The NOMINATE project: Novel Outcome Measures IN AuToimmune DiseasE
In some rare cases, a pregnant woman’s immune system can produce antibodies that attack the red blood cells of their own child (through a process called maternal alloimmunization). This can result in severe diseases like Hemolytic Disease of the Fetus and Newborn (HDFN). HDFN occurs when the fetal red blood cells are not compatible with the mother`s. In these cases, the mother’s immune system sees the baby’s red blood cells as foreign and produces antibodies against them. When these antibodies bind to the baby’s red blood cells, the cells break down leading to the potentially life-threatening condition of fetal anemia.
Project overview and objectives
The NOMINATE project (“Novel Outcome Measures IN AuToimmune DiseasE”) seeks to identify new or existing research outcomes in the management of the alloimmune condition of HDFN.
The NOMINATE project is being led and funded by Janssen (the Pharmaceutical Companies of Johnson&Johnson).
The study approach consists of a literature review to identify previously reported outcomes that are considered of most relevance by healthcare professionals (HCPs), patients (or parents) and regulators and health technology assessment agencies.
Advisory boards involving both healthcare professionals (HCPs) and patients (and/or parents) will be executed within this project to discuss the outcomes identified in the literature review and to produce a ranking in terms of clinical relevance / credibility and applicability.
Following this, a Delphi exercise will seek to establish a consensus between HCPs and patients on the most relevant outcomes that may optimally reflect the impact of the disease from both patient’s and the HCP’s perspectives.
The aim is that outcomes identified within this project will be integrated into evidence-based approaches for future studies in the field.
The main objectives of involving the patient voice now and in future approaches are:
- to understand what it is like to live with such conditions,
- how it impacts life (pregnancy) and family planning (patient journey and quality of life),
- learning about their thoughts on treatments and procedures,
- to understand what is most important to achieve with treatment and procedures,
- how operations and communications could be optimised.
The role of EFCNI
- ensuring patient involvement at all stages of the research project
- set-up of a standing advisory board consisting of patients or family representatives affected by HDFN
- to advise on research protocol, design and how to best communicate with patients and/or family representatives
- input on ethical review and parent questionnaires
- dissemination and communication
- support in building a network and raising awareness for rare diseases like HDFN
Transparency: The NOMINATE project is being led and funded by Janssen (the Pharmaceutical Companies of Johnson&Johnson). EFCNI receives funding in relation to the above-mentioned services provided in support of that project.