Work at ROCKET PHARMA and help cure rare diseases!

Rocket Pharma is a fully integrated, clinical-stage company advancing gene therapies with curative potential for multiple rare childhood diseases. Rocket places enormous value on people and considers team member progress and well-being to be as important as the progress of its pipeline. We are looking for hands-on team players who enjoy collaborating with colleagues. We share a passion for seeking gene therapy cures for devastating diseases and making a difference for patients. This is an opportunity to become an integral part of a small, highly productive team and to help grow an entrepreneurial, scientifically driven organization that is increasingly recognized as a leading biotechnology innovator. In addition to a competitive compensation package featuring a generous 401K match and stock options, the position includes excellent health benefits.

The Director Real-World Data Analytics & Innovation position is a unique opportunity to join a dynamic team to make an impact in the lives of patients with rare diseases. This role is responsible for developing and implementing evidence strategies to deliver innovative and robust evidence supporting Rocket clinical programs. These evidence strategies will inform business decisions about the research and development of Rocket products and support healthcare decisions by patients, physicians, health authorities, payers and policymakers. The role will serve as an expert for real-world evidence (RWE), being a core member of cross-functional teams, personally owning the strategy on real world evidence requirements and execution, ‘fit for purpose’ data source assessment, study design and robust analytic methodologies, ultimately driving the development of integrated evidence approaches that support programs across the full lifecycle.

The Director Real World Data Analytics & Innovation will work closely with the Head of RWE and other team members; and will collaborate extensively with the HEOR, Value and Access and Clinical teams. Excellent interpersonal and communication skills will be necessary to support effective partnership with other key internal stakeholders from Value and Access, Marketing, Medical and Clinical Development. Demonstrated ability to effectively collaborate with external stakeholders including data partners, patient advocacy groups and disease area/HEOR/RWE experts will be required, as will a strong understanding of EU/US real world data sources and the regulatory/payer requirements for impactful evidence generation. An innovative mindset, high attention to detail, a data-driven problem-solving attitude and a multi-stakeholder cross functional approach will be key.

• Develop, own and drive innovative integrated evidence strategies, in collaboration with cross-functional partners (e.g. medical affairs, value & access, clinical, regulatory), to ensure the value of Rocket therapies are fully supported by evidence
• Ask the right scientific questions to identify the evidence needs to optimize regulatory approval, market access and clinical adoption of therapies
• Provide subject matter expertise on ‘fit for purpose’ data and evidence generation solutions, considering a holistic and efficient product lifecycle perspective
• Collaborate with internal and external partners ensuring scientific rigor in study design and analyses
• Represent the Real-World Analytics & Innovation team as a core member of internal cross-functional teams and initiatives to ensure the expertise and perspective of the team is represented in strategic plans for Rocket therapies
• Responsibility for project management and study execution of RWE studies
• Own development of RWE study documentation (e.g. protocol, analysis plan, report) in line with applicable processes
• Publish and communicate study results in collaboration with internal partners, as aligned with the Rocket publication strategy
• Mentor junior team members, as required, to ensure competence and development in epidemiologic skills and knowledge

• PhD, PharmD, MD, or equivalent (Education in a discipline related to health services research, epidemiology, biostatistics, or public health is a plus)
• 10 years of RWE-related experience, with a preference for experience in rare/complex diseases
• Demonstrated experience in applying observational research methods to RWE sources across various use cases, such as early-stage clinical development, support to regulatory approvals, safety commitments and HTA submissions required
• Understanding of regulatory and HTA requirements as they relate to evidence generation
• Direct experience in designing and conducting observational research, including protocol writing, analysis plan and study report development
• Experience with various data sources, such as electronic medical records, registries and/or medical claims databases is preferred
• Advanced experience in a pharmaceutical or biotech organization is preferred, with demonstrated success working within matrixed cross-functional teams and collaborating with both internal and external partners
• Demonstrated ability to function with autonomy and develop productive cross-functional collaborations
• Demonstrated strategic thinking and strong leadership skills
• Ability to synthesize and convey complex data or concepts in an understandable way, uncovering key messaging in support of Rocket strategic objectives
• Strong interpersonal skills
• Ability to effectively project manage and meet timelines
• Excellent written and oral communication

A diverse workforce fosters innovation and strengthens Rocket’s business. We ensure equal opportunity without discrimination or harassment in the workplace on the basis of gender, race, color, religion, national origin, age, physical or mental disability, pregnancy, citizenship, status as a protected veteran, marital status, sexual orientation, gender identity and expression, genetic information, or any other characteristic protected by applicable local, state, and federal laws.