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Patient Advocacy Lead, Early Development

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.




An understanding of the patient experience, and decision making based on that understanding, has become increasingly important in the drug development process.  The patient community demands and deserves to be engaged early and often.  The Patient Advocacy Lead, Early Development, will be responsible for delivering on that engagement and ensuring consideration and integration of the collective and unique experiences of the patient communities we serve into our early development programs, from discovery through the candidate optimization/IND-enabling phase.   


This role is responsible for supporting Spark’s interactions with specific non-profit advocacy organizations aligned with Spark’s therapeutic areas in preclinical and early clinical development, under the direction of the Head, Patient Advocacy.  This role will help to build an internal culture focused on understanding and incorporating patient and family perspectives into early decision making about Spark’s programs and will work with colleagues 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 to increase the awareness and understanding of Spark’s leading gene therapy technology among key audiences and foster an 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 defined communities aligned with Spark’s early development programs 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.  




Conduct comprehensive landscape assessments for defined patient advocacy communities, and action upon areas of opportunity identified by the landscape assessments, commensurate with program lifecycle stage

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

Serve as point of contact for groups and act as a Spark ambassador to external patient advocacy groups, patient advocates and other relevant stakeholders

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

As indicated, create educational and awareness materials and resources to address unmet needs in patient communities Spark supports aligned with the early development pipeline

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 communities aligned with Spark early development programs

Support Research and Development, Medical Affairs, Candidate Development Teams, and Asset Teams in partnership with the Patient Advocacy team members and consistent with Corporate Communications efforts

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

Provide regular updates to advocacy organizations aligned with Spark’s early development pipeline and find opportunities to engage

Effectively navigate transition of patient advocacy group relationships to members of the patient advocacy department as a program progresses from candidate optimization to clinical development

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


Education and Experience Requirements



  • 5+ years of experience in the Bio/Pharma industry; prior experience working in rare disease patient advocacy; experience in research and development, new product planning, program management, or clinical development/operations preferred
  • Bachelor’s degree required
  • Master’s degree in Genetic Counseling, Nursing, Public Health, Social Work, or other related/science degree highly desired


Key Skills Abilities and Competencies



  • Experienced biotechnology/pharmaceutical professional with relevant experience in patient advocacy at a national level
  • Prior experience in Rare Diseases
  • Keen awareness of preclinical, clinical, and regulatory processes related to drug development
  • Demonstrated examples of effective patient engagement and stakeholder management
  • Strong communication and interpersonal skills with an ability to build genuine relationships quickly with diverse populations
  • Exceedingly strong scientific acumen with ability to understand and engage in sophisticated conversations in cross functional settings for a multitude of programs simultaneously
  • Honed ability to translate complex scientific information to a broad audience, and to develop and deliver scientific educational content appropriately to patient audience
  • Comfort with the dynamic nature of drug development with a keen ability to assess the impact of and tailor communications related to program developments in an appropriate fashion
  • Strong business acumen with an ability to understand key clinical, scientific, and commercial information and relay it to stakeholders in an authentic manner
  • Collaborative work style with an equally strong ability to work independently and with a sense of urgency
  • Ability to influence in a proactive, objective, and diplomatic manner within all levels of the company and outside community leaders
  • Proficiency in navigating a high energy, matrixed, and dynamic work environment alongside dedicated colleagues at all management levels with ease and comfort


  Complexity and Problem Solving      


With a keen sensibility regarding the sensitivity of early development program information, 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.

Drug development is rarely a linear process.  This role must effectively manage expectations of both internal and external stakeholders to ensure productive navigation of potentially difficult situations or unwelcome news (i.e. timeline delays, program discontinuation).  Success in this role will be dependent upon a candidate’s ability to determine the breadth and depth of engagement that is appropriate for the community and commensurate with the program stage. 


Nearest Major Market: Philadelphia