Transparent mechanisms for deciding on the use of high-cost drugs for rare disorders are needed, says Michael Sobanja

Orphan drugs aremedicines intended to treat rare diseases and some 434 products fall into this category. However, with a lack of knowledge in the NHS and inconsistency in funding, are patients being treated fairly?

It has been estimated that there are 6,000-8,000 rare conditions in the EU requiring treatment by orphan drugs, out of a total of 30,000 worldwide. Examples includeacute promyelocytic leukaemia, malignant gastrointestinal stromaltumours, Fabry disease and narcolepsy.

There is considerable variation in the attitude to orphan drugs across Europe. Early access to these medicines is typically easy inGermanyandSweden,slow inAustriaand the UKand extremely slow inBelgium.

In February, a survey was carried out in the NHS to examine theUKposition in more detail. Participants included those employed in medicines management, public health and commissioning directorates. The results demonstrated just how far there is to go if we are to create a level playing field for people needing orphan drugs.

Although 95 per cent of respondents had heard of the term 'orphan drugs', only 55 per cent were aware they were used in rare conditions, hardly any were aware of the requirements for granting orphan drug status and nobody was aware of assistance offered by the EU. Ninety per cent of responders automatically assumed orphan drugs would be expensive.

Most respondents said they had a local process to manage the entry of new medicines, but there was no specific process for managing the entry of orphan drugs.

They reported that it was rare for local health economies to proactively review orphan drugs and most waited for a specialist to request the medicine. Most respondents would use the same criteria to assess orphan and non-orphan drugs, although they would accept that some areas would not have been covered so comprehensively in orphan drug trials.

Funding routes for orphan drugs were unclear. Different handling of drugs considered to be in the nationally determined tariff under the payment by results mechanism was reported by most respondents. None described the application of pass-through payments. Several discussed affordability and equity of access as applicable concepts in this situation. Many thought these drugs should be managed through specialist commissioning or an exceptional-use committee.

Little understanding

The results of this survey suggest that there is little common understanding of orphan drugs among those most involved in managing the entry of new medicines

Funding mechanisms also appear unclear and primary care trusts are able to regard the cost of orphan drugs as being ‘in tariff’ as a means of resisting claims for additional funding, when in reality there is flexibility for local arrangements to be agreed between commissioners and providers. This may mean patients do not receive appropriate medication on funding grounds alone.

There is now an opportunity for a better approach for patients with most rare conditions with the review of specialist commissioning. Services for these rare conditions in the future should be appropriately commissioned and the funding for medicines considered at strategic health authority level. This could help to create a level playing field and avoid unjustified postcode prescribing.

There is a strong argument that when rare conditions are managed within standard outpatients, the healthcare resource group is unlikely to be set up to manage the use of more expensive orphan drugs and these should all be considered excluded from tariff and managed through a service level agreement or as pass-through expenses.

The assessment of orphan drugs for managed entry is a complex issue. Clinical trials may be small and the standard health economic arguments may be underdeveloped or unobtainable. These important medicines should be reviewed using flexible criteria, reflecting the real need in patients and the potential benefits to individuals.

Leading role

The National Institute for Health and Clinical Excellence, the Scottish Medicines Consortium and the All Wales Medicines Strategy Group could take the lead in providing credible early reviews of these products to ensure national consistency. This would require their constitution to be varied, but in the case of NICE, a formal review is under way and there is an opportunity for revision.

The NICE citizens' council was asked to consider whether the NHS should be prepared to pay premium prices for drugs to treat patients with very rare diseases. It recommended that the NHS should consider paying premium prices based on three criteria: the severity of the disease, evidence of health gain, and whether the disease is life threatening.

The Department of Health's decision to ring-fence funding for enzyme replacement therapy for lysosomal disorders, with expected high annual costs above£100,000 per patient for a year, suggests that central government also believes premium prices should be paid. Some orphan drugs are much less expensive at around£10,000 per year and these may be expected to be funded at a local level.

The government has given its commitment to developing treatments for patients with rare conditions. This commitment may be undermined by a lack of knowledge within the NHS and a lack of a consistent approach to funding and medicines management controls. Recent changes with payment by results inEnglandhave created an opportunity for even more inconsistency. The reorganisation of specialist commissioning and a lead by the health technology bodies in theUKoffer an opportunity to produce fair solutions for patients and pharmaceutical companies.

It is time we took a more rational approach to the treatment of rare conditions. Orphan drugs have a legitimate role to play subject to effectiveness and cost considerations. Not having any transparent mechanisms to consider and decide on orphan drugs is no longer acceptable.