PKuReD

Prepared 10/01/2008By Sean Flaherty, Mark Stone & Ken KahtavaFor the Polycystic Kidney Disease Foundation

A Simple Set of Concepts• Concept A: There is enough information available in the

world to solve any problem.

• Concept B: Breakthroughs in science and solutions are a series of serendipitous discoveries made by very smart people looking for answers.

• Concept C: Very large amounts of data, organized effectively - will point to patterns and relationships between genetic, medical and environmental factors that might otherwise go un-noticed. They can point researchers in directions that they would not have other considered.

The Goal

• Speed the process of serendipitous discovery while providing those suffering from the disease tools that will enable them to participate in the finding of a cure.

The Idea

If we could harness the breadth and passion of the PKD community, we could assemble an unprecedented database that would add immeasurable value to the research being conducted in the Polycystic Kidney Disease space.

• The concept: Build a community of constituents to inspire hope and allow them to interact with each other in their own safe environment.

• In just over 4 weeks, it already has 300+ members with 45 Blogs and 14 self-identified groups.

• Groups are already self-organizing in very interesting ways. Regionally, internationally, by age, etc.

• Consider how useful it will be to provide access to this community to both the pharmaceutical industry and the research community.

• Couple that with other data that we are uniquely positioned to gather and provide.

• Access for both clinical trials and research questionaires.

• We have access to multiple databases of genetic markers that are currently in existence.

• We could provide incentives for HopeSquare members to contribute their DNA in order to add their unique profiles to the database for research purposes.

• This alone would make this an incredible asset for the research community.

• And – it will give our constituents a very unique way to contribute to future generations and the cure for the disease.

Roadblock:• People are generally fearful of providing their

genetic information for various reasons.– GINA (Genetic Information Non-Discrimination

Act) should help us to get over that barrier.

• It will be an expensive proposition to get profiles completed.

• As the foundation builds out a network of clinics, (similar in concept to the Cystic Fibrosis Foundations efforts), we will have access to clinical information that has not been available in a single place.

• Tied to the genetic data and the community that we are building to add more power to the available dataset for research.

Roadblock:• We do not currently have infrastructure to

support data gathering at clinics. • It will be an expensive proposition to establish

these facilities and to incent the clinics to provide good data (This is an issue that the CF Foundation had to overcome.)

• At a minimum, for all of the clinical trials that the foundation funds, we should establish a repository for the data that is collected for future studies and longitudinal studies. (There is an effort underway currently with Lorrie’s team to accomplish this.)

• If we were able to pull this data into our repository and couple it to the clinic data, the genetic data and the community this would provide an unprecedented dataset for researchers to leverage.

• There is an immense amount of information locked in those clinical trials that is ripe for further analysis.

Roadblock: • Clinical researchers and especially the

pharmaceutical industry is particularly protective of their data.

• We need to institute contractual policy to ensure that the data becomes available once research is published or clinical trials are complete if we are involved in them.

• Leveraging the power of social networking in the “Patients-Like-Me” model where we allow patients to self-report on their clinical indications and treatments.

• Allow members of HopeSquare to report on their experiences and discuss treatments, trials and their indications.

Roadblock: • The scientific community is highly, highly skeptical of

PRO data for various reasons. • Our belief is that while PRO data may not be

definitive or perfectly scientific, it will have statistical value across broad populations with many data points. It will not provide the answers, but it may point science in a direction that may not have come about otherwise.

• Indications and treatments will need to be “vetted” by PKD Clinical staff to ensure consistency and usefulness.

The End Result• The goal of this

initiative would be to ultimately make all of this information available through a vetted process for:– The research

community (for free)

– The Pharmaceutical industry (Through a hefty subscription fee model.)

Note: The information provided would be either De-Identified or – with EXPLICIT permission from the patients

Next Steps

• Obviously a LOT of work needs to go into this initiative to make it work.– Assembling and architecting data structures– Gathering and transforming data– Building applications

• Funding Needs to be acquired to get started– Kauffman Foundation– The Transmed Partnership– Government Lobbying