Benchmarking access to care in specific disease areas using the

PACT 5-As tool: the case of b-thalassaemia in Greece

BEST PRACTICES SESSION

Kyriakos Souliotis, PhD

Associate Professor of Health Policy, University of Peloponnese, Greece
Associate Director, LSE Enterprise – Health Technology Assessment

Christina Golna, LLB,LLM,MSc

Chief Development Officer, Innowth

Kyriakos Souliotis
 Survey rationale and applicability
Mapping β-thalassaemia unmet needs through a structured framework that can assist policy change

Understand current standard of care in β-thalassaemia and investigate and report

Goal on any access challenges patients face to produce a composite, measurable access
indicator that can assist decision making to improve health outcomes

The 5As conceptual framework to measure and report on access to health care
Tool
services, as developed in collaboration with PACT
This tool is a composite framework to measure access to health care services across
5 access areas. We customized the tool (questions and close-ended responses) to
Customization
the challenges specific to this condition to better reflect the reality transfused β-
thalassaemia patients have to face when accessing care
Survey results bring forward in a measurable manner the challenges patients face
when accessing care, all primarily related to the absence of a strong and
Outcomes
interconnected primary health care network and the geographical spread of
services, coupled with financial challenges, exacerbated by the economic crisis
This tool can be used to map and measure access within specific therapeutic areas
Applicability across a number of health systems and countries, allowing for comparisons of the
contribution of structural elements on outcomes

Kyriakos Souliotis
 Survey identity
Mapping β-thalassaemia unmet needs through a structured framework that can assist policy change

Identity Descriptive, non-experimental, with cross-sectional design

Timeline November 2018 – January 2019

Transfused β – thalassaemia patients, who were also members of the Panhellenic

Association of Patients with Thalassaemia and the Hellenic Association of Thalassaemia
Inclusion criteria
(n=116). Both associations are based in Athens, Greece but their members may reside
across the country, therefore the sample can be characterized as nationwide

Exclusion criteria Not currently undergoing transfusion, aged less than 18 years

Purposive sampling was used to select study participants, i.e. a non-probability

sampling method, in which participants are selected with a purpose (a criterion of
Sampling
selection) in mind, which relied on researcher’s personal judgement when sampling the
population (Lepkowski J, 1991)

Participation invitation, informed consent, information on the study and a link to the
Materials
questionnaire was sent by each Association to their members.

Kyriakos Souliotis
 Why Thalassaemia?
Thalassaemia is a hereditary, blood related genetic disorder, which involves the absence of or errors in genes
responsible for production of heamoglobin

B – Thlassaemia is a blood
disorder that reduces
haemoglobin production

People with β -
thalassaemia lack
red blood cells
and are at an
increased risk of
developing
abnormal blood
clots

NIH, US National Library of Medicine, https://ghr.nlm.nih.gov/condition/beta-thalassemia

Kyriakos Souliotis
 Thalassaemia treatment approach
Early treatment of thalassaemia is effective in improving patient quality of life (WHO 2018)

14 μήνες Personalized
Inhibitors of the TGF-β
family of growth factors approach

Systematic
transfusions to
manage anaemia

Allogeneic hematopoietic stem

Bone marrow cell transplantation
transplantation
Iron chelation therapy
to manage iron
overload in the body
Splenectormy

Kyriakos Souliotis
 Thalassaemia: highly prevalent in Southern Europe

It was named after the

Mediterranean Sea
(“Thalassa”) and the word Its distribution follows the
blood (“αίμα”, haema) typical malaria distribution
from ancient times to this
day

Thalassaemia heterozygosity
confers a degree of immunity
against malaria =>
particularly high prevalence
in areas where malaria has
Thalassaemia is highly been endemic or widespread
prevalent in countries
around the Mediterranean
basin

Kyriakos Souliotis
 Particularly in Greece
In Greece, prevalence is estimated at 8%, though in some areas it ranges between 15 and 20 %

>25 mutations have been

recorded involving the β-
globin gene

Globally, >350 mutations

have been recorded (wide
diversity, small number of
specific mutations in
individual populations)

Triantafyllou et al, 2014, Voskaridou et al, 2012, Kalleas et al, 2012, De Sanctis et al, 2018 Kyriakos Souliotis
with a high cost and a high “complacency” as to policy aspirations
In Greece, cost of blood is high and literature on unmet thalassaemia patient needs very limited

High cost of blood

“Compromised” (?)
pressure on health care Is there no
Blood shortages or
system to ask for more unmet need?

Availability of chelators

Kanavos P et al, 2006, Fragoulakis V et al, 2014, Marantidou O et al, 2007, Hauk L, 2018 Kyriakos Souliotis
 Survey tool
The survey customized and used the PACT 5As framework for measuring access to health care
services

5As

Accessibility

Adequacy

Affordability

Appropriateness

Availability

Kyriakos Souliotis
 Survey tool
The survey customized and used the PACT 5As framework for measuring access to health care
services

5As

The ability of use health services without facing undue constraints

Accessibility
(related to e.g. distance, working hours, transportation, discrimination)
Adequacy

Affordability

Appropriateness

Availability

Kyriakos Souliotis
 Survey tool
The survey customized and used the PACT 5As framework for measuring access to health care
services

5As

Accessibility

Provision of health services that are satisfactory in quantity and quality

Adequacy
to meet actual health need

Affordability

Appropriateness

Availability

Kyriakos Souliotis
 Survey tool
The survey customized and used the PACT 5As framework for measuring access to health care
services

5As

Accessibility

Adequacy

Ability to access and receive health services at a cost that is not too high
Affordability
for the patient and his/her family to bear

Appropriateness

Availability

Kyriakos Souliotis
 Survey tool
The survey customized and used the PACT 5As framework for measuring access to health care
services

5As

Accessibility

Adequacy

Affordability

Suitability of health services provided for the particular health needs at

Appropriateness
the particular occasion/situation
Availability

Kyriakos Souliotis
 Survey tool
The survey customized and used the PACT 5As framework for measuring access to health care
services

5As

Accessibility

Availability as a concept
Adequacy encompasses some of the
elements of the previous 4 As
and may be used as a cross
Affordability validation of responses in
previous sections of the
questionnaire
Appropriateness

Fact or possibility of health services being offered in such a manner so

Availability that they can be bought or received

Kyriakos Souliotis
 Measurement areas
We measured transfusion burden as the sum of the burden 6 types of health care services place on
patient and family:

Transfusion General health

(the process) services
(outpatient)* 5 point Likert Scale

General health Medications

services (prescription)* 0 – Not at all
(inpatient)*

4 – Very
Medications
Lab tests *
(dispensing)*

* All in relation to a transfusion Kyriakos Souliotis

 Accessibility
Transfusion unit Outpatient g.h. services Inpatient g.h. services

Prescription Dispensing Lab tests

Not accessible at all Somewhat accessible, but only after I Occasionally accessible, depending on
face a lot of barriers when I visit
Mostly accessible Very accessible

Kyriakos Souliotis
 Adequacy
Transfusion unit Outpatient g.h. services Inpatient g.h. services

Prescription Dispensing Lab tests

Not adequate at all Somewhat adequate, but I feel I do not get Occasionally adequate, depending on when
the optimum care or enough attention I visit (choose to go when staff I am friendy
Mostly adequate with)
Very adequate

Kyriakos Souliotis
 Affordability
Transfusion unit
Μονάδα Μετάγγισης Outpatient g.h. services Inpatient g.h. services

2%6%
14%

47%

31%

Prescription Dispensing Lab tests

Not affordable at all
Most affordable
To be able to afford them, we have to We can afford them, but I feel my care is
regularly give up other critical expenses placing a substantial/disproportionate
burden on me and my family
Very affordable

Kyriakos Souliotis
 Appropriateness
Transfusion unit Outpatient g.h. services Inpatient g.h. services

Prescription Dispensing Lab tests

Not appropriate at all I feel I do not receive the care I need most The care I receive appears appropriate but
of the tines I have second thoughts and look for
Mostly appropriate Very appropriate additional information / services

Kyriakos Souliotis
 Availability
Transfusion unit Outpatient g.h. services Inpatient g.h. services

Prescription Dispensing Lab tests

Not available at all Somewhat available, but I am frequently Mostly available, but this depends on when,
faced with unavailability and have to wait where and from whom I request these services
Regularly available Always available

Kyriakos Souliotis
 Key findings
Access to primary care services poses the greatest challenge for transfused β-thalassaemia patients

Transfusion units

Higher access scores

Inpatient services Lower access scores

Medications

Lab tests

Outpatient services

Kyriakos Souliotis
 Next steps
Benchmarking access to care across systems and disease categories using the customized 5As framework

Going forward, the tool could be used to assess level and barriers of access to care in the same
disease area across more European countries.

This could contribute to the development of a benchmarking dataset of performance of health

systems on access in specific disease areas, such as thalassaemia.

We, as PACT, hope to be able to correlate survey outcomes with system characteristics (structure,
organization, funding) and produce integrated, disease specific access reports.

“surgical” health to improve and measurably enhance

system effectiveness at system performance and
interventions the disease level patient experience with care

Kyriakos Souliotis
 References
NIH, Genetics Home Reference, Beta –thalassaemia, US National Library of Medicine, https://ghr.nlm.nih.gov/condition/beta-thalassemia

Triantafyllou A, Vyssoulis G, Karpanou E et al. Impact of β-thalassaemia trait carrier state on cardiovascular risk factors and metabolic profile in
patients with newly diagnosed hypertension. J Hum Hypertens. 2013; 28 (5): 328-332. doi:10.1038/jhh.2013.102

Voskaridou E, Ladis V, Kattamis A et al. A national registry of haemoglobinopathies in Greece: Deducted demographics, trends in mortality and
affected births. Ann Hematol. 2012;91(9):1451-1458. doi:10.1007/s00277-012-1465-7

Kalleas C, Anagnostopoulos K, Sinopoulou K et al. Phenotype and Genotype Frequency of β-Thalassemia and Sickle Cell Disease Carriers in
Halkidiki, Northern Greece. Hemoglobin. 2011;36(1):64-72. doi:10.3109/03630269.2011.642489

De Sanctis V, Soliman A, Elsefdy H et al. Bone disease in β thalassemia patients: past, present and future perspectives. Metabolism. 2018;
80:66-79. doi:10.1016/j.metabol.2017.09.012

Kanavos P, Yfantopoulos J, Vandoros C, Politis C. The economics of blood: Gift of life or a commodity?. Int J Technol Assess Health Care.
2006;22(3):338-343. doi:10.1017/s0266462306051233

Fragoulakis V, Stamoulis K, Grouzi E, Maniadakis N. The Cost of Blood Collection in Greece: An Economic Analysis. Clin Ther. 2014;36(7):1028-
1036.e5. doi:10.1016/j.clinthera.2014.05.003

Marantidou O, Loukopoulou L, Zervou E et al. Factors that motivate and hinder blood donation in Greece. Transfusion Medicine.
2007;17(6):443-450. doi:10.1111/j.1365-3148.2007.00797.

Hauk L. Severe blood shortages reinforce the importance of reducing wastage. AORN J. 2018;107(5):P4-P4. doi:10.1002/aorn.12160

Souliotis K, Hasardzhiev S, Agapidaki E. A Conceptual Framework of Mapping Access to Health Care across EU Countries: The Patient Access
Initiative. Public Health Genomics. 2016;19(3):153-159. doi:10.1159/00044653

Kyriakos Souliotis