Managed access programmes (MAPs) have been around for some time, offering poorly people access to drugs that aren’t currently fully licensed or are unavailable in a particular country.
Patients often receive the new, unapproved medication on compassionate grounds, while the MAPs themselves tend to be overseen by specialist drugs firms.
Here in the UK, Clinigen has run over 250 programmes providing ‘off-label’ treatments to those in acute medical need.
Eighteen of the top 20 big pharmaceutical companies now provide early access to their drugs.
Source of rich data
In fact, MAPs are becoming a rich data source with patient-reported outcome information bundled up with the stats gleaned from clinical trials, which is then submitted to the regulator.
“Merck ran a MAP programme for Keytruda that formed an invaluable part of the regulatory filing that they were able to get reimbursement on,” said Tim McCarthy, chairman of ImmuPharma (LON:IMM).
Keytruda is one of a new breed of immunotherapy drugs that could help revolutionise the treatment of the killer disease.
McCarthy’s reference to the Merck breakthrough was by way of example as of what can be achieved using MAPs as ImmuPharma prepares to go down the same route.
500 patients to be treated
It will offer 500 patients with the autoimmune disease lupus access to Lupuzor, which generated mixed data in phase III clinical trial.
“We can get valuable data on the drug,” McCarthy told Proactive’s, Andrew Scott.
“We call this real-world data. I should stress, this is not a clinical study. All patients will get the drug, there will be no comparator group, blinding or randomisation.”
The MAP will be around a tenth of the cost of the last ImmuPharma clinical with patients receiving the medication free of charge for two years.
“This approach is getting a lot of traction now in our industry,” said McCarthy.
Despite the phase III setback for Lupuzor, ImmuPharma still plans to make a regulatory filing.