OBJECTIVES: Berger’s disease is a chronic progressive orphan renal disease which is a major cause of kidney failure in Europe. Up to 50% of the patients undergo dialysis after 20-30 years of diagnosis. It affects around 130,000 in the 5 major European markets. There are no indicated therapies, except for treatments for the exacerbating factors of the disease. This project aimed to assess the market access landscape for Orphan renal disease and to understand key reimbursement challenges for drug innovation in this therapy area

METHODS: In-depth interviews with ex-payers in HTA related organisations in EU5 and KOLs in clinical nephrology specialties to provide understanding of the disease area, the key unmet needs and access challenges

RESULTS: The market is dominated by off-label drugs like ACEi/ARBs and corticosteroids in severe cases (proteinuria >3g/kg). The payer interviews revealed that development of disease modifying therapy is difficult because of the long-term safety issues and challenges in producing evidence on cost offsets of dialysis. End points in clinical trials such as glomerular filtration rate (eGFR) and total protein to creatinine ratio (UPCR) linked to improvement in QoL and morbidity were considered valuable for emerging therapies. However, early diagnosis is difficult with most patients being diagnosed at later stages of the disease when they are already in a high-risk group

CONCLUSIONS: End points for clinical trials of new disease modifying therapies should involve improvement of clinical kidney parameters such as eGFR. The patient population is not clearly defined because of lack of proper diagnosis or no clinical symptoms in early stage of disease. The presence of very low-cost therapies could pose a problem for Payers in budget impact estimations and is likely to reduce their willingness to pay