Progress with caution. Clinical trial waiver, a step to improve patient access, in need of guardrails bl-premium-article-image

PT Jyothi Datta Updated - August 18, 2024 at 07:04 PM.

Experts call for ensuring patient safety while expanding access to new drugs

The affordability question: New policy may speed up access but must address high costs and local testing concerns | Photo Credit: istock.com

Last year, patient families from India took part in an international “right to breathe” campaign seeking access to cystic fibrosis (CF) therapy Trikafta, from the American biotech company Vertex Pharmaceuticals.

Cystic Fibrosis causes sticky mucus to build up in the lungs and digestive system, and the undiagnosed often die in their infancy, patient families said, in their letter to the Government. Besides India, campaigns were mounted from South Africa, Ukraine and Brazil, coordinated by a United Kingdom-based campaigner for patient rights, along with other international organisations.

The experience, though, repeats itself with different illnesses — where patients seek access to newer therapies that are still to come to India.

Landmark waiver

Earlier this month, the Centre took a step to address such situations, by waiving clinical trials in five categories, if the drugs had been approved in stringent regulatory markets including the US, UK, Japan, Australia, Canada and the European Union.

The waiver covered drugs and vaccines falling into categories including orphan drugs for rare diseases, gene and cellular therapy products, new drugs used in pandemic situations, new drugs used for special defence purposes and new drugs having significant therapeutic advances over the current standard care. The waiver was facilitated under Rule 101 of the New Drugs and Clinical Trial Rules, 2019.

The move was welcomed in sections of civil society as being in the interest of patients. However, some industry-watchers have expressed caution, concerned about introducing a therapy without local trials.

There are drugs approved by stringent authorities sitting on the periphery, while patients cannot access or afford it, says Chetali Rao, legal and policy advisor with Third World Network.

Earlier, drugs made it to India only if the country was part of the multinational company’s global trials. The waiver decision takes it a step ahead by making these drugs available to the local population if countries with stringent authorities approve it, she explains. However, she adds, this may not do much to reduce the high prices (running into lakhs of rupees) of these drugs. “Affordability is still an issue,” she says, noting that some of these new therapies are not even registered in India.

Drug companies, especially multi-nationals, point to their patient assistance programmes as avenues to improve patient access. But Rao says they come with conditions that makes it difficult for all patients who may need the medicine.

Besides, in the absence of patient registries, there is no clear picture on the number of patients who actually need the therapy. Only local production by indigenous companies will help to make these drugs more accessible, she says.

Call to expand

Following the Government’s decision, the Organisation of Pharmaceutical Producers of India (OPPI) said, the move was progressive and would benefit domestic and foreign drug manufacturers “by expediting the approval process and facilitating faster access to essential medications for Indian patients.”

Calling it a “commendable beginning”, the OPPI called for the waivers to be extended to a broader range of therapeutic categories, to enhance access to cutting-edge treatments. “We urge the Government of India to consider additional therapeutic areas where similar waivers could significantly impact patient access. Moreover, it is pertinent to understand how the criterion for ‘new drugs having significant therapeutic advance over the current standard care’ is defined and implemented. This could set a precedent for recognising and adopting breakthrough therapies that offer superior clinical benefits,” the platform, largely for multinational companies, said.

Patient safety

A report by the Global Trade Research Initiative, however, raised patient safety concerns, if there was a waiver. “By overlooking India’s unique genetic diversity, the waiver could lead to unexpected safety and effectiveness issues,” says the report.

Ajay Srivastava, former Indian Trade Service Officer and founder of GTRI, further points to the lack of reciprocity in the move, besides the high costs Indian companies incur in undertaking trials abroad. Also, insisting on post marketing surveillance after a drug is launched, will result in mere data collection, he says, without a local trial.

While it’s true that patients need quicker access to break-through drugs, S Srinivasan of LOCOST (a producer of less-expensive drugs) and representative of Aidan (All India Drug Action Network), says, it should not involve dilution of governance. He calls for “bridging trials” to evaluate therapies in a local population, adding that the option of waivers can be adopted in special cases. The process to make new drugs available to patients, he says, should be both simple and safe.

Published on August 18, 2024 10:28

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