Almost two-thirds of UK members of Parliament believe there should be no maximum price set for orphan drugs, according to research commissioned by the BioIndustry Association (BIA).
The survey of MPs comes ahead of a consultation on evaluation framework for therapies intended to treat ultra-rare diseases by the National Institute for Health and Care Excellence (NICE), which is due later this year.
It also found that 68 per cent of MPs believe access to treatments should be based on clinical need rather than the ability of the NHS to foot the bill, according to the BIA report.
Drugs used to treat rare diseases account for around 10 per cent of global pharma sales, according to consultancy Alcimed, which predicts the market for these orphan drugs will grow 7 per cent a year to reach $127bn in 2018.
Typically, pharma companies charge very high prices for orphan drugs, arguing that the high therapeutic value and small number of target patients justifies premium pricing in the context of the investment needed to develop them.
The cost of the drugs has always been controversial but is stimulating further debate with the increasing levels of investment in the area by Big Pharma companies, which see growth opportunities in a sector formerly dominated by small biotechs.
The shift towards personalised therapies means that an increasing number of drugs are now orphan or ultra-orphan therapies, and that in turn raises questions about the ability of national health systems to fund them.
“Being rare is becoming increasingly common,” notes the BIA report, which highlights the complexities in supporting R&D into rare diseases – so that they are treated equitably with more common illnesses – and making sure they are affordable.
One key concern voiced by the industry group is that responsibility to arrange delivery of ultra-orphan therapies – formerly held by the Advisory Group for National Specialised Services (AGNSS) – has now shifted to NICE.
Clearly, the methodology currently employed by NICE for more common therapies is too blunt a tool to assess ultra-rare diseases, and while it welcomes moves to adopt a specialised framework the believes this should draw on the more ‘holistic’ approach set up by the AGNSS.
“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,” it notes.
It is also important to distinguish between orphan and ultra-orphan, says the BIA, which calls for a separate framework for conditions affecting no more than 1 in 50,000 people.
“It is vitally important that patients with rare diseases get access to clinically effective medicines and that there is no discrimination against such patients when it comes to making innovative treatments available,” commented Andrew Miller, Chair of the House of Commons Science and Technology Select Committee.
“Equitable access for all patients, whether the condition is rare or common, is a fundamental principle of the NHS and the new evaluation procedure for such medicines will be an important component of this,” he added.
