Earlier this month the UK Bio-Industry Association published a report detailing their recommendations for the new ultra-orphan medicines evaluation framework for ultra-orphan drugs currently being developed by the UK’s Nation Institute for Health and Care Excellence (NICE).
The report recognises that national regulators within the EU have failed to strike an appropriate balance between the need of treatment of rare diseases, against the high cost of development and market size. The problem is that, whilst regulators wish to offer treatment to everyone, regardless of whether they have a common or rare illness, the cost of orphan drugs is substantially higher and represents a lower value for money than found amounts treatments for more common diseases. And even with tax credits and regulatory help from the EMA, orphan drug costs remain high.
The report highlights a further side to the coin which could see the EMA subsidising the development of medicines which then cannot be given to patients on the basis of high cost. But despite these monetary concerns and the European climate of financial austerity, British members of parliament remain broadly in favour of equal entitlement to treatment. 68% of MPs agree or strongly agree that access to treatments on the NHS for very rare diseases should be based on clinical need and not the NHS’s ability to pay.
The Bio-Industry Association has made a complex and articulated recommendation.
They say onsideration should be given to the incentives to develop orphan and ultra-orphan medicines and avoid the outcome whereby clinically effective medicines have been developed but patients cannot access them. Governments have recognised the societal need to provide equal opportunity for treatments and have sought to incentivise the research and development of orphan and ultra-orphan medicines. Such incentives are undermined if these principles are not recognised and adopted further down the development and regulatory process.
Ultra-orphan medicines require a separate evaluation framework. There is a clear and justifiable need for this because of the unique characteristics and challenges involved with evaluating medicines for very rare conditions. The independent research released in this document would appear to demonstrate political support for this.
There is a need to ensure integrated implementation and service delivery. There is a further need to closely align the evaluation and service delivery elements of ultra-orphan medicines recognising that the functions are now split between NICE and NHS England whereas under the AGNSS framework they were considered together.
In a recent draft guidance, the UK’s National Institute for Health and Care Excellence (NICE) asked Alexion Pharma UK to provide information regarding the cost of manufacturing, research and development of its orphan drug Soliris (eculizumab). The Institute, which assesses the cost-effectiveness of drugs for use on the NHS, said that it considers eculizumab an effective treatment for the extremely rare condition atypical haemolytic uraemic syndrome (aHUS). It also pointed out that it is well aware of the special pricing concerns with orphan drugs. Andrew Dillon, Chief Executive at NICE, commented: “Drugs for very rare conditions that affect just a few people in the country are inevitably more expensive than for more common diseases.” However, in light of the extremely high treatment cost of £340,200 (€414,121) per adult patient in the first year, NICE needs more information.