EUROPEAN-ADPKD-FORUM

Today marks the publication of a report by the European ADPKD Forum (EAF), a multidisciplinary group of leading medical and patient group experts dedicated to improving the health and quality of life of people with autosomal dominant polycystic kidney disease (ADPKD).

ADPKD is a chronic, progressive, inherited disease in which fluid-filled cysts grow in the kidneys and liver.¹ ADPKD is an important cause of chronic kidney disease and kidney failure, affecting hundreds of thousands of people in Europe. It is responsible for up to one in ten of all patients needing dialysis or transplantation, corresponding to approximately 50,000 people across Europe.² This figure represents only a proportion of the total number of cases as many patients living with ADPKD have yet to develop kidney failure or remain undiagnosed.

The report, entitled ‘Translating Science into Policy to Improve ADPKD Care in Europe’ , reveals that, despite European Commission (EC) initiatives to tackle health inequalities, widespread variations in ADPKD care exist² owing to a lack of coordinated policies and standardised care pathways. The EAF makes six policy-focused recommendations to help address the existing unmet needs and to promote access to high-quality care for all patients with ADPKD in Europe.

The EAF Report first highlights the significant and under-recognised impact on patients, their families, and healthcare systems in Europe. As ADPKD is incurable, it has a lifelong impact on patients’ quality of life; people with ADPKD commonly suffer from acute and chronic pain,^3-8 and are more likely to experience anxiety and depression compared to the general population.^9,10

Patients with ADPKD need lifelong care, the costs of which rise precipitously when kidney failure occurs and dialysis or a kidney transplant is needed.^11,12 ADPKD accounts for around one in ten patients needing these treatments, at a cost of €1.5 billion/year across Europe.¹³ These costs are expected to rise as the number of recipients increases and their life expectancy improves.¹³

Commenting on the publication of the EAF report, Dr Richard Sandford, Consultant Clinical Geneticist at Addenbrooke’s Hospital, Cambridge, UK, and co-chair of the EAF, said: “Previous and sometimes outdated approaches to the management of ADPKD must be reconsidered if health services are to meet the quality improvement targets that the European Commission has identified. Innovative, long-term solutions should be sought to ensure that health services can meet the clinical, financial and societal challenges that ADPKD represents.”

The EAF report recommends the following key strategies for policy-makers to improve ADPKD across Europe in context with relevant European Union (EU) policy initiatives:

• The implementation of tiered care: Governments should support the development of a nationally co-ordinated, tiered approach to ADPKD care in collaboration with experts, patient advocacy organisations and other stakeholders, in line with the European Commission (EC) policy priority to address health inequalities
• The establishment of specialist ADPKD centres: An expanded European Reference Network of specialist ADPKD centres should be established to facilitate further research and harmonised, integrated, patient-centred care pathways, ensuring best practice and consistency of care
• Supporting therapeutic innovation: The EC and national governments should support research to develop disease-modifying treatments with the potential to maintain quality of life, delay renal decline and improve life expectancy among patients and to reduce the economic impact on healthcare systems
• Prioritisation of prognosis: Governments and healthcare providers should support the routine implementation of methods to assess prognosis in ADPKD to inform clinical decision making, research and innovation
• Empowering patients: All stakeholders, including the European Commission, national governments and healthcare providers, should support efforts to better inform individual patients and families affected by ADPKD, and to involve patient organisations in policy making regarding healthcare planning and delivery related to ADPKD
• Engaging patients in health technology assessment: Health Technology Assessment (HTA) organisations should seek to engage ADPKD patient organisations in their assessments in order to consider their unique knowledge about the impact of living with ADPKD, and their aspirations for new treatments, according to the HTA International Quality Standards for Patient Involvement in HTA

The EAF recommendations were developed in line with the EC’s Reflection Process on Chronic Diseases, which calls for “a reorientation of budgets towards innovative approaches with an impact on the quality of life of people affected or at risk of chronic diseases [requiring] changes in the systems, infrastructures, policies and legislation”.¹⁴

Commenting on the publication of the report, Tess Harris, co-chair of the EAF and President of PKD International, said: “The EAF report provides comprehensive evidence on the significant impact of ADPKD on health systems and patients throughout Europe. The policy recommendations outlined in the Report, which are the culmination of years of research and analysis, must now be implemented to ensure that the promises made by the European Commission are fulfilled and the development and delivery of care improves patient outcomes throughout Europe.”

The EAF Report is endorsed by two leading patient organisations PKD International and the Patients Network for Medical Research and Health (EGAN). Its recommendations have been captured in the Brussels Declaration on ADPKD which is being launched today alongside the report as a stand-alone EU policy action plan, which should now serve as a backbone for ADPKD advocacy across Europe. The EAF Faculty will invite all other stakeholders to support the Brussels Declaration and to work together in order to see these important strategies translated into concrete action at national level.

- ENDS -

NOTES TO EDITORS

ABOUT ADPKD:

ADPKD is an important cause of chronic kidney disease and kidney failure, affecting hundreds of thousands of people in Europe. It is responsible for up to one in ten of all patients needing dialysis or transplantation, corresponding to approximately 50,000 people across Europe.¹³ This figure represents only a proportion of the total number of cases as many patients living with ADPKD have yet to develop kidney failure or remain undiagnosed.

The findings of the largest ever survey of people with ADPKD in Europe detailed in the report, conducted among 730 people with the condition, highlighted the negative impact the disease has on families (77% of respondents), relationships (41%), sexual relationships (42%), social lives (33%) and the decision to have children (35%).¹⁵

For more detailed information about ADPKD, see the accompanying media backgrounder ‘About ADPKD in Europe’.

ABOUT THE EAF

The EAF is a multidisciplinary, international faculty of experts dedicated to improving the health and quality of life of people with ADPKD. The EAF was initiated by, and is solely supported financially by, Otsuka Pharmaceutical Europe Ltd. Faculty members receive no fees in respect of their roles in the initiative. The contents of the EAF Report and Brussels Declaration on ADPKD are the opinions of the EAF Faculty and do not necessarily represent those of Otsuka.

The EAF initiative has been funded by Otsuka Pharmaceutical Europe Ltd. Editorial control of the EAF report remains with the faculty and their opinions do not necessarily represent those of Otsuka.

OPEL/0814/GEN/1044

January 2015

REFERENCES:

1. Torres VE , et al. Autosomal dominant polycystic kidney disease. Lancet  2007;369:1287–301

2. Spithoven EM , et al. Analysis of data from the ERA-EDTA Registry indicates that conventional treatments for chronic kidney disease do not reduce the need for renal replacement therapy in autosomal dominant polycystic kidney disease. Kidney Int  2014 Dec;86(6):1244-52

3. Bajwa ZH , et al. Pain patterns in patients with polycystic kidney disease. Kidney Int  2004;66:1561–9

4. Oberdhan D, et al. Patient-reported pain in autosomal dominant polycystic kidney disease: initial concepts based on patient focus group discussions [Abs SA-PO283]. J Am Soc Nephrol 2013;24 Abs Suppl:692A

5. Oberdahn D, et al. Patient experience with pain related to autosomal dominant polycystic kidney disease (ADPKD) [Abs PUB285]. J Am Soc Nephrol 2014;25 Abs Suppl:960A

6. Miskulin DC , et al. Health-related quality of life in patients with autosomal dominant polycystic kidney disease and CKD Stages 1-4: a cross-sectional study. Am J Kidney Dis 2014;63:214–26

7. Heiwe S , et al . An evil heritage: interview study of pain and autosomal dominant polycystic kidney disease. Pain Manag Nurs   2009;10:134–41

8. Casteleijn NF , et al. A stepwise approach for effective management of chronic pain in autosomal-dominant polycystic kidney disease. Nephrol Dial Transplant  2014;29 suppl 4:iv142–53

9. de Barros BP , et al. Anxiety, depression, and quality of life in patients with familial glomerulonephritis or autosomal dominant polycystic kidney disease. J Bras Nefrol  2011;33:120–8

10. Pérez-Dominguez T , et al. Progression of chronic kidney disease. Prevalence of anxiety and depression in autosomal dominant polycystic kidney disease. Nefrologia  2012;32:397v9

11. Knight TG, et al. Economic burden of autosomal dominant polycystic kidney disease in the United States (Abstract 160). Am J Kidney Dis 2013;61(4):A55

12. Lentine KL , et al. Renal function and healthcare costs in patients with polycystic kidney disease. Clin J Am Soc Nephrol  2010;5:1471–9

13. Spithoven EM , et al. Renal replacement therapy for autosomal dominant polycystic kidney disease (ADPKD) in Europe: prevalence and survival-an analysis of data from the ERA-EDTA Registry. Nephrol Dial Transplant  2014;29 (Suppl 4):iv15-iv25

14. European Commission. Reflection process on chronic diseases (final report; 12983/13). Brussels , 2013

15. Otsuka Pharmaceutical Europe Ltd. Market research data, 2014

Contact:

Virgo Health for European ADPKD Forum
Ed Purkis
T. +44 (0)20 8939 1261
Ed.Purkis@virgohealth.com
or
Adam Pittard
T. +44 (0)20 8939 1274
Adam.Pittard@virgohealth.com