TB: last hope denied
Health-e News Service
SA’s Medicines Control Council (MCC) has confirmed that it will continue to deny compassionate access to a TB drug that for many drug-resistant patients is their last hope.
Compassionate use allows for the dispensing of investigational drugs (with good efficacy and safety in trials) before they are registered and enter the market.
Bedaquiline (TMC207) is being made available under compassionate use in several countries with stringent regulations on drug safety – including France, Germany and the UK.
These countries have made the drug available under compassionate-use regulations, despite having far fewer patients.
Bedaquiline is not registered anywhere in the world, but its application for registration is being fast-tracked in the US and Europe seeking conditional approval to make it available.
Médecins Sans Frontières (MSF), also known as Doctors Without Borders, in SA have tried using bedaquiline for a select few patients with pre-extensively drug-resistant (pre-XDR) and multidrug-resistant (MDR) TB.
But several attempts by MSF to gain compassionate access have been declined by the MCC.
For many of these patients there is no other hope and bedaquiline offers a last chance of survival. Many patients have died while waiting for compassionate access to be granted.
Dr Gilles van Cutsem, medical co-ordinator at MSF in SA, said the council’s reluctance to allow compassionate use in the country is the big hurdle to access.
“Their call for a clinical trial is a delaying tactic and de facto prevents access to this life-saving drug for patients who need it.
“Interestingly, the only other countries I’m aware of who developed a clinical trial approach for expanded access as opposed to compassionate use are the Russian Federation and Lithuania,” said Van Cutsem.
MCC spokesman Fidel Hadebe confirmed that the council has declined a request by MSF for patients to access bedaquiline.
He said it was resolved at an MCC meeting in May that patients receiving bedaquiline as part of their treatment, in accordance with prior MCC approval, be allowed to complete their therapy.
“It was also resolved that current data available to the MCC remains Phase II in nature which is insufficient to allow for approval for use outside of a well-designed clinical trial,” said Hadebe.
The World Health Organisation is supporting compassionate access and has urged countries to bring their regulations in step in order to facilitate compassionate access, especially for DR TB patients.
Dr Helen Cox, epidemiologist at MSF SA, said they were yet to be given an explanation about why the MCC reversed an initial decision to grant compassionate access to bedaquiline.
Several attempts to get the MCC to explain the reversal were unsuccessful.
In another development, the Health Department is also accused of closing an avenue that could allow use – expanded access.
Expanded access refers to when manufacturers make Investigational New Drugs available to treat patients who have a serious condition and who further cannot participate in a controlled clinical trial.
MSF and other role-players have worked closely with the Health Department to develop an expanded access proposal that had to be submitted to the MCC.
But the proposal has become stuck in the Health Department for no apparent reason.