2.1 Creation of an index
In 2019, discussions started regarding how to monitor the implementation of the PID Principles of Care IPOPI had developed in the different countries, and to track how countries performed in terms of PID care to their patients. Those discussions resulted in the inception of the PID Life Index.
The PID Life Index was developed in several stages, the first step being to assess the described principles in the 2014 article [3], consider necessary updates and how these could be measured through quantitative criteria. For this assessment, IPOPI engaged the expertise of 3 dedicated experts in the field of PIDs. Discussions led to the agreement on the following topics for the principles: diagnosis, treatment, universal health coverage, specialised centres, national patient organisations and registries:
- Diagnosis measures the availability of tests for the diagnosis of PID patients within a given country, as well as the diagnosis rate expressed as a percentage, built based on the theoretical number of PID patients (1/2,000) and the known number of patients in that country.
- Treatment considers the availability of treatments for patients with PIDs in a given country and the availability of reliable plasma and blood collection infrastructure.
- Universal health coverage shows the level of reimbursement or coverage by the national/regional health or social system of a country on diagnostics or treatments for patients with PIDs. This data is provided as a percentage, estimating the part of the diagnostic or treatment that is provided free of charge or reimbursed to patients with PIDs compared to patients’ out of pocket expenses.
- Specialised centres for children and adults working in the frame of a national network are considered the best model to address complex rare conditions such as PIDs in their different dimensions (diagnosis, treatment, care provision, transitioning and ageing care). Information is given on the availability of these networks/centres and their specificities for each participating country.
- National organisations of patients with PIDs have a significant role to play in healthcare systems. It is well recognised that patient representatives have become experts on their conditions and relevant treatments and bring unique and personal perspectives on the impact of diagnosis and treatment to their communities [3]. Additionally, they work to improve patients and healthcare professional’s awareness and education on these conditions and advocate so the health authorities adapt the legal framework to these rare conditions.
- Patient registries constitute important instruments to serve research in the field of rare diseases and to improve patient care and healthcare planning. They help pool data in order to achieve a sufficient sample size for epidemiological and/or clinical research [4], assess treatments and facilitate/encourage clinical trials.
From the 2014 landmark publication, the only differing principle is universal health coverage, which relates to “managing PID diagnosis and care in all countries” and links the Index with the WHO initiative on Universal Health Coverage [5].
Each of the principles were described by a series of measurable criteria (Table 1). Each principle has been given the same weight, as they have been considered to all be equally important. Specific weights were attributed to the criteria depending on their relevance and importance to quantify the principle. This allows for observing progress in the implementation, with the final objective of seeing countries scoring steadily higher in every principle over time, indicating that the PID environment in the country is improving. The Index allows for updates and future calibration in line with the global evolution of the PID environment.
Table 1: Principles and criteria used to build the PID Life Index
Principles
|
Criteria
|
PID diagnosis
|
- Diagnosis rate
- Biological diagnosis availability
- Genetic diagnosis availability
- Prenatal diagnosis availability
- Newborn screening for SCID
|
Treatments
|
- Anti-infectious availability
- Immunoglobulin availability
- Vaccine availability
- Curative treatments
- Biological and targeted therapies
- Plasma collection
|
Universal Health Coverage
|
- Diagnosis reimbursement
- Anti-infectious reimbursement
- Immunoglobulin reimbursement
- Vaccine reimbursement
- Curative treatments reimbursement
- Biological and targeted therapies reimbursement
|
Specialised centres
|
- National PID specialised centre/ network
- Adult PID services
- Transition care
|
National patient organisations
|
- Established national group in the country
- Professional paid staff
- Main working areas
|
Registries
|
- National PID registry
- Bone marrow donor registry
|
2.2 Creation of a PID Life Index database
Once the principles and criteria were defined, they were translated into a questionnaire used to gather data from the different countries. This questionnaire was tested in a pilot phase in a reduced number of countries from different regions in the world to improve and validate it. Once the questionnaire was validated, the data collection process was enlarged to include all IPOPI national patient organisations and those countries in which IPOPI has specialised medical relationships. The information received was provided by IPOPI’s counterparts to the best of their knowledge. This questionnaire will also be used for a yearly revision of the data, however, data are open for revision at any time.
2.3 Display of the data
The database with the collected information was uploaded to an online platform that displays information on the PID care globally through different settings; a web-based visualisation system and a world map presenting the global index, the different principles as well as the criteria, and a country. Each country shows a percentage score in an interactive circle. The maximum score is 100% for each principle. The PID Life Index supports web-based resource mapping of aggregated (global, by continent) and individual data, including for example showing relations between countries within the same region for each principle.
Additional features of the tool include: 1) a list of specialised centres for PIDs for children and adults and their location within each country, 2) a list of immunoglobulin replacement therapies registered per country, 3) a country page that summarises the data that are available in the database and allows for the provision of further comments detailing the specificities of the country if need be, 4) a glossary to help centralise the information on the terms used in a simple manner for the reader.
The PID Life Index is freely accessible on IPOPI website or through this link [6] on a computer, tablet or mobile phone.