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Fast-track approval processes are accelerating the path of new drugs to patients on both sides of the Atlantic, as regulators try to help industry cut the time and cost of developing medicines.
The US Food and Drug Administration (FDA) took the lead in 2012 with its “breakthrough therapy designation”, which allows products offering the biggest potential medical benefits to be reviewed at an accelerated pace. This could include drugs for previously untreatable rare diseases or new approaches to more common conditions that offer a substantive improvement over existing therapies.
The FDA’s counterpart across the Atlantic, the European Medicines Agency, has followed suit this year with a similar scheme called Prime, which aims to increase regulatory support for the most promising drugs and cut their assessment times by nearly a third, from 210 days to 150 days.
These, and a range of similar measures, are designed not only to bring important new treatments to patients faster but also to reward pharma companies with a quicker return on their investment when they produce a genuine advance in medical science.
These approaches appear to be working; new drug approvals by the FDA have risen to near-record levels in the past two years: of the 45 medicines approved by the agency in 2015, 60 per cent went through one of the agency’s fast-track processes.
This is good news for a pharma industry under pressure from investors and society to find ways of reducing the 10-15 years it typically takes to get a new drug to market and the resulting heavy costs and high prices.
As well as making faster decisions, regulators are allowing drugs to be filed for approval earlier if clinical data look promising. Instead of waiting for the results of large late-stage trials traditionally required to win authorisation, a growing range of options enable companies to start selling a product on a conditional basis, while continuing to gather evidence on safety and efficacy.
Emer Cooke, head of international affairs at the EMA, says the London-based agency studied the FDA’s breakthrough scheme closely while designing Prime. In other areas, such as regulation of a new category of copycat biological drugs called biosimilars, Europe has provided lessons for the FDA. “We learn from each other,” says Ms Cooke, noting that both agencies have full-time liaison officers posted at each other’s headquarters. “We’re not competitors, we’re collaborators.”
Critics query whether regulators have shifted too much focus towards speed at the expense of safety. Conditional approval can mean drugs reaching the market based on data from a few hundred patients rather than the thousands typically involved in late-stage trials. The numbers can be even smaller for drugs targeting rare or niche conditions, increasing the risk of safety problems going undetected.
Ilaria Passarani, head of food and health for BEUC, the European consumer organisation, says: “We support faster access to medicines, but faster doesn’t have to mean easier.”
Advocates for fast-track schemes say that, because they are only open to drugs offering a substantial advance over existing treatments, patients are usually happy to accept a level of risk. Meanwhile, advances in digital technology, remote monitoring and big data analytics are providing new ways to spot safety problems and measure efficacy once a product is on the market.
Industry believes the regulatory process can be streamlined further by forging more common standards and practices between the FDA and the EMA. Drug companies want this to be one goal of the proposed Transatlantic Trade and Investment Partnership (TTIP) between the US and the European Union.
There is broad political support for the idea of increasing co-operation and reducing duplication between the agencies. For example, joint recognition of clinical trials would remove the need for drugmakers to carry out separate studies to meet different requirements on opposite sides of the Atlantic.
“We support the idea of avoiding duplication in clinical trials because it is important to avoid exposing patients to unnecessary risks,” says Ms Passarani. “The more information shared between regulators, the better.”
In another area, however, consumer activists fear the watering down of European standards. The EMA has tougher requirements than the US on disclosure of clinical trial results to prevent data being manipulated in ways that could make drugs look more effective and safer than they really are. Some US drug companies have argued that the rules put valuable commercial secrets at risk and are pushing for safeguards in any trade deal.
“Europe is at the forefront of clinical trial transparency and we have fought hard for it,” says Ms Passarani. “We are worried that this could be undermined by TTIP.”
Ms Cooke says there are enough areas of consensus for the FDA and EMA to increase co-operation — while each keeping their independence. “There’s a lot of collaboration going on already but sometimes something like TTIP can give something an extra nudge,” she says. “We’re looking to see how we can do things smarter.”