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“2020health is an important and thoughtful contributor to the health debate”

Dr Sarah Wollaston MP, Chairman, Health Select Committee

 
 

Rare & Forgotten Conditions

Research

2020health Report Orphan Medicines: Special treatment required?

Orphan Medicines: Special treatment required?

This report was prompted by our concern that patients and their clinicians are not always able to access medicines for those with rare diseases. The ongoing reforms to the NHS and development of a strategy for rare disease offer an opportunity to explicitly consider access to medicines for those with orphan or ultra-orphan conditions. read on Orphan Medicines: Special treatment required?

2020health Report Forgotten Conditions: misdiagnosed and unsupported, how patients are being let down.

Forgotten Conditions: misdiagnosed and unsupported, how patients are being let down.

This paper is a report on a roundtable held by 2020health on 12 June 2012 in Portcullis House, London, on “Forgotten Conditions”. These are health conditions and diseases that due to their low prevalence mean patients often get neglected or overlooked. The event was hosted by Andrew George MP and chaired by Lord Clement-Jones. read on Forgotten Conditions: misdiagnosed and unsupported, how patients are being let down.

In Europe, a disease is considered rare when it affects fewer than one in 2,000 people.  For some of these rare disease there are drugs approved for treatment.  This means they offer more clinical benefits than harm, at an acceptable risk, as determined by the regulatory agency, the European Medicines Agency (EMA).  Some are formally designated as ‘orphan’ drugs in the European Union (EU), treating less than 5 in 10,000 people in the EU.  However, not all approved drugs will be paid for (reimbursed) by the National Health Service (NHS).  That reflects the need to consider value for money. This can be a source of frustration for many: patients, carers, and treating clinicians. Evidence suggests that current access to orphan drugs in the UK is at best inconsistent: whether you look internationally, or speak to clinicians and patient organisations who help individual patients when access is blocked within the UK. This is not a fair situation for those who face serious illness.

Successfully bringing a medicine all the way from initial basic research to the patient is difficult. The pathway for access to orphan drugs includes a number of key decisions: those made by researchers and companies during the research and development (R&D) phase, decisions by regulators, payers and those that advise payers as well as prescribing clinicians who make decisions with their patients.