Head of Rare Metabolic/Renal (MD) Clinical Research

Cambridge, MA
Sep 24, 2021
Required Education
Position Type
Full time


Pfizer Rare Disease is pursuing projects in rare metabolic and renal indications with a view to expanding the clinical portfolio in these areas of very significant unmet patient need. Three early phase clinical programs (P1/2) are currently running with multiple programs proximal to first in human studies and in discovery. The programs span multiple indications and pharmacological modalities, including small molecules, biotherapeutics and AAV gene therapy. The Rare Disease Research Unit is keen to identify an creative, innovative research focused physician with significant experience in inborn errors of metabolism, or other metabolic, renal or bone disease with a background in genetics, clinical care or experimental medicine (biomarkers, imaging, clinical trial endpoint development) to lead the clinical translation and early development strategy for the selected indications. Some industry experience is essential although for the right candidate, further training may be provided to fill out any areas of relative inexperience in drug development.

  • To guide drug discovery and to lead the early clinical development of rare metabolic/renal/bone programs with a particular focus on monogenic disease.

  • To extend and build the current rare disease metabolic/renal/bone portfolio through new project initiation, in licensing and efficient progression of current programs

  • Responsible for the guidance of discovery teams in rare metabolic disease regarding indication selection, population identification, and target selection

  • Responsible for the formulation of clinical development plans at candidate selection

  • Responsible for the early clinical development of rare metabolic projects – conception and leadership of clinical strategy for Rare metabolic projects.

  • Partner with colleagues in clinical pharmacology, statistics, digital and precision medicine to optimize innovative plans for compound and efficacy evaluation.

  • Responsible for the development of natural history, biomarker, and translational strategies for rare metabolic/renal and bone disease

  • Build, develop, and lead a motivated and engaged team of line and matrixed clinicians who are responsible for the day-to-day design and execution of rare metabolic clinical programs

  • Provide oversight of the rare metabolic clinical team, including:

    • Lead preparation of clinical sections of all relevant regulatory filings (IND, study reports, NDA/BLA, etc)
    • Oversee management of studies and serve as expert on clinical and medical issues
    • Oversee review of study data, sometimes including safety monitoring and SAE reporting
    • Oversee writing of final study reports and preparation of documents for submission
  • Lead interactions with business unit and commercial colleagues with regard to clinical elements of progression agreements into late stage development including definition of POM/POC success criteria

  • Interact with key opinion leaders and clinician scientists to ensure implementation of latest clinical thinking and guidelines into clinical development plans

  • Serve as medical and technical resource for clinical issues raised by internal and external collaborators, investigators, consultants and contractors relevant to rare metabolic programs

  • Represent the company at scientific meetings and presentations, interactions with rare metabolic disease foundations and PAGs

  • Assist in the development of publications, abstracts, and presentations

  • Support business development activities as needed, including due diligence activities


Candidate demonstrates a breadth of diverse leadership experiences and capabilities including: the ability to influence and collaborate with peers, develop and coach others, oversee and guide the work of other colleagues to achieve meaningful outcomes and create business impact.

  • A clear working knowledge of the clinical development process

  • A proven track record of scientific and operational success within a large matrixed environment

  • Excellent understanding of pathophysiology, clinical phenotype and genetics of rare metabolic/renal or bone disease

  • Demonstrated ability to formulate research and development plans

  • Clinical background in industry or academia, in metabolic/renal or bone disease

  • Full grasp of the clinical development process

  • Strong organizational skills and attention to detail

  • Ability to interface with senior management and enhance the credibility of the rare disease unit

  • Excellent written and oral communication skills


  • MD or equivalent medical qualification (MD, PhD preferred)

  • Deep understanding of the biopharma environment and the drug development process; 5+ years of relevant experience in drug development with proven track record of success in executing development programs to completion

  • Strong interpersonal skills and expert team player with demonstrated ability to build consensus and drive change across all levels of the organization, including senior management

Other Job Details:

Eligible for Relocation Package

Eligible for Employee Referral Bonus


Pfizer requires all U.S. new hires to be fully vaccinated for COVID-19 prior to the first date of employment.  As required by applicable law, Pfizer will consider requests for Reasonable Accommodations.

Sunshine Act

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EEO & Employment Eligibility

Pfizer is committed to equal opportunity in the terms and conditions of employment for all employees and job applicants without regard to race, color, religion, sex, sexual orientation, age, gender identity or gender expression, national origin, disability or veteran status.  Pfizer also complies with all applicable national, state and local laws governing nondiscrimination in employment as well as work authorization and employment eligibility verification requirements of the Immigration and Nationality Act and IRCA.  Pfizer is an E-Verify employer.