Job Description
Who We Are For more than two decades, going our own way has led to countless breakthroughs, bettering the lives of those suffering from rare genetic disease. In 1997 we were founded to make a big difference in small patient populations. Now we seek to make an even greater impact by applying the same science-driven, patient-forward approach that propelled our last 25 years of drug development to larger genetic disorders, as well as genetic subsets of more common conditions. Through our unparalleled expertise in genetics and molecular biology, we will continue to develop targeted therapies that address the root cause of the conditions we seek to treat. Applying our knowledge to make a transformative impact is not just a calling, but an obligation to those who will benefit most. The end goal has always been better lives and now we can reach more. And the more people we reach, the more our impact can grow. We transform lives through genetic discovery. Our Culture Our desire to make a positive impact on our patients extends to our employees and BioMarin is committed to fostering an inclusive environment where every person feels seen, valued, and heard \u2013 so employees can thrive in all areas of their lives, in and outside of work. We seek to provide an open, flexible, and friendly work environment to empower people and to provide them with the ability to develop their long-term careers. Ultimately, we want to be an organization where people enjoy coming to work and take pride in our efforts to help patients. BioMarin Early Clinical Development (ECD) is responsible for early clinical development programs from initiation of investigational new drug (IND) to completed proof of concept (POC) studies. The Early Clinical Development Team provides leadership for clinical strategy and oversight to ensure excellence in clinical trial conduct, data analysis and interpretation, publication preparation, and safety monitoring.\u00A0 \u00A0 The\u202FAssociate Scientific Director / Associate Medical Director\u202Fis an emerging leadership position within ECD. She/he leads tasks related to a gene therapy program core deliverables on execution, data analysis and regulatory filings as delegated by the Early Clinical Development Team lead. This includes developing key documents to support study execution;\u202Fbuilding and leveraging relationships with external parties (KOLs, Investigators); conducting data analysis in support of reports and key messaging\u00A0The\u202FAssociate Scientific Director / Associate Medical Director\u202Fwill primarily take on the role of Medical Monitor for an ongoing study in the field of Hereditary Angioedema and will keep close contact to study sites in the US. \u00A0 Responsibilities may include, but are not limited to:Clinical Study Monitoring\u00A0 Act as the scientific subject matter expert and primary medical contact to study sites for assigned clinical study; take a proactive approach to identifying issues and mitigating risk at study site level\u00A0 Provide strong leadership to sites by developing or participating in training, answering investigator/site/HA questions about the protocol Conduct review, assessment and interpretation of clinical and safety data to ensure that the data are correct and presented with the appropriate interpretation including thorough review of SAEs and other important AEs (per the study specific Medical Monitoring Plan) Conduct periodic review of protocol deviations in collaboration with Clinical Operations Study Lead or designee (per protocol specific Protocol Deviation Plan) Attend and present at Investigator Meetings, as needed Provide leadership in compliance with GCP and appropriate reporting standards Work closely with the Pharmacovigilance representatives providing medical input into safety reports including, SAE narratives and analysis of similar events, Development Safety Update Reports (DSURs) and Suspected Unexpected Serious Adverse Reactions (SUSARs) reports, Company Core Safety Information (CCSI), Investigator Brochure (IB), Risk Management Plans, Integrated Summaries of Safety and Efficacy, Clinical Study Reports and preparation of labels Collaborate with Biometrics to identify key issues, prepare content and facilitate discussions at study Data Review Board (internal) or Data Monitoring Committee (DMC) Lead the selection of and interactions with independent Data Monitoring Committees (DMC) Participate in and provide scientific advice, as appropriate, during key Database Lock (DBL) activities (final listing review, review of blinded tables, listings and figures (TLFs), etc.) and actively participate in all data snapshots taken (not limited to final DBL) Contribute to and review Clinical Study Report (CSR) writing (review final TLFs, work with medical writer to review & revise CSR document, draft discussion and conclusions, participate in comment resolution meetings) Present study updates and topline results to CLS leadership, as needed The Associate Medical Director will also act as the medical subject matter expert for review of clinical and safety data to ensure data are correct and presented with the appropriate medical interpretation and for discussing safety concerns with site Provide timely and high-quality functional deliverables and contributions to Study Execution Team (SET) Act as primary point of contact between SET and ECD\u00A0 to ensure appropriate dissemination of information and communication (including functional managers and other relevant individuals) Contribute to the development and maintenance of study- specific plans; manage the development of study-specific plans\u00A0 Escalate issues affecting ECD function deliverable quality, timelines, resources or budget, as appropriate\u00A0Scientific Expertise\u00A0 Develop key documents to support the components of trial execution and regulatory submission (including IND Annual Reports, Investigator Brochures, Briefing Books and Pediatric Plans) Develop scientific rationale for methods, design and implementation of clinical protocol changes, data collection systems and final reports Support therapy-specific publication plans and work with internal and external colleagues to prepare study results for timely publication Attend and contribute to relevant scientific conferences, seminars or presentations Engage and establish rapport with KOLs and scientific or clinical experts Contribute to content and submission of abstracts to key symposia Work with Clinical Operations to ensure the success of Investigator Meetings by contributing and presenting scientific content\u202F\u00A0Data Analysis\u00A0 Develop thorough understanding of clinical trial data to help guide the drafting of CSRs and Key Messages. Perform preliminary analyses on clinical study data as it becomes available to help build and guide Statistical Analysis Plans (SAPs) Help define study quality metrics and perform study data reviews during study execution to ensure integrity of accruing study data\u202FServe as CLS representative and liaise with Pharmacovigilance, Clinical Operations, Medical Affairs, Biometrics, Regulatory Affairs, Data Monitoring Committees (DMCs) and other internal or external entities as needed\u00A0 PLEASE NOTE: Absent a Medical or Religious reason that prohibits vaccinations, all our incoming employees must be vaccinated for COVID-19. US remote based position, BioMarin headquarters are in San Rafael, CA.Salary range posted is for a Medical Doctor (MD), however we will also consider a PhD with relevant clinical trial experience (medical monitoring) from Pharma/ CRO, and therapeutic experience is not a must.\u00A0 The salary range for a non MD is $140,000 to $210,000. We are an equal opportunity employer and all qualified applicants will receive consideration for employment without regard to race, color, religion, gender, gender identity, sexual orientation, national origin, disability status, protected veteran status, or any other characteristic protected by law.
