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Patient Advocacy Lead, Pompe

Date: Feb 26, 2021

Location: Philadelphia, PA, US

Company: Spark Therapeutics

Join the Spark Team


We were born of innovation, springing from the curiosity, imagination and dedication of remarkable scientists and healthcare visionaries. Our shared mission is to challenge the inevitability of genetic disease by discovering, developing, and delivering treatments in ways unimaginable – until now.


We don’t follow footsteps. We create the path.







The Patient Advocacy Lead, Pompe, is responsible for supporting Spark’s interactions with specific non-profit advocacy organizations, primarily in Pompe disease and other rare disease communities under the direction of the Head of Patient Advocacy.  This role will help to build an internal culture focused on understanding and incorporating patient and family perspectives into decision making about Spark’s programs and will work to position Spark as the advocacy leader among rare disease gene therapy companies.  This is a cross functional position responsible for developing strategies and programs, streamlining communications, and building and maintaining effective relationships with patient communities, particularly Pompe disease, to increase the awareness and understanding of Spark’s leading gene therapy technology among key audiences and foster a connection to the patient experience within Spark.
Working in close partnership with other members of the patient advocacy team, this individual will actively contribute to creating the advocacy strategy and support local execution of tactics within defined communities.  They will gain a deep understanding of the advocacy landscape in Pompe disease and build collaborative and mutually beneficial relationships.  This individual must maintain a keen awareness of the dynamic nature of the drug development process and develop authentic, transparent, timely, and compliant communications that meet the needs of the business and of the patient communities.  




Develop strong collaborations with advocacy organizations focused on Pompe disease. Serve as the primary point of contact for groups and act as a Spark ambassador to external patient advocacy groups, patient advocates and other relevant stakeholders

Provide key insights gleaned from the patient communities to cross functional internal colleagues to inform the direction of the Pompe program, including input regarding unmet needs, outcome measures, and community gene therapy understanding

Develop consistent and compliant pathways to communicate with advocacy groups to keep them abreast of Spark programs and opportunities for forums to glean patient insights such as through advisory boards, focus groups and in depth interviews involvement

Create educational and awareness materials and resources to address unmet needs in patient communities Spark supports aligned with the Pompe disease program

Develop and implement short and long-term strategies to integrate and advance the various Spark resources and services offered to the rare diseases patient communities we serve

Create and execute on collaboration plans with advocacy groups based on areas of mutual interest; identify opportunities to improve outcomes, and build innovative solutions with patient organizations to improve patient care, and understanding of and access to potential gene therapies

Create educational and awareness materials and resources to address unmet needs in patient communities Spark supports aligned with the Pompe disease program

Represent the patient perspective internally and champion it in critical decision making; proactively drive ongoing internal communications about Spark’s advocacy function and foster a culture centered on our “Champion the Patient” value

Help build awareness of Spark at relevant advocacy meetings in the Pompe disease community

Acknowledge community milestones and events (i.e. awareness days) by developing and executing Spark’s efforts to amplify community messages and disease awareness, in collaboration with Spark patient advocacy and communications team members

Strategically manage and budget for sponsorships and charitable donations to defined advocacy organizations

Stay current with relevant information and resources regarding industry and health care legislation

Collaborate with Roche group Patient Partnerships colleagues regarding ex-US patient advocacy relationships, particularly in shared therapeutic areas


Education and Experience Requirements

  • 7 Years of experience in the Bio/Pharma industry; prior experience working in rare disease patient advocacy; experience in public affairs/external affairs/corporate communications or non-profit sector preferred
  • Bachelor’s Degree Required
  • Master’s degree in Genetic Counseling, Nursing, Public Health, Social Work, or other related/science degree highly desired
  • Willingness to travel 20-30% time


Key Skills Abilities and Competencies

  • Experienced biotechnology/pharmaceutical professional with relevant experience in patient advocacy
  • Prior experience in Rare Diseases
  • Keen awareness of preclinical, clinical, and regulatory processes related to drug development


Complexity and Problem Solving

With a keen sensibility regarding the drug development process, develop consistent pathways to communicate with advocacy groups to keep them abreast of Spark programs and opportunities for involvement; establish forums to glean patient insights, including advisory boards, focus groups and in-depth interviews, and engagement with patient speakers.  Utilize community insights to develop educational programming focused on gene therapy research relevant to the Pompe and broader neuromuscular and lysosomal disease/glycogen storage disease communities.  Collaborate effectively and consistently with colleagues in legal and compliance, with constant diligence regarding the development and execution of patient-facing materials and communications. 


Nearest Major Market: Philadelphia