Designing a Regulatory Framework for Ultra-Orphan Drugs

In Regulation & Government by Cameron

UK BioIndustry Association ultra rare orphan

Earlier this week the UK BioIndustry 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 to treat the treatment of rare diseases and more common diseases equally, 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.

Here are the BioIndustry Association’s recommendations:

Consideration 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.
The new validated NICE framework should build upon the validated Advisory Group for National Specialised Services (AGNSS) framework. The creation of this framework involved a wide range of stakeholders and careful analysis. NICE should be expected to build upon this expertise. In some areas this is clearly already happening.
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.

Outside of these recommendation though I believe The BioIndustry Association hits on something much more profound in the wider scheme of orphan drugs. The authors mention the increasing level of personalised medicine and raise concerns that increasingly specialised/personalised medicines could well result in a wave of drugs which meet the current criteria for orphan drug status but which treat more common diseases which already have broader (though perhaps less effective) treatments. This would of course be a financial nightmare for regulators who could find themselves subsiding a lot more research and paying much higher prices for personalised treatments replacing broader treatment options. The answer that has already emerged for such a problem is the need for developers to demonstrate the use of distinct technologies for each drug, avoiding any chance of orphan status for drugs that have merely been ‘tweaked’.

But the BioIndustry Association offers a caution that criteria could well stifle the creation and eventual affordability of innovative new products. Their recommendation is that, in harmony with their more general recommendations, the evaluation of rare disease treatments in regards to cost, value for money, and patient outcomes needs to be approached in a unique fashion, utter distinct form the frameworks utilised for more common treatments.

Budget holders may understandably be nervous of the financial implications of this new wave of personalised medicines… One can see that criteria have been established to take account of these issues, including reference to a need for the technology to be clinically distinct and that it is to be used exclusively for that purpose. The impact this will have on eligibility of innovative new products will need to be monitored.

Find the full report here.
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