Director, Patient Advocacy & Engagement
Egetis Therapeutics, Boston, MA, United States
Join us in helping patients with rare diseases. At Egetis Therapeutics we challenge the status quo for the benefit of those in need. We offer an open, positive and supportive culture where everyone can be their best. Through trust, we work seamlessly across functions. As an employee you are empowered and encouraged to have an entrepreneurial spirit, enjoying an organization with conscious and efficient decision‑making and we take pride in our collaborative working environment!
In February 2025, the European Commission approved Egetis’ Emcitate® (tiratricol) as the first and only treatment of peripheral thyrotoxicosis in patients with monocarboxylate transporter 8 (MCT8) deficiency (Allan‑Herndon‑Dudley Syndrome). In January 2026, Egetis completed the U.S. rolling NDA submission to the FDA for Emcitate® (tiratricol) for treatment of patients with MCT8 deficiency.
As a committed, courageous and collaborative organization, we are seeking to grow our team to drive success both in the pre‑launch and post launch phases of our anticipated U.S. approval.
For more information, please visit our website at www.egetis.com or follow us on LikedIn and Facebook
Our Values
Courageous - Challenge the status quo for the benefit of the patient
Committed - Operate with high ethical standards through efficient organization to deliver top quality results.
Collaborative - Through trust, work seamlessly across functions and with external partners.
Position Overview The Director, Patient Advocacy & Engagement will design and lead Egetis’ US advocacy strategy for its MCT8 deficiency program. Reporting directly to the Head of Medical Affairs, North America, this individual will partner across the US organization to build Egetis’ patient advocacy and engagement capabilities from the ground up and embed the patient voice into Medical Affairs, Clinical Development, and Commercial planning.
The ideal candidate brings substantial biopharma advocacy experience, deep expertise in rare disease, and a track record of building trusted, high integrity relationships with patients, caregivers, and advocacy organizations. They will drive initiatives to increase disease awareness, address unmet needs in the MCT8 deficiency community, and ensure that patient perspectives shape evidence generation, access strategy, and educational efforts.
This is a highly collaborative, hands‑on role in a small, nimble, and mission‑driven team that demands excellent communication, strategic thinking, and cross‑functional partnership. As a key member of the US Medical Affairs organization, the Director, Patient Advocacy & Engagement will play a pivotal role in shaping Egetis’ patient‑centric culture and contributing broadly to the success of the first US launch and future rare disease programs.
Job Responsibilities Strategic Leadership and Cross Functional Integration
Serve as the senior liaison between Egetis and MCT8deficiency‑specific and rare‑disease patient organizations, building trusted, long‑term relationships that reflect Egetis’ patient‑first commitment.
Develop and execute a strategic Patient Advocacy and Engagement plan aligned with corporate and functional objectives, delivering innovative, high‑impact value to the MCT8 deficiency community.
Champion and govern integration of patient and caregiver perspectives across US Medical Affairs and cross‑functional teams to inform priorities, strategy, and decision‑making.
Partner with Commercial and Market Access leadership to ensure patient insights inform launch planning, value narratives, services, evidence generation, and access strategies.
Serve as the internal subject‑matter expert on patient advocacy and engagement, advising leaders on best practices, regulations, and ethical, compliant partnership models.
Build scalable patient‑engagement frameworks to support future pipeline programs and assume responsibility for Patient Advocacy and Engagement as the pipeline expands.
Patient & Caregiver Engagement Execution
Lead patient and caregiver engagement initiatives (e.g., caregiver panels, advisory meetings, educational events) to integrate diagnostic and disease‑journey insights into organizational understanding of unmet needs.
Oversee development and delivery of clear, balanced, and compliant patient‑facing materials addressing disease education, clinical trial participation, approval/launch, and access to treatment.
Represent Egetis externally at key patient advocacy, rare‑disease, and professional forums to elevate the patient voice and strengthen the ecosystem.
Governance, Compliance & Ecosystem Stewardship
Provide strategic oversight of patient‑organization funding and collaborations; establish compliant policies, processes, and tracking systems for funding and sponsorship requests and outcomes.
Monitor relevant policy, access, and standards‑of‑care developments in rare disease and guide appropriate, compliant engagement to support evolution of the ecosystem.
Measurement, Budget & Operational Management
Define success metrics and KPIs for advocacy and engagement, track impact, and communicate insights and recommendations to senior leadership.
Own and manage the patient advocacy budget and external partners (e.g., consultants, agencies) to ensure efficient resource use and high‑value execution.
Qualifications & Experience
8+ years of experience in patient advocacy, patient engagement, Medical Affairs, public affairs, market access, or related functions within biopharma, biotech including rare disease‑focused organizations.
Proven leadership in developing and executing patient advocacy and engagement strategies aligned with medical, clinical, commercial, and corporate objectives.
Demonstrated ability to build trusted partnerships with patient and caregiver organizations, particularly in rare or specialty disease areas.
Experience translating patient and caregiver insights into development, launch, access, and lifecycle strategies.
Cross‑functional leadership and influence, including collaboration with Medical Affairs, Clinical Development, Commercial, and Market Access teams.
Experience serving as a subject‑matter expert, advising senior leaders on best practices, regulations, and compliant engagement models.
Strategic planning and measurement experience, including defining KPIs, assessing impact, and communicating insights to leadership.
Budget ownership and vendor management experience, including oversight of agencies and consultants.
Experience representing organizations externally at patient advocacy or rare disease forums.
Ability to travel up to 30%.
Willingness and ability to work in a hybrid model, with required on‑site presence as defined by business needs.
Strong understanding of the patient and caregiver journey, with experience translating insights into development, launch, access, and lifecycle strategies.
Deep knowledge of regulatory, compliance, and ethical standards governing interactions with patient organizations, funding, and communications.
Executive‑level communication skills (written and verbal), including the ability to influence senior stakeholders.
Cross‑functional influence and stakeholder management capabilities.
Strategic thinking with the ability to connect patient engagement initiatives to enterprise objectives.
Analytical mindset with ability to define metrics, evaluate impact, and derive actionable insights.
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In February 2025, the European Commission approved Egetis’ Emcitate® (tiratricol) as the first and only treatment of peripheral thyrotoxicosis in patients with monocarboxylate transporter 8 (MCT8) deficiency (Allan‑Herndon‑Dudley Syndrome). In January 2026, Egetis completed the U.S. rolling NDA submission to the FDA for Emcitate® (tiratricol) for treatment of patients with MCT8 deficiency.
As a committed, courageous and collaborative organization, we are seeking to grow our team to drive success both in the pre‑launch and post launch phases of our anticipated U.S. approval.
For more information, please visit our website at www.egetis.com or follow us on LikedIn and Facebook
Our Values
Courageous - Challenge the status quo for the benefit of the patient
Committed - Operate with high ethical standards through efficient organization to deliver top quality results.
Collaborative - Through trust, work seamlessly across functions and with external partners.
Position Overview The Director, Patient Advocacy & Engagement will design and lead Egetis’ US advocacy strategy for its MCT8 deficiency program. Reporting directly to the Head of Medical Affairs, North America, this individual will partner across the US organization to build Egetis’ patient advocacy and engagement capabilities from the ground up and embed the patient voice into Medical Affairs, Clinical Development, and Commercial planning.
The ideal candidate brings substantial biopharma advocacy experience, deep expertise in rare disease, and a track record of building trusted, high integrity relationships with patients, caregivers, and advocacy organizations. They will drive initiatives to increase disease awareness, address unmet needs in the MCT8 deficiency community, and ensure that patient perspectives shape evidence generation, access strategy, and educational efforts.
This is a highly collaborative, hands‑on role in a small, nimble, and mission‑driven team that demands excellent communication, strategic thinking, and cross‑functional partnership. As a key member of the US Medical Affairs organization, the Director, Patient Advocacy & Engagement will play a pivotal role in shaping Egetis’ patient‑centric culture and contributing broadly to the success of the first US launch and future rare disease programs.
Job Responsibilities Strategic Leadership and Cross Functional Integration
Serve as the senior liaison between Egetis and MCT8deficiency‑specific and rare‑disease patient organizations, building trusted, long‑term relationships that reflect Egetis’ patient‑first commitment.
Develop and execute a strategic Patient Advocacy and Engagement plan aligned with corporate and functional objectives, delivering innovative, high‑impact value to the MCT8 deficiency community.
Champion and govern integration of patient and caregiver perspectives across US Medical Affairs and cross‑functional teams to inform priorities, strategy, and decision‑making.
Partner with Commercial and Market Access leadership to ensure patient insights inform launch planning, value narratives, services, evidence generation, and access strategies.
Serve as the internal subject‑matter expert on patient advocacy and engagement, advising leaders on best practices, regulations, and ethical, compliant partnership models.
Build scalable patient‑engagement frameworks to support future pipeline programs and assume responsibility for Patient Advocacy and Engagement as the pipeline expands.
Patient & Caregiver Engagement Execution
Lead patient and caregiver engagement initiatives (e.g., caregiver panels, advisory meetings, educational events) to integrate diagnostic and disease‑journey insights into organizational understanding of unmet needs.
Oversee development and delivery of clear, balanced, and compliant patient‑facing materials addressing disease education, clinical trial participation, approval/launch, and access to treatment.
Represent Egetis externally at key patient advocacy, rare‑disease, and professional forums to elevate the patient voice and strengthen the ecosystem.
Governance, Compliance & Ecosystem Stewardship
Provide strategic oversight of patient‑organization funding and collaborations; establish compliant policies, processes, and tracking systems for funding and sponsorship requests and outcomes.
Monitor relevant policy, access, and standards‑of‑care developments in rare disease and guide appropriate, compliant engagement to support evolution of the ecosystem.
Measurement, Budget & Operational Management
Define success metrics and KPIs for advocacy and engagement, track impact, and communicate insights and recommendations to senior leadership.
Own and manage the patient advocacy budget and external partners (e.g., consultants, agencies) to ensure efficient resource use and high‑value execution.
Qualifications & Experience
8+ years of experience in patient advocacy, patient engagement, Medical Affairs, public affairs, market access, or related functions within biopharma, biotech including rare disease‑focused organizations.
Proven leadership in developing and executing patient advocacy and engagement strategies aligned with medical, clinical, commercial, and corporate objectives.
Demonstrated ability to build trusted partnerships with patient and caregiver organizations, particularly in rare or specialty disease areas.
Experience translating patient and caregiver insights into development, launch, access, and lifecycle strategies.
Cross‑functional leadership and influence, including collaboration with Medical Affairs, Clinical Development, Commercial, and Market Access teams.
Experience serving as a subject‑matter expert, advising senior leaders on best practices, regulations, and compliant engagement models.
Strategic planning and measurement experience, including defining KPIs, assessing impact, and communicating insights to leadership.
Budget ownership and vendor management experience, including oversight of agencies and consultants.
Experience representing organizations externally at patient advocacy or rare disease forums.
Ability to travel up to 30%.
Willingness and ability to work in a hybrid model, with required on‑site presence as defined by business needs.
Strong understanding of the patient and caregiver journey, with experience translating insights into development, launch, access, and lifecycle strategies.
Deep knowledge of regulatory, compliance, and ethical standards governing interactions with patient organizations, funding, and communications.
Executive‑level communication skills (written and verbal), including the ability to influence senior stakeholders.
Cross‑functional influence and stakeholder management capabilities.
Strategic thinking with the ability to connect patient engagement initiatives to enterprise objectives.
Analytical mindset with ability to define metrics, evaluate impact, and derive actionable insights.
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