Co-authored by: Dr Kate Armstrong (Caring & Living As Neighbours)

1. A Rapid Assessment Protocol for Improving Access (RAPIA) to Medicine and Care for Children living with a Chronic Condition (Congenital Adrenal Hyperplasia) in Vietnam David Beran on behalf of Kate Armstrong Co-authored by: Dr Kate Armstrong (Caring & Living As Neighbours) David Beran (International Insulin Foundation) Assoc Prof Maria Craig (The Children’s Hospital at Westmead) Claire Henderson (Caring & Living As Neighbours)

2. Chronic Disease & Children • Chronic health conditions are a major global health policy issue (UN High Level Meeting on Non-Communicable Diseases (NCDs) in New York, September 2011) • NCDs and other chronic health conditions affect children and not just adults. Almost 1 in 4 children in the US are estimated to have a chronic health condition; 1 in 10 are affected by Asthma alone. • Barriers to affordable access to essential medicine and care result in increased morbidity and mortality for children with chronic health conditions eg. Insulin and expert care required for Type 1 Diabetes. • Children in low and middle income countries (LMICs) are at especial risk. In 2002, more than 1.2 million people below the age of 20 died of a NCD. Surveys in LMICs indicate almost 1 in 4 children between the ages of 2 and 9 years have (or are at risk of) a disability

3. It’s not “too hard” • CLAN (Caring & Living As Neighbours) is an Australian-based NGO committed to helping children with chronic health conditions in LMICs enjoy the highest quality of life possible • Identifying children with the same chronic health condition as members of a non-geographically based “community”, CLAN collaborates with a range of multisectoral partners to support rights-based, community development approaches to sustainable, scalable, population-based change • CLAN’s strategic framework for action focuses the actions of community members and all partners on “5 Pillars”: • Affordable access to medicine and equipment • Education, research and advocacy • Optimisation of medical management • Encouragement of family support groups • Helping families achieve financial independence and overcome poverty

4. Example of an NCD in Childhood - CAH • Congenital adrenal hyperplasia (CAH) is the most common adrenal condition of childhood • Genetically acquired. Not curable. Lifelong condition • Cortisol (as hydrocortisone tablets or injection during emergencies) and aldosterone (as fludrocortisone tablets) replacement are essential to survival. • Medicine is taken two to three times a day in childhood • Failure to take any medicine is not compatible with survival. Under-dosing results in short stature, precocious puberty and virilisation. With correct dosing children can enjoy a normal quality of life.

5. Understanding CAH in Vietnam • In 2004, CLAN became aware of the high mortality and morbidity associated with CAH in Vietnam through anecdotal reports • In 2005, a needs analysis of families determined unaffordable access to medicine was their primary concern • Children were being diagnosed at birth, but lost to follow-up (sharp drop-off in age distribution beyond early childhood) • The average family income for children not lost to follow-up was about 40% higher than the national average income and 93% higher than income of rural poor families

6. Objectives: • The Diabetes RAPIA survey was conducted in Vietnam by the IIF in 2008 to explore barriers to insulin access and diabetes care. • CLAN took this opportunity to implement an adapted protocol and clearly identify barriers to accessing medicine and care for children with CAH in Vietnam. • Although the exact incidence of CAH in Vietnam is not yet known, initial newborn screening trials in 2007 suggest it may be higher in Vietnam (closer to 1:6,000, as is found in the Philippines) than Australia, the United States, and the United Kingdom (generally around 1:18,000).

7. Design: • Structured surveys and templates used in the Diabetes RAPIA were adjusted to specifically address CAH. • The CAH RAPIA was not a statistical assessment of the Vietnamese health system, but rather a rapid collation of qualitative and quantitative data to analyse CAH in a low-income setting.

8. Design Care Medicines Other Problems Ministry of Health Country Individual

9. Design Multi-level assessment of Health system • Micro • Healthcare Workers • Traditional Doctors • Patients • Meso • Regional Health Organisation • Hospitals, Health Centres, etc. • Pharmacies, Drug Dispensaries • Macro • Ministry of Health • Ministry of Trade • Ministry of Finance • Central Medical Store • National Diabetes Association • Private/Public drug importer • Educators Perspectives on the problem of access to Insulin and Diabetes care Beran, D et al. BMC Health Serv Res, 2006

10. Setting: • The CAH RAPIA was a multi-level assessment of factors influencing access to medicine and care for people living with CAH in Vietnam, and had three components: macro (ministerial levels, private sector), meso (provincial levels and health care settings), and micro (caregivers and people living with CAH). • Data were collected in Hanoi, Ho Chi Minh City, Thai Nguyen Province, and Dong Nai Province.

11. Study Population: • 204 interviews (for diabetes and CAH) were conducted. • Participants were selected on the basis of role (ministerial and health sector) and convenience sampling (CAH families) from the four provinces (two mainly urban and two relatively wealthy and urban) • The three largest Children’s Hospitals in Vietnam were involved in the CAH RAPIA (CAH is a complex chronic condition, and health professionals at provincial hospitals are not trained in the management of CAH, hence all children with CAH once diagnosed are generally referred to at least one of these three hospitals)

12. Outcome measures for CAH RAPIA: • Understanding key barriers to accessing medicines and care for people living with CAH in Vietnam • Using CAH RAPIA to inform future action to maximise quality of life for children living with CAH in Vietnam • Focus on key policy change (particularly regarding registration and importation of hydrocortisone and fludrocortisone and financial burdens on CAH families). • Advocacy for change at all levels • Family and community level • Local health facility level • Ministry of Health • WHO

13. Results of the CAH RAPIA • Lack of older children with CAH (over 90% of patients registered with CAH were younger than 15), • interviews withhealth care workers confirmed that more young children with CAH were surviving than had been the case previously • 60% of families identified financial burdens (cost of medicines in the main) as their greatest concern. • Barriers to affordable access to hydrocortisone and fludrocortisone were identified as a mix of national and international factors: • neither drug included in the WHO EMLc, • neither drug registered in Vietnam, with variable pricing and quality a result • Barriers to care that were identified included: • lack of access to trained health professionals, particularly beyond major centres; • travel costs • health systems not developed for paediatric chronic disease • clinical infrastructure unavailable • a mismatch between actual and insurance-approved referral pathways. • CAH family support clubs were identified as effective for education and support.

14. Conclusions: Positive outcomes for the CAH Community in Vietnam have included: • Raised understanding and awareness: • RAPIA highlighted key opportunities for ongoing action; shared with all stakeholders • Comprehensive educational resources on CAH translated into Vietnamese language for Club meetings now available to Universities, Hospitals and all CAH families in Vietnam • Media (national TV) has attended annual CAH Club meetings (500+ people attend) in 2010 and 2011 • Grassroots work informed advocacy ahead of the UN High Level Meeting on NCDs in 2011 • Measurable improvements in quality of life and health outcomes for children with CAH: • Increase in number of survivors (a 5 fold increase over the last decade) • Decline in mortality • Urgent and random presentations to Emergency and Outpatient Departments dropped markedly (family education & improved access to medicines); scheduled reviews most usual presentation now • CAH Club meetings now scheduled to run in provinces (saves families travelling to capital cities) • New healthy public policy decisions: • CLAN partnered with other NGOs to successfully advocate for inclusion of hydrocortisone and fludrocortisone tablets in the WHO essential medicine list for children (EMLc) - approved October 2008 • 2010 - Vietnamese government imports both hydrocortisone and fludrocortisone tablets • 2011 - hydrocortisone and fludrocortisone tablets both included in the national insurance scheme for inpatients and outpatients, making them affordably available for all • 2010-11 - Vietnamese Ministry of Health includes CAH within National Newborn Screening Panel • Hospital Outpatient Departments streamlining & systematising routine care

15. Implications • A strategic, community development approach (utilising CLAN’s 5 Pillars) has been effective in facilitating sustainable, long-term improvements in quality of life and survival for children living with CAH in Vietnam • The RAPIA is an adaptable tool that empowers communities to improve access to essential medicines by helping all stakeholders better understand the key barriers to access and affordability • Framing the RAPIA within a broader grassroots, community development strategic approach to NCDs has the potential to effect population-based change • A model that combines CLAN’s 5 Pillars and the RAPIA could be adapted to other chronic conditions of childhood (and adults)

16. Acknowledgements • The CAH RAPIA was funded by CLAN with generous practical support from David Beran of the International Insulin Foundation.