Collaborating to Accelerate Outcome Measures for Severely Affected

DEE-P COMBINED Coalition - DEE-P Connections (a partnership of 33 patient advocacy groups - PAGs) and COMBINEDBrain (a consortium of 26 PAGs) were the founding partners of this initiative. We set out to survey the literature, assess ongoing initiatives, and develop a Blueprint for actions to accelerate progress toward validated outcome measures for severely affected DEEs.


We are building a coalition of a broad spectrum of stakeholders collaborating to pursue an integrated strategy to accelerate outcome measures capable of capturing meaningful improvements in all domains for severely affected populations. 

Limits of Current Ad Hoc Initiatives

Our landscape analysis revealed that many efforts are underway to begin to address the limitations of existing tools and measurements. Individual disease groups are exploring these issues, individual researchers are studying measures in selected disorders, and we fully understand this has been out of necessity. As promising as many of these efforts are, they are uncoordinated and often underfunded.

For significant progress to be made for all DEEs, efforts are needed to combine the dedication, engagement, and commitment of diverse developers and stakeholders.

Achieving a shared vision of the need to accelerate for Improved outcome measurements will be measurably enhanced by efforts being coordinated 

Matthew Leavens Sept 2013


Develop a collaborative effort to bring a strategic and coordinated focus to a drive to improve outcomes measures which capture improvements among the most severely affected populations. We seek collaborators across the range of stakeholders - industry, researchers, and PAGS - to  

  • Pool resources and intellect, bringing critical voices and experiences to a single table, which will optimize the rate of progress and success.
  • Maximize efficiencies - diverse stakeholders working concurrently to advance these, as well as other yet to be identified, tools eliminates the disease-by-disease, tool-by-tool process that drags out progress for the wider DEE/severely affected population
  • Avoid a fractured, piecemeal approach which would delay progress and limit continuous learning.
  • Establish a strategic collaborative approach which will be comprehensive, holistic, and robust.

Promising Projects for Collaborative Support

The landscape phase of our collaborative efforts has identified multiple projects which are ready for adaptation, extended scaling, or testing  for exploration by potential consortium partners include:

  • Adapting the Vineland - Evaluate opportunities to adapt features of the Vineland tool to the broader severe populations; PI: Dr. Anne Berg
  • Health-Related Quality of Life Scale for DEEs - extend Quality of Life Inventory of Disabilities to include refined tools that recognize diverse impacts of severe disabilities; PI: Dr. Jenny Downs in collaboration with Epilepsy Foundation Epilepsy Learning Healthcare System team
  • Goal Attainment Scaling - adaptation of existing tools already in use for clinical trials in rare epilepsies to go extend several  standard deviations further; PI: Chere Chapman, Ardea Outcomes
  • Rehabilitation Scales - adaptation of broad measures and tools developed for rehabilitation of individuals with profound trauma for broad DEE populations; PI: Dr. Natasha Ludwig, Kennedy-Krieger Institute
  • Final Disease Concept Map of select severe DEEs to provide reliable baseline of performance challenges and measurement requirements in severe DEEs; PI: Dr. Terry Jo Bichell
  • Ongoing Scouting and Documentation - ongoing collaborative effort to evaluate additional emerging opportunities on critical issues such as CVI, sleep, etc.; PI: Gabi Conecker


Get engaged - help end the floor effect now!



Industry - particularly companies with potentially game-changing disease-altering new treatments in their pipeline, stand to experience early and significant payoffs from collaborating through the new Collaborative Effort. 

  • Consult and collaborate with others confronting time-sensitive and high-stakes decisions on outcome measures  - primary, secondary and exploratory - on imminent new clinical trials; get efficiently informed about cutting edge new options for new tools with promise to better capture potentially significant improved outcomes for those most severely affected by underlying diseases
  • Play a lead role in shaping the nature and focus of the Collaborative efforts to best meet their long term and more immediate needs
  • When appropriate, work collaboratively to prioritize and accelerate implementation of the highest payoff new tools and measures under development
  • Help jump start and accelerate progress towards new validated measures for the most severely affected by combining the expertise, focus, and commitment of the broad range of stakeholders.

We propose that corporate partners, combined, provide $500,000-$750,000 in seed funding to formalize an operational collaborative organization that will direct, manage, champion, and develop full research proposals and budgets for Phase II projects. 

Contributions can be scaled to the size of applicable commercial interests and stage of development (e.g. all compounds still in clinical phase vs those with existing revenue streams).

Individual industry contributions might range from a minimum of $75,000 for small companies still in the clinical phase to $250,000 for larger corporations with a larger pipeline and an existing revenue stream.

Support/partnership with 10 companies would provide an essential industry voice as well as a substantive foundation for moving forward on all the key elements outlined above.


Participation by Researchers working on new and improved tools sharing a commitment to improving suitability for severely affected populations is central to the success of the Collaborative effort.  Consider a range of benefits in collaborating with others in your current and planned research and development efforts:

  • Collegial forum to coordinate and collaborate; develop a team-science environment of collaboration to maximize the impact of individual programs
  • Expand your impact by participating in a collaborative approach with a “rising tide” effect
  • Become eligible for assessment as a high priority project where consortium partners will collaborate to secure funding for implementation of selected projects



Patient advocate leaders will be the heart and soul of the Collaborative effort - it’s our children who all too often remain unseen, unmeasured, and off the radar in the evaluation of disease-altering treatments. Benefits from being a founding member could include:

  • Maximize efficiencies - diverse stakeholders working concurrently to advance identified and other promising new tools 
  • Pool efforts to coordinate, extend and adapt disease-specific development efforts
  • Collaborate to accelerate new measures to promote for inclusion in upcoming clinical trials

The collaborative effort will welcome engagement by all partners sharing the commitment to accelerating testing and validation of new outcome measures for those most severely affected by complex diseases. Broad advocacy organizations, patients/disability rights organizations, professional associations, key governmental organizations, and others are welcome.

We are deeply grateful to our Phase I sponsors for allowing this critical work to get started.


Supporting Donors

praxis pm

Patron/Partner Donors