Prioritization of Therapy Uncertainties in Dystrophic Epidermolysis Bullosa: where Should Research Direct To? An Example of Priority Setting Partnership in Very Rare Disorders

Davila-Seijo et al. (2013) full text summary PDF

For which topic were research priorities identified?

dystrophic epidermolysis bullosa

In which location was the research priority setting conducted?

Europe - Spain

Why was it conducted at all?

Dystrophic Epidermolysis Bullosa (DEB) is a rare genodermatosis (7 cases per million) that causes blisters and erosions with minor trauma in skin and mucosa, and other systemic complications. A recently updated systematic review showed that the research evidence about DEB therapies is poor. As new trials in DEB are difficult and expensive, it is important to prioritizise research that patients and clinicians consider more relevant.

What was the objective?

to describe and prioritize the most important uncertainties about Dystrophic Epidermolysis Bullosa treatment shared by patients, carers and health care professionals in order to promote research in those areas

What was the outcome?

a ranking list of 10 research questions

How long did the research prioritization take?

November 2011 - October 2012

Which methods were used to identify research priorities?

JLA method

How were the priorities for research identified exactly?

Step 1: setting up PSP: stakeholder mapping. Step 2: survey: collecting treatment uncertainties, participants were asked: What question(s) about DEB treatment would you like to be answered by research?, Step 3: data processing: collation of treatment uncertainties, removing any uncertainties not related with therapy, comments or personal queries, refining uncertainties into PICO format, combining similar questions, removing duplicates, additional uncertainties from literature added, resulting in final list of 77 uncertainties. Step 4: interim ranking: survey asking participants to select a maximum of five main uncertainties. Step 5: final prioritization: workshop: workshop using the nominal group technique, small group discussions, each small group independently selected their top ten uncertainties, then the three lists were joined into an overall ranking, top ten list established by consensus

Which stakeholders took part?

Patients, carers, health care professionals. Survey: 62 participants: 23 patients, 18 carers and 21 healthcare professionals. Interim ranking: 46 participants. Workshop: 3 patients, 2 carers, 6 HCPs and a representative of DEBRA Spain, and 3 facilitators.

How were stakeholders recruited?

Potential partner organizations and individuals were contacted using the Steering Group members' networks and through the DEBRA Spain´s register of affiliates. To identify experts on the disease information from DEBRA, DEBRA associates, physicians working in centers with expertise in DEB care reference, were contacted and all authors of papers included in Medline, with an address in Spain, and reporting a minimum of three patients were contacted. Also, all experts were asked to identify other experts and justify their selection. To identify patients and carers all members of DEBRA Spain with DEB (as many of them were children, their parents were contacted) were contacted.

Were stakeholders actively involved or did they just participate?

Stakeholders not only participated but were also actively involved in the research prioritization process: They were part of a steering group. The steering group consisted of 8 people with experience in DEB including patients/carers, 1 representative from the Dystrophic Epidermolysis Bullosa Research Association Spain (DEBRA Spain), healthcare professionals (dermatologists and nurses), and researchers/epidemiologists. The members participated in the workshop.