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Table 5 Individual free text comments from RDCRN consortium principal investigators and patient advocacy groups

From: The partnership of patient advocacy groups and clinical investigators in the rare diseases clinical research network

Patient advocacy group
 Development and growth of the Consortium-sponsored symposia has had a tremendously positive effect on the rare disease community.
 Assisting in recruitment through specific protocols and through conferences has been very rewarding. Additionally data derived on and subsequently published has made an immediate impact to the patient and medical community. This has been made possible through RDCRN. Working with other investigators and PAGs has also provided opportunities to learn from each other through professional interaction.
 A big challenge was understanding the limits of the contact registry. [The PAG is] responsible for getting >91 % of patients who are registered. Yet, we could not access those patients nor did the patients receive significant benefit
 I am very happy to be the PAG representative. It would be wonderful if someone took notes on conference calls for those who cannot make them.
 During the first couple of years the PAGs had a monthly conference call with the project manager to discuss what was going on. Would be good to reinstate these calls - maybe on a quarterly basis to keep PAGs up-to-date.
 Expanding our relationship with our PIs through Consortium activities has been one of the most fulfilling parts of our involvement in the RDCRN. The Consortium has been a wonderful platform to expand our outreach & growth to the affected community. It has been wonderful to work with investigators and PAGs from other Consortia and to learn from their experiences. We are forever grateful to the RDCRN and hope to continue this partnership for years to come.
 In the first few years of our Consortium it was an uphill rocky climb to get researchers to accept and trust our PAG input related to how best to engage and recruit participants and to enroll participating research sites. With continued communication and low turnout becoming evident the tide has now turned for the better and we are seeing our input now valued and increased patient participation.
 Our PAG became very involved with the RDCRN in about 2005, and involvement has been steady and positive since that time, but ebbs and flows with renewals and travel site scheduling. Involvement in the study design is highest and most collaborative right now in 2013 with our PAG interest in 2014 renewal. RDCRN has been very responsive to our PAG on any data questions and publishing on issues important to our community that can be revealed by the database.
 Our PAG is pleased with the positive results of our collaboration.
 Our Consortium is quite different from most others in that there is not a clinical trial associated with it (yet). The focus is on basic and translational research and the PAG-researcher relationship has been very constructive in that space.
 The [Consortium] PAGs are still mostly arm's length from operations. Leadership wants to be open but on a practical basis operates in a fairly closed circle.
 The cooperation and communication between the PAGs and the PIs has been wonderful. It has strengthened the community as we feel we are all working together for a common goal. The researchers respect and embrace the role the PAGs play to help support the Consortium and together we are moving research forward impacting the lives of patients. It has also allowed for smaller PAGs to make an impact on research by supporting programs like Travel Scholarships helping with recruitment and promoting the work of the [Consortium].
 Time is a challenge - we would like to participate more. We include Consortium activities in our newsletter.
 Wonderful collaborative relationship and feel like we have been accepted as equal partners.
Principal investigator
 Our relationship with our associated PAGs is excellent and our PAGs are an essential component to the success of our Consortium.
 Overall the Consortium has benefited significantly from the support of PAG groups affiliated with it.
 Our PAGs have been very supportive in communicating our study and research activities to the patient community, and providing in-kind support by sending out letters describing our studies or printing educational materials, etc. They have provided some trainee support in the form of travel scholarships to attend respective scientific meetings hosted by the PAG.
 Support from our PAGS has allowed us to add multiple sites to our RDCRC. Without their support we would not be able to accomplish much of what we have done over the past 4 years
 The PAG has been involved in all aspects of the Consortium activities and all interactions have been only positive
 The PAGs are an enormous asset to the Consortia and they are becoming a greater asset as time goes forward.
 The PAGs try very hard to help and they do help meaningfully in some ways. They are very busy have day jobs and little resources. Also they are lay people do not express interest in reviewing study documents.
 The regular intellectually invested and committed involvement of our PAG is the key benefit… it really helps to have constant feedback and input. It would be a bonus if they could be able to provide some funding but that is a secondary issue for us.
 The partnership of our PAGs and the Consortium has been very effective toward achieving our common mission of advancing understanding of these diseases developing more effective treatments and assuring that all patients have access to current correct information. Much more is achieved by this partnership than would be possible by either of us working alone.