Europe is within reach of a major transition to improve the lives of the 30 million persons living with a rare disease and their caregivers. The United Nations has just adopted its first-ever Resolution on Persons living with a Rare Disease, the French EU Presidency is prioritising rare diseases, starting with a ministerial meeting in February to coincide with Rare Disease Day, and the EU plans to revise rules governing medicines and legislation on blood, tissues and cells.
There is no better time to rethink and rework the rare disease eco-system – and to do this in partnership. The need is evident. For the 5% of rare diseases with at least one authorised treatment, the standard of care for many patients can still be improved. And it’s vital to continue the search for treatments for the remaining 95% of rare diseases, and overcoming the challenge that more than 80% of rare diseases have a prevalence of less than 1 in 100,000. Diagnosis is often late and patients face a bewildering odyssey through healthcare systems that often starts early in childhood.
READ MORE: This article is published as part of our special supplement looking at how Europe can come together to find solutions for people suffering from a rare disease. To read the full supplement click here.
Plasma-derived therapies, for instance, are critical life-saving and life-sustaining medicines that are often the only treatment option for people with rare and complex chronic diseases, like immunodeficiency diseases. They can only be made from human plasma. Agreement is needed urgently on how to increase plasma donation in Europe to meet growing demand for these medicines - otherwise, more patients lives may be at risk.
Takeda and the private sector are ready to partner to bring treatments and solutions for persons living with a rare disease into a new era
Today, we must address issues relating to the entire cycle of drug development for tackling rare diseases. Europe faces challenges in basic research, diagnostic capacity, infrastructure, consensus on regulatory and market access evidence, uptake of innovation, equitable access to treatment, and security of supply. Incentives for more innovation in rare diseases might come from evolving the current EU regulation on orphan drugs to create more incentives for under-invested disease areas, while recalibrating rewards where progress in standard of care will not be impacted.
A Takeda-commissioned study identified practical potential mechanisms to unlock wider acceptance of new medicines across Europe, such as a public-private partnership for basic research funding that captures the patient perspective, an early evidence partnership to increase predictability among developers, regulators and payers, or value-based risk-sharing contracts, where appropriate. These partnerships would promote a more comprehensive and effective vision of European intent, that recognises that science is not linear, that shares the data that feeds research, permits diagnosis, and translates innovation into treatments for patients.
It’s encouraging to see more unity and closer collaboration through Rare 2030 and a European Action Plan for Rare Diseases, and we’re looking forward to achieving tangible results in the near future. The pandemic has unveiled innovative new ways of working and has created unexpected but valuable alliances that we can build on. Takeda and the private sector are ready to partner to bring treatments and solutions for persons living with a rare disease into a new era.