RI Presents Data on Impact of IRD’s

Retina International Presents Data on the Socio-Economic Impact of IRDs at Major International Conference

Retina International’s Director of Research Policy, Orla Galvin PhD, presented data from the recent cost-of-illness study, IRD COUNTS, at the biennial European Conference on Rare Diseases and Orphan Drugs yesterday, May 14th. The event, due to be held in Stockholm, Sweden, was moved online and had over 1400 attendees representing patient groups, industry, policy makers and HTA agencies, as well as researchers and clinicians.

Orla presented data from the IRD Counts pilot study which demonstrated that the model for health technology assessment in many European countries does not appropriately consider the financial and wellbeing costs on people living with IRDs and their families. In conclusion, Orla stated “Although living with individually rare conditions, IRD patients and their families have three common issues which urgently need to be addressed; the need for timely equitable access to genetic testing and counselling; the need for equality in accessing employment; and, a revision of the assessment process for reimbursement of therapies currently focused on the cost-of-illness to the healthcare system. IRD patients do not frequently engage the healthcare system and as such, suggests a cost-of-illness model from a societal perspective may be a better format.”

The session, Rare Disease in Numbers, was chaired by RI CEO Avril Daly and discussed achieving the triple A by 2030; Accessible, Available and Affordable therapies for people living with rare diseases.

In Europe, the Rare Disease community faces a number of challenges in accessing approved therapies, which indicates that the system in its current design is not functioning to the benefit of all. There is increasing concern that innovative and improved therapies will not be available to those who may benefit from them at a time when they can have the most impact on disease – or even worse, they may not be available at all.

A lack of consideration of the true value of investment in innovation for rare conditions is an ongoing challenge that can have a negative impact on drug development in this space. At the event, Avril Daly stated “Those of us who are engaged in therapy development from concept through to delivery are used to the refrain – the research is too niche to support – the cost of development is too great and the benefit for too few to have societal consequence.”

She continued “Similarly – therapies that have been through the regulatory approval process are too often significantly delayed due to weaknesses and gaps in the drug approval and reimbursement process. Approved treatments that have not been accessible in some European countries while they are widely available in others and in some, they have not even been submitted for approval. Diversity in the evaluation of value, impact and durability of orphan therapies is leading to a growing apartheid among our citizens, who are deemed to have equal rights under EU Law.”

Experts agree that there is an absence of adequate and equitable strategies and systems built on the principles of fairness, equality, value for money, transparency, effectiveness and sustainability. If left unresolved, this will lead to less innovation and greater impact on the health of our citizens.

Many agree that current frameworks are not fit to serve the Rare Disease community in particular; the result is a decrease to the quality of care and of life that all patients have a fundamental right to enjoy.  A growing body of research is pointing to the fact that the assessment of the value of investment in and access to emerging innovative technology is far too narrow – a 360° view of the full impact of innovation on the Rare Disease community should be better understood in order to make fairer decisions based on real world evidence.

The session presented examples of structured evidence generation that have the potential to contribute to practical solutions. They demonstrated how many are affected, where the impact of disease on health, on wellbeing, on independence and productivity, the value in investing in development and the emerging evidence that amplifies and validates the long held view that assessment of complex conditions cannot be adjudicated through a one size fits all process, that assessment must be fit for purpose and greater understanding of populations can lead to better outcomes for patients, governments and society at large!

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