Compassionate Access with reference to Tuberculosis

India is battling multidrug-resistant TB, with about 2.5% of all new TB cases in the country being resistant to Rifampicin, or to both Rifampicin and isoniazid – the two most commonly used anti-TB drugs. At the end of 2015, India had 79,000 cases of drug-resistant TB, 11% more than in 2014.Bedaquiline drug can be an effective replacement for Rifampicin but this is accessible only through “compassionate use or compassionate access”.

MDR and XDR TB

There are two main types of drug resistant TB viz. MDR-TB and XDR-TB. MDR-TB is when the bacteria are resistant to Rifampicin and Isoniazid.  XDR-TB (extensively drug resistant TB) is when the bacteria are resistant to rifampicin and isoniazid and also at least one of the second line injectable TB drugs viz. amikacin, kanamycin or capreomycin.

Bedaquiline and Compassionate Use

Rifampicin was introduced in 1960s to treat TB. Since then, resistance to rifampicin has been increasingly reported in the world which is a major concern given that it is the most effective anti-TB drugs available today. Bedaquiline has been made mainly to treat TB patients with bacteria that are resistant to rifampicin, and thus suffer from multidrug-resistant tuberculosis

While bedaquiline has shown beneficial effect in studies including two Phase trials, Phase III trials have not been completed. However, given the serious threat posed by MDR-TB both to the individual patient and to the community, some regulatory authorities have used an accelerated procedure for the approval of bedaquiline under so called compassionate use in order to ensure that eligible patients may benefit from this new drug when used under defined conditions.

“Compassionate Use” or “Compassionate Access” refers to the treatment of life threatening disease using investigational drug when no other treatments are available. In other words it refers to drugs that are being scientifically tested but have not yet been approved by the United States Food and Drug Administration (FDA) are called investigational drugs. Under strict conditions, products in development can be made available to groups of patients who have a disease with no satisfactory therapies and who cannot enter clinical trials.

Compassionate use and Government’s of India conditional access programme:

Bedaquiline was available for compassionate use (CU) in Multiple Drug Resistant-TB patients in India from 2012. As part of a Compassionate Access Programme the pharmaceutical company Johnson who holds the patent for this drug, had donated 600 doses to USAID, which gave half of the medicines to the Indian government  but still enrollment rate for this drug is dismal because it is  strictly regulated by the government and it is confined to just six cities.

• One of the reasons for the controlled access to these new TB drugs in the government centres offering them is because of logistical issues.It is hard to monitor patiebt in far-flung and rural areas.
• The six centres providing bedaquiline under the Conditional Access Programme are the only ones equipped and trained to manage the programme so far.Hence people living in remote area are deprived of this drug Indiscriminate use of drugs.

Recently government announced that Patients with extensively drug-resistant tuberculosis (XDR TB) can separately import the potentially life-saving ‘bedaquiline’ if they don’t qualify for the drug under India’s conditional access programme with US drugmaker Johnson & Johnson.

Limitation of drugs under compassionate use

We note here that there may still be some uncertainties about the best way to give the medicine to patients, such as the exact dose to use, and the dose frequency, and the medicine’s safety profile (which side effects it can cause) is not yet fully established. However, there is a need to increase availability and access of drug to needy patient. Non–availability of TB drugs is detrimental to patients because it results in complication and proliferation of drug resistant strains of TB.