Do you have a passion for generating and interpreting data to increase the understanding of how medicines work in real life? Do you want to be at the forefront of creating new evidence standards for a new branch of medicine – gene therapy? Join bluebird bio’s enthusiastic and collaborative team to contribute to the overall success of our novel gene therapy approach. Within Medical Affairs, you will lead RWE activities for β-thalassemia; you will develop and implement the integrated RWE strategy for our β-thalassemia medicines globally. You will lead the development, launch, and implementation (in partnership with the operation group) of a post-marketing registry to characterize the real-world outcomes of betibeglogene autotemcel, bluebird’s gene therapy for transfusion dependent β-thalassemia (TDT), accounting for the needs of the patient and clinical community and expectations from global regulatory agencies and payers. You will participate in regulatory interactions around the registries and RWE for β-thalassemia. You will lead additional observational studies defined by the Medical Affairs and β-thalassemia teams as needed, which may include chart reviews and investigator-sponsored research. The position is preferably based in Cambridge, MA and reports to the Head of RWE.
About the Role:
- Act as the RWE lead for β-thalassemia, responsible for the strategy and design of the global post-marketing registry, including close collaboration with team members & vendors responsible for the start-up and execution of the registry.
- Provide leadership to an internal cross-functional RWE team of regulatory, medical, scientific communications, pharmacovigilance, clinical, patient advocacy, health economics, market access, commercial, biostatistics, data management, and site operations colleagues to identify evidence needs across external and internal stakeholders and develop and implement the evidence generation plan to meet those needs.
- Serve as the internal point of contact for RWE needs in β-thalassemia, actively engaging with and contributing your expertise to the medical strategy team, program team, and commercialization team. Serve as the RWE subject matter expert for regulatory dialogue for TDT.
- Oversee the design and execution of RWE projects, from study planning to report or publication, in collaboration with internal colleagues, clinical centers, contract research organizations, and other external partners.
- Lead the development and amendments of study protocols and analysis plans. Ensure the quality, completeness, and relevance of real-world data.
- Develop strategies to utilize the strengths and minimize the limitations of real-world datasets, including disease and transplant registries and other clinical data sources, in the context of designing studies utilizing real world data.
- Develop and manage internal the β-thalassemia budgets and timelines for all RWE projects. Liaise with site operations and business operations on study contracting, invoicing, and financial tracking.
- Advanced degree in outcomes research, epidemiology, public health, health economics, pharmaceutical sciences, life sciences, or related discipline required (Masters, PharmD, PhD, MD, MPH)
- Experience and passion working with underserved and underrepresented communities
- Experience developing and implementing registries for regulatory purposes is a plus
- Experience with observational or clinical research experience, preferably in rare diseases; must have experience in study design and conduct, as well as the analysis, interpretation, and communication of data; industry experience preferred
- Experience working with a wide variety of external stakeholders including, but not limited to, physicians, researchers, payers, regulators, and patient groups; proven track record of building strong relationships with research collaborators
- Outstanding interpersonal and communication skills; ability to communicate information/evidence to multiple internal and external audiences (both lay and experienced, including senior leadership), with strong written and verbal presentation skills
- Experience leading cross-functional teams; strong project management/organizational skills, experience interacting with clinical research organization, study activation, contracting and other activities that are part of clinical study management.
- Able to travel in the US and internationally as needed <20%
- An unwavering commitment to upholding the highest ethical, legal, regulatory, and scientific standards; familiar with the relevant standards and guidelines for post-marketing commitments