpret clinical study protocols to determine drug demand and supply strategies
• Support the Clinical Supply team in completing day-to-day drug supply management tasks
• Observe how Clinical Supply team interacts with other functions within Agios and external vendors and actively participate in these interactions
• Gain an understanding of and hands-on training in applicable clinical supply technology platforms
• Transition to full responsibility of clinical supply activities for early phase and Investigator Sponsored Trials
• Develop cross-functional leadership skills by coordinating and leading clinical supply plan meetings
• Gain exposure to the entire Supply Chain Organization, including Commercial Supply, Clinical/Commercial Supply Planning, and Logistics

Clinical Supply Chain Year 2

• Take responsibility for drug supply activities as a Clinical Supply lead for one or more global clinical trials
• Be responsible for drug demand forecasting, trial monitoring & resupply planning, study technology set-up, risk mitigation plans, and global distribution strategy and activities
• Schedule and facilitate packaging and labeling manufacturing with external vendors
• Collaborate regularly with Clinical Operations, Quality Assurance, and Regulatory Affairs to efficiently deliver clinical supplies while ensuring compliance to all applicable laws and regulations
• Contribute to program level supply planning to determine drug product manufacturing timing and allocate inventory amongst clinical studies
• Become proficient in using drug forecasting technology to create a drug demand model for applicable clinical studies
• Represent Clinical Supply Chain as an active member of clinical trial working groups and project teams

Drug Development Quality Assurance is a specific function within quality assurance responsible for manufacturing oversight, lot release and vendor oversight for the CMC quality aspects of pharmaceutical clinical development programs.  The Fellow will work with the QA team in support of lot release of investigational products Phase I through Phase 3.  This team collaborates with a diverse cross-functional team, including CMC (chemistry and manufacturing controls), analytical development, quality systems-QA, quality control-QA, regulatory affairs and clinical supply chain to provide ensure lot are released according to approved specifications to meet supply chain timelines.

The fellow will gain hands-on knowledge of drug development quality assurance activities within the Technical Operations organization and gain an extensive understanding of the drug development process.

Objectives include:

• Develop end-to-end quality oversight knowledge primarily in the clinical space.
• Cultivate strong communication, leadership, and project management/organization skills
• Collaborate cross-functionally with various departments within the company as a partner in the drug development process
• Understand the requirements for supplying drug for phase 1-3 studies, US only and global studies, blinded studies, Investigator Sponsored Trials (IST), and Expanded Access Programs (EAP)
• Recognize common quality challenges and act proactively to ensure product quality is met throughout the clinical trial process
• Effectively coordinate activities and communication with vendors within an external manufacturing network

Drug Development Quality Assurance Program Design

The Fellowship follows a rotational development plan with the first year focused on the clinical packaging and labeling (P&L) QA space, specific to release of finished goods in support of clinical trials.  End of year 1 and year 2 may expand into quality oversight and release of commercial finished goods as well as clinical drug product and drug substance.  The fellow will gain hands-on GMP-Quality Assurance experiences to further enhance their professional knowledge. Assuming increasing responsibility over the two years, the fellow will obtain a deep comprehension of GMP-QA’s role, responsibilities, and day-to-day tasks as part of a cross-functional team to support global clinical and commercial drug development.

Through the afforded mentorship, leadership, and empowerment of the fellowship team at Agios, the fellow will have access to the following responsibilities:

Drug Development Quality Assurance Year 1

• Integrate into the Quality Assurance/Technical Operations teams
• Observe how Drug Development Quality Assurance team interacts with other functions within Agios and external vendors and actively participate in these interactions
• Gain an understanding of and hands-on training in applicable clinical supply and quality assurance technology platforms
• Work with CMO’s (Contract Manufacturing Organizations) to provide quality oversight of all primary and secondary packaging as well as labeling activities of clinical supplies, including review of master batch records, packaging specifications and executed batch records.
• Work with quality manager to fully investigate any P&L manufacturing deviations.
• Provide Agios sponsor lot release on all primary and secondary packaging as well as labeling activities of clinical supplies.
• Support QP release for EU clinical trials.
• Gain exposure to the entire Quality Assurance Organization, including Commercial GMP-QA and Quality Systems.

Drug Development Quality Assurance Year 2

• Expand experience to provide quality oversight of all primary and secondary packaging as well as labeling activities of commercial supplies, including review of master batch records, packaging specifications and executed batch records (may start in year 1).
• Expand experience to provide quality oversight of clinical drug product and/or drug substance manufacturing, including review of master batch records, executed batch records, product specifications, and part number generation.
• Collaborate regularly with supply chain and CMC to efficiently deliver clinical supplies while ensuring compliance to all applicable laws and regulations
• Become proficient in clinical inventory system (PMD) as well as electronic document management system (eDMS) and electronic quality management system (eQMS) in support of change controls, CAPAs and effectiveness checks.

About Health Economics and Outcomes Research Fellowship

The Health Economics & Outcomes Research (HEOR) department at Agios is responsible for generating, interpreting, and communicating high quality evidence, supporting assets from early development through post marketing, to demonstrate value and help ensure optimal access to Agios’ therapies. The HEOR team leads patient-centered research via the incorporation of clinical outcomes assessments within trials, evidence generation (real-world evidence, observational studies, systematic literature reviews, etc.), health economics (cost-effectiveness models, budget impact models), and value demonstration. These activities serve to highlight the unmet need and communicate value to key stakeholders including patients, health care providers, payers, and regulatory bodies. As an integral part of drug development and commercialization, the HEOR group continuously collaborates cross-functionally to ensure alignment of strategy across teams.

HEOR Fellowship Goals and Objectives

The goal of the Agios HEOR Fellowship Program is to equip the fellow with technical skills and provide opportunities for real-world application of HEOR in the pharmaceutical industry. Objectives include:

• To provide training and experience in the areas of health economics, outcomes research, and market access.
• To enhance the fellow’s technical expertise in literature evaluation, epidemiology, research methodology, economic modeling, value communication, and biostatistics.
• To provide experience contributing to research and development of investigational products with a focus on clinical outcomes assessments.

HEOR PROGRAM DESIGN

As an integrated member of the HEOR team at Agios, the fellow will achieve a deeper understanding of the complexities surrounding cost and access of pharmaceuticals within the genetically-defined disease portfolio, including pyruvate kinase deficiency, thalassemia, and sickle cell disease. Over two years, the fellow will gain proficiency in the analytical methods required to evaluate pharmaceutical products and communicate their value to patients and other stakeholders within the healthcare system. Agios offers experienced leadership to guide the fellow in key areas of HEOR to become a well-rounded researcher and strategist. Activities and responsibilities over the two years may include and are not limited to the following:

HEOR YEAR 1

Integrate into the HEOR and relevant cross functional teams by collaborating regularly with Clinical Development, Medical Affairs, Market Access, Marketing, and Regulatory

Learn to demonstrate value by contributing to development of value dossiers (AMCP dossier, Global value dossier)

Develop skills in health economics by contributing to budget impact models, burden of illness models, and cost-effectiveness models.

Conduct HTA surveillance, systematic literature reviews, and other evidence generation activities.

HEOR YEAR 2  

Gain experience in patient-centered research and improve understanding of clinical outcomes assessments and their role in drug development and commercialization.

Become proficient in generating real-world evidence (cohort studies, claims analyses, indirect treatment comparisons, etc.) using advanced outcomes research methodology.

About Medical Safety and Risk Management

Medical Safety & Risk Management (MSRM) is responsible for managing the safety profile of products throughout their lifecycle through the continuous monitoring, assessment, and communication of safety information. As such, MSRM plays a key role in the management of emerging safety signals in drug development and for maintaining this activity for products in the post-marketing setting. MSRM also ensures on-time reporting of high-quality individual case reports as well as the creation and timely submission of aggregate safety reports to fulfill global regulatory requirements. In addition, MSRM is an active participant in the creation of clear, consistent, and accurate product labeling, risk management plans, and other product-related information.

MSRM Fellowship Goals and Objectives

The goal of the Agios MSRM Fellowship Program is to give the fellow hands-on knowledge of US and global pharmacovigilance activities including a broad understanding of global pharmacovigilance regulations, pharmacovigilance operations, aggregate safety reporting, signal detection, and risk management across all stages of drug development, from early stage to post marketing.

Objectives include:

• Developing a strong safety risk management knowledge base and applying those skills practically in the arenas of signal detection and signal management
• Obtaining well-rounded knowledge of country-specific pharmacovigilance regulations
• Cultivating strong communications and project management skills
• Collaborating cross-functionally with various departments within the company as a partner in the product development process

MSRM Program Design

As an integrated member of the MSRM team at Agios, the fellow will be provided with a vast array of invaluable opportunities across both the rare genetic disorders and oncology portfolios. These opportunities will enable the fellow to gain real-life pharmacovigilance experience and will deeply enhance their professional knowledge. Over the two years, the fellow will take on projects of increasing scope and responsibility while obtaining a deep comprehension of safety’s roles, responsibilities, and day-to-day tasks, as well as familiarize themselves with the complexities and cross-functional nature of the drug development process.

With the mentorship of the fellowship team at Agios, the fellow will have access to the following career-enhancing activities and responsibilities:

MSRM Year 1

• Work with safety operations colleagues to gain a solid understanding of roles and responsibilities in individual case management and medical review of cases
• Understand global pharmacovigilance regulations and begin applying knowledge to ensure compliance
• Observe how safety interacts with other functions and actively participate in these interactions
• Participate in the creation of aggregate safety reports for submission to regulatory agencies worldwide
• Gain a solid understanding of signal detection, signal evaluation, and risk management activities
• Become integrated in the cross-functional teams
• Review safety sections of the protocol, informed consent form, and other study-specific documents ensuring accuracy and consistency

MSRM Year 2

• Take responsibility for safety for one or more protocols as a Global Safety Lead and represent MSRM at cross-functional meetings
• Author/review safety sections of the protocol, informed consent form, and other study-specific documents
• Participate in generating responses to safety-related health authority queries
• Contribute to the creation and maintenance of Safety Risk Management Plans
• Contribute to the safety sections of the Investigator’s Brochure and aggregate reports including DSUR, PBRER, and PADER
• Assist with the coordination of the Safety Management Team for the assigned product

About the Regulatory Affairs Fellowship

Through the Regulatory Affairs Fellowship, the fellow will obtain in-depth and hands-on experience in Regulatory Affairs, with the opportunity to complete rotations to gain exposure to various Regulatory Affairs functional areas, namely Clinical Strategy; Chemistry, Manufacture, and Controls (CMC); and Regulatory Operations. Upon completion of the program, the fellow will have gained the foundational knowledge and key competencies to begin a career in the dynamic field of Regulatory Affairs.

About Regulatory Affairs – Clinical Strategy

Regulatory Affairs –Clinical Strategy (RA–CS) professionals are responsible for developing regulatory strategy and leading health authority interactions to support global drug development from pre-IND through registration and life cycle management. They manage the planning, coordination, and execution of high-quality regulatory submissions of original applications (i.e. NDAs/MAAs, INDs/CTAs) and any subsequent major amendments, supplements, and variations. The RA–CS team interfaces with a diverse cross-functional team to ensure the regulatory strategy is aligned with the overall clinical development plan and corporate objectives.

Goals and Objectives

The goal of the RA-CS rotation is to provide the fellow with hands-on global experience in clinical strategy in order to develop a comprehensive understanding of this regulatory affairs’ role in the drug development process from early stage to post-marketing.

Objectives include:

• Develop a strong global regulatory strategy skillset while actively contributing to regulatory planning and submissions
• Obtain well-rounded knowledge of country-specific regulatory processes
• Cultivate strong communication, leadership, and project management skills by collaborating cross-functionally with numerous departments within the company, as well as with external vendors, as a partner in the drug development process.

About Regulatory Affairs – Chemistry, Manufacturing, and Controls (CMC)

Regulatory Affairs – Chemistry, Manufacturing, and Controls (RA–CMC) is a specific function responsible for providing regulatory strategy and leadership for the CMC/quality aspects of pharmaceutical development programs and product registration, including global regulatory submissions and CMC-focused health authority interactions. The Fellow will work with the RA–CMC team in the planning and execution of quality aspects of clinical trial applications ranging from Phase I through Phase 3 (i.e. INDs, Investigational Medicinal Product Dossiers, Quality Overall Summaries), and commercial marketing applications (i.e. NDAs, MAAs). This team collaborates with a diverse cross-functional team, including technical operations, quality assurance, supply chain, and clinical development, to assess the impact of quality/manufacturing changes, determine the appropriate regulatory filing strategy/timelines, and prepare the appropriate amendments, supplements, and variations.

Goals and Objectives

The goal of the RA-CMC rotation is to give the fellow hands-on knowledge of US and Global RA-CMC strategy and tactics, including an understanding of quality pharmaceutical development, CMC dossier planning and development, and health authority interactions.

Objectives include:

• Develop a strong understanding of US and global CMC regulatory guidance and industry practice
• Obtaining well-rounded knowledge of the process for planning, preparing, and submitting CMC dossiers
• Cultivate strong communication and project management skills by collaborating cross-functionally with numerous departments within the company as a partner in pharmaceutical development.

About Regulatory Affairs – Operations

Regulatory Affairs – Operations is a specific function within Regulatory Affairs that is responsible for the coordination of essential documentation and resources required for the filing of global applications. Additionally, they provide tools, training, and overall support to submission stakeholders while proactively implementing efficient information management, scalable operational excellence, and innovative digital solutions. They partner with other functions (e.g. Clinical and Quality) to be in line with the requirements from health authorities within regulatory information systems and provide a clear framework internally to maintain streamlined submission timelines.

Goals and Objectives

The goal of the RA-Operations rotation is to provide the fellow with hands-on knowledge and experience with the planning and operational management of Regulatory submissions, including exposure to submission systems, processes, and industry standards and guidelines.

Objectives include:

• Develop an understanding of the eCTD structure required for health authority submissions and the systems and tools used to support the management of regulatory information and submission development
• Obtain exposure to and assist with the preparation of submission deliverables for eCTD submissions to FDA and other health authorities, coordinate activities with external publishing vendor 
• Cultivate strong communication, project management skills, and a working knowledge of the regulations and processes that govern the content and maintenance of controlled documents required by the FDA, EMA, and ICH

Regulatory Affairs Fellowship Program Design

The Regulatory Affairs Fellowship follows a rotational development plan in the first year, with ~3-month rotations across different Regulatory focus areas, namely Clinical Strategy, Chemistry, Manufacturing, and Controls (CMC), and Regulatory Operations. The fellow will gain hands-on experience in multiple positions within an accelerated period, providing a unique opportunity to clarify their interests in the diverse field of Regulatory Affairs. In each rotation, the fellow will have the opportunity to integrate with working groups and learn to navigate the team environment at Agios. Following the completion of the rotational structure, fellows will select the Regulatory function in which they will focus for the second year of the fellowship, tailored to their personal interests, strengths, and targeted areas for development.

Activities and responsibilities over the two years may include and are not limited to the following:

Year 1: Regulatory Affairs Rotation in Clinical Strategy, CMC, and Operations

• Rotate through the 3 Regulatory Affairs functions at Agios (Clinical Strategy; CMC, Regulatory Operations) to gain exposure to various Regulatory disciplines and guide selection of the area of focus for Year 2 of the fellowship.
• Integrate into cross-functional teams (e.g., Technical Operations/CMC; Quality Assurance; Clinical Trial Working Group) to understand Regulatory Affairs’ roles and responsibilities and gain familiarity with the complex cross-functional nature of the drug development process.
• Support Regulatory leads in planning and executing Regulatory submissions to support Agios’ clinical development programs.
• Gain an understanding of Regulatory requirements, guidance documents, and resources for US and ex-US drug development, including timelines and processes for US IND submissions and ex-US clinical trial applications.
• Gain a complete understanding of Agios’ portfolio, processes, and the applicable systems used for regulatory planning and health authority submissions

Year 2: Deep Dive in Selected Regulatory Focus Area 

• Increased responsibility and ownership for planning, preparation, and submission of Regulatory documents to global Health Authorities 
• Observe how to develop regulatory strategy, including a deep dive into global regulatory requirements and research of Regulatory intelligence and precedent
• Assist with planning, preparing, and tracking responses to Health Authority queries 
• Represent Regulatory Affairs on cross-functional team sub-teams; provide Regulatory recommendations and guidance to cross-functional teams 
• Gain experience with planning and preparing for Health Authority meetings.
• Cultivate communication and submission management/project management skills