MPS I Registry Project Lead (Real-World & Late-Phase Research) $110K-$125K | Durham, NC or remote (U

Have you built your career running real-world or late-phase studies at a CRO, pharma, or biotech company, and found yourself wanting to meet the people your work is for?
For more than 50 years, the National MPS Society has worked alongside families to find new therapies and, one day, a cure for MPS and ML. We're looking for a Registry Project Lead to guide our international MPS I Registry, a resource that helps shape regulatory decisions, clinical care, and the development of new therapies for individuals with MPS I.
This isn't a role where you'll manage one study among many. As the sponsor lead, you'll set the Registry's priorities, hold our CRO accountable for quality, timelines, and a budget of $0.5M or more, and keep the work compliant with ICH GCP and audit-ready. You'll turn data into reports and publications that move the field forward. You'll also partner with Society leadership to build a sustainable business model for the Registry and help grow it into a family of registries for related disorders.
We're looking for someone with significant experience overseeing observational or registry studies, a working knowledge of RWE methods and data governance, a track record of directing vendors, and the ability to explain complex science clearly to clinicians and families alike. An advanced degree and rare disease experience are a plus.
You'll join a team of 10 that works directly with leading international experts, and at our conferences, you'll meet the families behind the data. In an ultra-rare disease, every well-run analysis makes a measurable difference, and every data point represents a person and a family.
Organization: National MPS Society, Inc., a 501(c)(3) nonprofit patient advocacy organization
Location: Durham, NC (onsite) or remote within the U.S. with expectation to work EST hours.
Employment: Full-time
Salary: $110,000–$125,000 per year
Travel: Up to 10%
Staff size: 10
The role

The National MPS Society is looking for a Registry Project Lead to manage the MPS I Registry on the Society's behalf. In this role you make sure the Registry produces reliable real-world evidence that helps families, clinicians, researchers and regulators understand and treat MPS I.
A contract research organization (CRO) runs the Registry day-to-day. You are the Society's sponsor lead: you set expectations, hold the CRO accountable, make the key decisions, and make sure the work reflects the Society's mission and the needs of the patient community. This is a hands-on leadership role in a small, mission-driven organization where your expertise and judgment directly shape the program and its outcomes.
What you will manage

You are accountable to the Society for the Registry's success: its scientific integrity, compliance, governance, timelines, budget, and value to the community. The CRO carries out the operational work, and you direct and oversee it with the goal of expanding the international registry into other related disorders over time to become an umbrella of registries.
You report to the National MPS Society CSO and work closely with Society leadership, the Scientific Advisory Board, and the Registry's clinical and industry partners.
Key responsibilities

Registry leadership and sponsor governance

Set the Registry's annual priorities and success measures with Society leadership, keeping them aligned with the Society's mission and the community's needs.
Serve as the Society's primary sponsor contact for the CRO's Project Leads, Project Directors and functional leads.
Track Registry progress, keep sponsor governance consistent across all CRO activities.
CRO oversight

Hold the CRO accountable for timelines, scope, quality and budget.
Monitor operational metrics, dashboards, risk indicators and KPIs; identify gaps and direct the CRO to resolve them promptly.
Review and approve key documents, including Project Management Plans, TMF Plans, monitoring and site management plans, and data management documentation.
Give the CRO timely feedback, corrective direction, elevate issues as needed to CRO leadership and follow through until issues are resolved.
Oversee critical activities such as interim analyses, database locks, periodic Registry reports, and any study closeout activities.
Quality, compliance and financial stewardship

Keep the Registry compliant with ICH GCP, applicable regulations, SOPs and data privacy requirements, and keep it ready for audit.
Manage the Registry budget, forecasts and CRO contract, including scope changes.
Partner with the CSO and CEO to develop and carry out a sustainable business model for the Registry, including funding partnerships, data-access agreements and long-term financial planning.
Evidence, communication and community

Prepare Registry summaries, reports, abstracts and publications with minimal oversight.
Explain complex scientific and operational information clearly to clinicians, researchers, industry partners, Society staff and the Board, and to patients and families.
Work with Society colleagues to share what the Registry is learning with the MPS community and to encourage participation.
Qualifications

We care most about what you have done and how you do it. If you meet most of the required qualifications and are excited about this work, we encourage you to apply.
Required

A bachelor's degree in life sciences, nursing, public health, epidemiology or a related field, plus four or more years of relevant experience. An equivalent combination of education and highly relevant experience will be considered.
Two or more years of managing or overseeing real-world, late-phase or non-interventional studies in a CRO, pharma, biotech, academic or registry setting.
A working knowledge of ICH GCP, regulatory expectations for observational research, and real-world evidence methods.
A working knowledge of data science including data governance, data-use agreements, principles of data sharing and interoperability to ensure data is manages safely, legally and effectively.
A track record of evaluating deliverables and guiding vendors or CRO teams to high-quality results.
Experience managing project budgets (of $0.5M or greater), forecasts and vendor contracts.
Strong written and verbal communication, including explaining complex scientific and operational information to technical and non-technical audiences and track record of publications in peer-reviewed journals.
Genuine interest in working in a patient-centered organization alongside the families we serve.
Preferred

An advanced degree (MS, MPH, MSN, PhD, PharmD or similar).
Direct experience supporting or overseeing patient registries, with a track record of publications and presentations.
Experience with metabolic or other rare diseases.
Experience preparing abstracts, manuscripts or regulatory reports from registry or observational data.
Experience working with patient advocacy organizations or patient communities.
If you have built your career in pharma or at a CRO, this role offers something those settings rarely can: the chance to see who your work is for.
Mission you can see. You will meet the children, adults and families whose data make up the Registry, at our family conferences and in our community. The evidence you help produce goes directly toward better care for them.
Real ownership. You will not be one of many study managers in a large portfolio. You will lead the Society's Registry, and your decisions will shape it.
Influence beyond the study. Registry evidence feeds into regulatory decisions, clinical guidance, newborn screening follow-up, and the development of new therapies across the MPS field.
A small team with a big footprint. Our staff of 10 works directly with our Board, our Scientific Advisory Board of international experts, leading clinicians, and industry partners.
Visible impact in an ultra-rare community. In a disease this rare, each well-run analysis and publication makes a measurable difference, and families notice.
Flexibility. You can work onsite in Durham, NC or remotely, with limited travel.
About the National MPS Society

The National MPS Society exists to find cures for the MPS and ML II/III disorders. For more than 50 years we have brought together research, advocacy and family support, serving more than 2,600 members on a $3 million annual budget.
Research: a family-funded extramural research program that awards $750,000 to $1,000,000 each year.
Advocacy: we led the effort to add MPS I and MPS II to the federal Recommended Uniform Screening Panel (RUSP) for newborns.
Family support: our Pathways Program gives intensive social work support to families in their first year after diagnosis, and our Family Support program stays with families throughout their journey.
An active Board of Directors and a Scientific Advisory Board of international experts guide our work, and we are recognized as a leader in the rare disease community. Learn more atmpssociety.org.
The National MPS Society is an Equal Employment Opportunity employer.

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