Ray of hope for terminally-ill patients as new norms may allow use of experimental drugs

Bringing a ray of hope to terminally ill patients in India who have exhausted all available treatment options, experts are deliberating allowing them to try experimental, under-development drugs still under development, two people aware of the matter said.

Currently, patients can use only those drugs which are approved by the drug regulator after it has passed clinical trials and found safe and efficacious, a process that takes several years.

However, a new policy in the works will allow them to access pre-approval drugs in cases of serious and life-threatening diseases on compassionate grounds, without clearing clinical trials.

Experts from the All India Institute of Medical Sciences (AIIMS), Indian Council of Medical Research (ICMR), Maulana Azad Medical College, Drug Controller General of India and representatives of pharmaceutical companies are deliberating on a policy for investigational drugs, the people cited above said, adding a draft policy is expected soon.

“The compassionate use programme is about providing pre-approval access to an investigational drug for patients suffering from rare diseases, serious and life-threatening illnesses and conditions, who have exhausted all approved medicines and are not eligible to participate in the ongoing clinical trials in investigational drugs relevant to such diseases and conditions," said Prof. YK Gupta, principal advisor, Translational Health Science And Technology Institute and president of AIIMS, Bhopal.

There will be no provision for compensation for the failure of such drugs. Experts have also suggested that neither patients nor manufacturers should be liable to pay the cost of treatment; rather, some government funds should be earmarked for providing medicines free of cost.

"The pricing of compassionate use drugs should not pose a financial burden on needy patients," added Prof. Gupta.

The policy would identify medicines, biological devices, use of stem cells, gene therapy and immunotherapy that can be covered under the compassionate use programme in brain-dead patients.

"Participation in clinical trials is challenging as not every patient meets the inclusion criteria," said one of the two persons cited earlier.

The right to access investigational drugs is a reasonable approach, said Archana Panda, co-founder, Cure SMA foundation of India, an NGO that represents 300 families in India with people with spinal muscular atrophy (SMA), a life-threatening neuro-muscular genetic disorder.

"The policy should have checks and balances in place so that the use remains ethical," she said.

The new policy is likely to be on the lines of the one adopted in the US. In 2018, President Donald Trump signed a bill giving terminally ill patients the right to try experimental treatments not approved by the government, triggering a storm with advocacy groups including the American Cancer Society opposing the Bill.

In the US, patients seeking access to investigational drugs now need only the approval of their physician and the drug manufacturer. The new bill also protects doctors and companies from the legal risks allowing unapproved treatments unless they intentionally harm patients.

"The expanded access programme provides a pathway for patient to obtain investigational new drugs, biological or devices that would otherwise be unavailable outside of a clinical trial setting to address a serious disease or condition for which there are no satisfactory alternatives," said the second person.

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