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Collaborating to Accelerate Outcome Measures for those who are Severely Affected

Our Goal

Within five years, we aim to
have FDA approved measures
that have been adapted to work for those
more severely impacted DEEs.

<-- Watch our latest Community Update on the project and learn how you can help make sure those most severely impacted can be included in clinical trials and their progress measured! 

Our Call to Action

Our Work

The Inchstone Project is a multidisciplinary group of consumers, stakeholders, researchers and clinicians collaborating to accelerate outcome measures development. It grew out of a widespread consensus among leaders of patient-advocacy groups representing diverse developmental and epileptic encephalopathies (DEEs) that the inability of existing tools to capture small improvements in severely affected children could jeopardize trials of new disease-altering treatments. The focus of this project is on critical outcome domains for individuals with DEEs who have severe functional and cognitive impairments and whose progress cannot be meaningfully captured with standard clinical outcome assessments.

We came together to address this unmet need and to identify and develop measures that are sensitive to the small but important changes (inchstones) that many at the severely affected end of the DEE spectrum may have in response to new therapies. Working together, we have developed a blueprint for accelerating progress toward validated measures suitable for those severely affected by DEEs as well as other severe populations.

Countless new targeted or gene-based therapies are nearing trials which could be jeopardized by the absence of tools to capture small but significant improvements in populations who routinely fall 3-5 standard deviations below the mean and are not currently measured.

The success of emerging precision medicines may well depend on new or adapted precision measurement tools with the ability to capture improvements within the most severely affected subpopulations. The Inchstone Project presents an efficient and focused collaborative effort to advance the “precision measurement” that is essential to the success of “precision medicine.”

In the first year of the Inchstone Project we have made substantial progress:

  1. Recruited a diverse group of high-caliber subject matter experts to help guide our efforts via our Steering Committee
  2. Gather our Industry Advisory Panel - we currently have six industry advisory groups who are involved.
  3. Provided feedback to the FDA on their drafts guidance "Patient-Focused Drug Development: Selecting, Developing, or Modifying Fit-for-Purpose Clinical Outcome Assessments". You can read our detailed feedback HERE.
  4. Conducted a pilot project to test adaptations to existing instruments for more severely impacted populations with our partners at FamilieSCN2A. As a result of this work, we had 4 abstracts accepted as posters at AES 2022 and two of our team helped plan and presented at an Investiagtor's Workshop on Non-Seizure Outcomes Measures for DEEs.
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DEE-P Connections—a collaborative project founded by the International SCN8A Alliance—was born out of the frustration that there was no space for families who had children with a range of severe epilepsies to come together to learn, ask questions and advance the understanding of rare epilepsies. 

With the nearing of clinical trials for many rare epilepsy communities, we began to feel the urgency for outcome measures that would actually measure the smaller improvements our children make over time—the inchstones, not milestones. Thus, DEE-P Connections launched a plan to work collaboratively with key stakeholders to develop consensus on how to tackle this gap.

In the foundational year of this project (throughout 2021) we comprehensively surveyed the literature on outcome measures and assessed ongoing initiatives working to develop outcome measures that captured more minute progress. We partnered with five researchers to develop a Blueprint to accelerate progress toward validated outcome measures for severely affected DEEs and other disorders.

We culminated that year of work with two open workshops to share what we had learned with the community and host an open discussion about how to advance this critical work. 

This phase of work was a joint effort of DEE-P Connections and COMBINEDBrain.

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