“Uncertainty and price” were the two words used by Dr Anna Bucsics of MoCA to summarise discussions held at this week’s World Orphan Drug Congress, Europe in Barcelona. As is typical with rare diseases, uncertainty challenges include data collection to assess the natural history of disease, relevant outcomes for marketing authorisation and reimbursement and finally long-term outcomes over many years. There are registries in place for various rare diseases and the EU is investing in the development of European Reference Networks, however the main aim of these networks is to support the delivery of healthcare rather than data collection. Particular challenges lie in the collection of data over the long term. There are no models to measure the lifetime benefits of a single treatment and developing the correct technology to support data collection over many years will be a challenge in itself.
What was particularly surprising to hear was that, once treated with a cell or gene therapy and disease free, some patients are no longer interested in attending clinics for follow-up; making it even more difficult to gather data on long term outcomes to support reimbursement criteria. While patient reported outcomes were discussed as particularly useful for orphan drug reimbursement negotiations, there was also a suggestion that long-term follow-up could possibly be supported by telehealth initiatives. The manufacturing logistics of cell and gene therapies is a major consideration for patient access and the requirement for patients to travel to treatment centres possibly outside their own country can lead to specific challenges both legal and financial.
Early discussions with regulators are encouraged by all companies in this area to agree relevant clinical trial endpoints. However some heated debate took place around the concept of early discussions with payers which was viewed by many as a potential inhibitor of innovation, due to the expectation of high prices. Manufacturers expressed their concern regarding reimbursement at prices required to cover basic manufacturing and logistical supply, regardless of securing a return on research and development investment. Key issues outlined for HTA agencies include evidence requirements, budget sustainability and price justification. Not many countries were considered to have special considerations for rare disease treatments when it comes to reimbursement. It would appear that the NICE HST process has been successful in facilitating access in the UK to a greater number of treatments eg. CAR-T therapies, when compared with France, Germany, Italy and Spain. Reimbursement criteria were a significant discussion item; in particular the use of cost per QALY versus multi criteria decision analysis for reimbursement assessments. There was extensive debate around the value of managed entry agreements and particularly the complexity of risk sharing and pay for performance schemes where implementation remains a challenge. While value assessment at EU level is considered to be desirable, pricing negotiations remain a subject for national review. However there is an expectation that there will be some collaboration between EU member states with the Benelux countries and Austria already conducting joint pricing negotiations for some rare disease treatments.
With 39 gene therapies expected to be licensed by 2022, the small patient numbers and limited datasets for rare diseases require innovative long term solutions to address uncertainty and pricing issues and secure patient access to these treatments.
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To read more about the meeting visit https://www.terrapinn.com/conference/world-orphan-drug-congress/index.stm