Severely ill to get 'early access' drugs

Patients that are suffering from rare debilitating conditions, and those that are severely ill, could be able to get new drugs ahead of their official licensing under new rules.

The Medicines and Healthcare Products Regulatory Agency (MHRA) will oversee the Early Access to Medicines scheme, which would enable a small number of medicines to be fast-tracked through the approval process, in a bid to help patients across England that have no other treatment options available to them.

The MHRA has stressed that the scheme, which will be launched in April, would not replace the normal system of checks and research that drugs have to go through - including clinical trials - before they are permitted to be used.

Health minister Jeremy Hunt said: "What patients want is sometimes to try medicines that may not be clinically proven to be effective but are clinically safe."

Mr Hunt added that the government is "streamlining the process" so that medicines can help patients much earlier, especially if they have "early promise". He continued to say that the new scheme will "bring hope to a lot of patients".

The normal process can typically take around a decade for a drug to be approved, which includes vast amounts of research, patient trials and assessments. For drugs to bypass this system, the unlicensed medicine must have evidence from several years of clinical trials that show it is a promising and innovative treatment.

Patients that are classed as seriously ill, with conditions like cancer, would be eligible for the scheme if no other treatments are available. However, the MHRA will still have to carry out a number of checks and assessments on the unlicensed drug to determine whether or not it is safe to use.

The MHRA has estimated that one or two medicines a year could qualify under the scheme, although some in the pharmaceutical industry think it could be higher.

Harpal Kumar, chief executive of Cancer Research UK, told the BBC: "Time is of the essence for many cancer patients, particularly those with more advanced disease.

"It can mean the difference between life and death. Therefore this scheme, which has at its heart the potential to bring promising new medicines to patients faster, is to be warmly welcomed."