SDS Alliance awarded PCORI Engagement Award funding to build capacity for patient-centered CER for Shwachman-Diamond Syndrome.

Project PACER kicked off in Cincinnati and online on June 4th, 2025, to build capacity for patient-centered CER for Shwachman-Diamond Syndrome. Focus: The Voice of the Patient.

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SDS Alliance awarded PCORI Engagement Award funding to build capacity for patient-centered CER for Shwachman-Diamond Syndrome

We are pleased to announce that the Shwachman-Diamond Syndrome Alliance (SDS Alliance) has been awarded funding through the Eugene Washington PCORI Engagement Award Program, an initiative of the Patient-Centered Outcomes Research Institute (PCORI). The funds will support building capacity for patient-centered Comparative Clinical Effectiveness Research (CER) on Shwachman-Diamond Syndrome (SDS), or Project PACER for short.

Eszter Hars, Ph.D. will lead the two-year engagement project (EASO-42419) through the SDS Alliance.

Shwachman-Diamond Syndrome (SDS) is a life-threatening, rare, genetic, bone marrow failure disorder that severely affects the quality of life and causes a high-risk of leukemia (1 in 3 patients by age 30) with catastrophic consequences. It affects about 2,000 patients in the US, many of whom are yet to be diagnosed.

Patients and caregivers feel hopeless and unheard, as there are no pathways or opportunities for them to partner in research planning and prioritization, resulting in slowed progress in research toward improved quality of life and therapies and reduced mortality. This project will focus on building capacity and opportunities for SDS patients to use their voice and effectively partner in all stages of research, in particular CER.

The project will

• Create a patient/family advisory board (FAB) comprising patients and caregivers who are knowledgeable and confident in sharing their lived experiences (the Patient Voice) to positively impact all aspects of research through training and workshops.
  

• Build a scientific/medical advisory board (SMAB) proficient in patient-centered CER and expand the research community with expertise in patient-centered CER and methods for meaningful engagement with patients through training and workshops.
  

• Create a Voice of the Patient Report and meeting to serve as a resource for patient-centered CER on SDS, and develop a framework for multi-stakeholder partnerships as well as a process/toolkit for multi-stakeholder patient-centered CER idea generation and prioritization.

  
  

The project will deliver a wide range of outputs, including multi-stakeholder meetings, a Voice of the Patient Report, surveys and evaluation tools, a stakeholder partnership and collaboration framework, training and learning resources, and toolkits, all geared toward achieving a long-lasting impact on research. In the short term, we will increase patient engagement as partners in all stages of research, enhance understanding of CER, and provide broad access to and use of the Voice of the Patient Report. In the medium and long term, we will build on these results and strive to facilitate and deliver impactful CER projects that are successfully executed, delivering benefits to patients, significantly improving quality of life and healthcare outcomes, and ultimately saving lives.

“We are incredibly excited for this work to elevate the patient voice and role in all phases of CER for SDS. We know that patients are not only essential as research participants, but that research is inevitably better and more relevant when they are involved early on and in all aspects of research planning,” shares Dr. Hars. 

This project, “Building capacity for patient-centered CER on Shwachman-Diamond Syndrome,” is part of a portfolio of projects funded by PCORI to help develop a community of patients, caregivers, clinicians, and other stakeholders who are better equipped to engage as partners in all phases of patient-centered comparative clinical effectiveness research (CER) and to disseminate results of PCORI-funded studies.

PCORI is a nonprofit organization with a mission to fund research that will provide patients, their caregivers, and clinicians with the evidence-based information that is needed to make better-informed health care decisions.

Project PACER kicked off with a meeting on June 4th, 2025 in Cincinnati, OH and online.

Watch the recording, now. To learn more and to participate in the project, visit the Project PACER page at www.SDSAlliance.org/pacer

The Externally-Led Patient Focused Drug Development Meeting (SDS PFDD) is part of Project PACER

The SDS Alliance hosted this milestone event to amplify the patient voice and communicate the unmet needs of the community, thereby accelerating therapy development and building capacity for patient-centered comparative clinical effectiveness research (CER). A comprehensive meeting report is in development and will be distributed later this fall.

The SDS PFDD Meeting was a dream come true. THANK YOU to the SDS community for showing up in all the right ways in full force. It was a very emotional event, but for a very important purpose: to communicate to the researchers and regulators what our unmet needs are, what would be a meaningful change, and that we are here to make therapies a reality. Learn all about the Externally-Led Patient Focused Drug Development Meeting on the SDS PFDD event page at www.sdsalliance.org/pfdd. The full raw recording is now posted.

Patients and caregivers! Please share your insights and feedback to be included in the report! While the offical deadline of July 4th has passed, we are still able to accept your comments. All details are available at on the SDS PFDD event page at www.sdsalliance.org/pfdd.

Watch the recording on the event page

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