Albireo Pharma Overview:
Albireo is a clinical-stage biopharmaceutical company focused on the development and potential commercialization of novel bile acid modulators to treat orphan pediatric liver diseases and other liver or gastrointestinal diseases and disorders. Albireo has deep expertise in bile acid biology and a pipeline of clinical and nonclinical programs.
The company’s first commercial launch will be Odevixibat, which is a potent and selective inhibitor of the ileal bile acid transporter (IBAT), sometimes referred to as the apical sodium dependent bile acid transporter (ASBT), that has minimal systemic exposure at therapeutic doses and acts locally in the gut.
Albireo is developing Odevixibat initially to treat patients with PFIC, a rare genetic liver disease, and plans to consider additional development in other pediatric cholestatic liver diseases and disorders in the future. Our Phase 3 program in PFIC includes a single randomized, double-blind, placebo controlled, multicenter clinical trial and an open-label long-term extension study. The double-blind trial, called PEDFIC1, is underway with data read out anticipated in mid- 2020.
The U.S. Food and Drug Administration (FDA) has granted to the Odevixibat PFIC program or elements of its fast track, rare pediatric disease and orphan drug designations. The European Medicines Agency (EMA) has granted Odevixibat orphan designation, as well as access to the Priority Medicine’s (PRIME) scheme for the treatment of PFIC. Its Pediatric Committee has agreed to Albireo’s Odevixibat Pediatric Investigation Plan for PFIC. Both FDA and EMA also have granted orphan drug designation to Odevixibat for the treatment of Alagille syndrome and primary biliary cholangitis. Albireo will commercialize Odevixibat in the US, Europe & Canada and is pursuing potential partners in remaining regions.
Reporting to VP of Biometrics , the Director of biostatistics within the Biometrics Department is expected to act independently as the statistics subject matter expert for all clinical development programs and studies, including oversight of data management and statistical programming activities, while ensuring adherence to all regulatory requirements. H/she is responsible for providing broad statistical support and contributing strategically to project decisions, with a focus on clinical planning, study design, protocol development, sample size / power calculations and simulations, statistical analysis methodology, statistical analysis plan preparation, blinded in-trial data quality review, pre-database lock data quality control, supporting development of table / listing / figure templates, programming and production, oversight of contract or CRO resources, as well as preparation, integration and documentation of clinical regulatory submission package, in accordance with CDISC standards.
The Lead Biostatistician is expected to have an in-depth understanding of advanced statistical methods, including innovative trial designs and endpoints, missing data and multiplicity handling. She/he will have a thorough understanding and capability to implement state-of-the-art innovative statistical methodology for the design, execution and evaluation of clinical trials. In addition, she/he will have the capacity to understand the relevant multi-disciplinary knowledge and interact effectively within the biometrics deparment and also with clinicians, clinical operations, medical writing, regulatory affairs, information technology and other colleagues, service/technology providers, as well as external drug development agencies or organizations. She/he will have a good understanding and the capacity to sustain the dialog in consultations or advisory board meetings with biostatistical, clinical and regulatory key opinion leaders about clinical study design, execution and evaluation from the statistical perspective.
Key Duties and Responsibilities:
- Extensive collaboration with integrated clinical teams to design studies, develop statistical analysis plans, and draft statistical reports / manuscripts for clinical studies in all phases for FDA/MAA, as well as for other global regulatory authorities
- Provide strategic and statistical input to Albireo clinical programs and clinical study teams, as well as advisory board or expert meetings
- Actively contribute to what and how data are collected, be familiar with CDISC data structure, formatting and documentation standards for clinical studies, as well as integrated safety and efficacy datasets
- Review documents related to clinical data management, including (e)CRFs, EDC, eSource/eCOA/ePRO and data management validation plans and their deployment
- Prepare randomization specifications and generate or oversee the setup of randomization processes and systems, including enrichment trial designs
- Plan, program and execute sample size and assumptions calculations and simulations for conventional, adaptive and enrichment trial designs, reflecting the actual methodology planned for the study evaluation, including statistical model, missing data and multiplicity handling, as well as sensitivity analyses
- Design, develop, modify, document and evaluate the technical data processes, systems and infrastructure to expedite and evaluate analysis and reporting of clinical studies
- Ensure accuracy, precision, efficiency, robustness in statistical planning, study design, statistical analysis, interpretation, reporting, and presentation of clinical study results
- Evaluate and interpret clinical trial data, write up reports, prepare slides and present results to internal and external stakeholders including regulatory agencies
- Assist in drafting and review of clinical study reports and statistical methodology and results sections of documents prepared for scientific communication (i.e., reports, manuscripts, posters, abstracts), including posting of results in public study registries
- Contribute to the selection, information, oversight and evaluation of expert consultants, contractors, CROs and other partners providing statistical services
- Participate in multi-disciplinary meetings and teleconferences and provide consulting, interpretive, and analytical support
- Identify and plan statistical tasks, timelines, resources and account for critical dependencies and resources in order to estimate, agree and ensure that statistical work meets deadline commitments and quality standards in a timely manner
- Lead projects by managing statistical tasks, resources, timelines and budgets as well as act as point of contact for internal and external teams including consultants, contractors, CROs and other vendors
- Contribute to the definition and oversight of the setup, methodology, tools decision rules, support and implementation of DMCs and ability to direct, mentor junior statisticians, data managers, and programmers
- Take an active role in task forces or process-improvement groups and assist in providing guidance related to biostatistics, programming, and data management
- Maintain exhaustive program and clinical trial documentation, including design, execution, evaluation, review, QC, tracking information, as well as internal and external communications
- Perform peer review of other statisticians’ work; Perform QC of analysis datasets, data displays for efficacy and safety variables
- A Ph.D. (or equivalent degree) in Biostatistics or Statistics with a minimum of 10 years of relevant clinical biostatistics experience in pharma, biotech or medical device industry -OR- a Master’s degree and a minimum of 12 years of relevant industry experience is required
- Experience of rare disease clinical development and NDA is highly preferred; Comprehensive knowledge of statistical theory and methods
- Demonstrated ability to apply statistical, programming, and data management knowledge to clinical studies and other supportive tasks
- Expertise in SAS and R programming for data management, conversion, review and visualization, statistical analysis, tabulation, listing and graphs of clinical trial data
- Excellent verbal and written communication skills; Leadership and team-ability with effective intercultural competences, diplomacy, negotiation and communication
- Self-management skills with the ability to take initiatives, develop and evaluate alternative scenarios and options, and present them effectively to the colleagues and leadership of the company
- Proficiency in MS Word, Excel, and PowerPoint software programs
- Excellent problem-solving and strategic-thinking skills
- Sound knowledge of relevant clinical and statistical regulatory requirements (e.g., GCP, ICH, CDISC, etc.)
- Excellent written and oral communication and presentation skills
- Ability to identify and address issues proactively in a timely manner
- Ability to make appropriate, rational and well-justified decisions in ambiguous situations with incomplete or uncertain information; Ability to prioritize tasks and direct team accordingly