Experts raise questions about current standards of drug regulation


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More and more drugs are approved on the basis of indirect (“surrogate”) means that do not always reliably predict the most important outcomes for the patient, such as longer life and better mood.

of BMJ Today, experts say that surrogate endpoints do not guarantee clinical benefit, Clinical trial.. Among the linked features, journalists Jeanne Lenzer and Shannon Brownlee Drug approval It does not benefit patients or public wallets.

By using surrogate endpoints to measure whether a new drug works, you can reduce the duration, cost, and complexity of clinical trials prior to regulatory assessment and accelerate them faster. Patient access In particular, new treatments for chronic diseases will be explained by Dalia Dawoud of the National Institute for Health and Care Technology (NICE) and his colleagues.

However, the use of such endpoints can have negative implications, such as making decisions more difficult for healthcare assessment agencies and complicating treatment decisions for patients and clinicians.

However, over the last three decades, the percentage of clinical studies measuring the efficacy of new drugs using only surrogate endpoints has increased from less than 50% in the mid-1990s to about 60% in 2015-17. Points out. In some therapeutic areas, such as cancer, surrogate endpoints account for nearly 80% of all clinical trials that support regulatory approval.

They called for more selective use of surrogate endpoints in assessing new drugs and used them. Chronic diseaseTrials with unattainable follow-up are required, especially when collecting data on patient-related clinical outcomes.

Greater involvement of the patient (and the organization representing the patient) in the regulatory and health technology assessment process is also essential to ensure that the conditions for accepting proxy endpoints for decision making are properly met. They say.

So do regulators need to approve medicines based on surrogate endpoints?

Jeanne Lenzer and Shannon Brownlee are considering a rapid approval process by the US Food and Drug Administration (FDA) to allow drugs to market before they prove effective.

Many of these approvals are based on surrogate endpoints, with 73% (43/59) of FDA-approved drugs receiving rapid approval in 2018.

As part of the accelerated approval process, manufacturers need to conduct accelerated approval trials to confirm their clinical benefit. If these trials do not work, you can revoke the drug’s approval. However, Lenzer and Brownlee said post-approval trials could be postponed for years, and the FDA does not hold companies accountable if they fail to prove such benefits. I am.

They point out that many patients are willing to take unproven drugs because of the belief that the FDA’s approval process guarantees the efficacy and safety of the drug. Doctors are prepared to prescribe based on similar beliefs.

However, by allowing drugs to be marketed on the basis of agents only, “the pharmaceutical industry and the FDA have the burden of proof on the shoulders of the public, along with the physical harm and financial costs of clinical trials. Claims to be mitigated. All). “

Although measures can be taken to mitigate the risks of using surrogate endpoints, some experts argue that the use of surrogate endpoints should be almost completely restricted to Phase II studies. (The test hypotheses and the benefits to harm are sufficient to justify a phase III study).

Others disagree, saying that it is unethical to keep patients dead while waiting for new treatment without using surrogate endpoints.

Meanwhile, the industry supports the use of surrogate endpoints, claiming that it would be too expensive to return to approval based on clinically significant endpoints. However, Jerome Hoffman, an emeritus professor at the UCLA Medical Center, said, “The final economic cost of approving and using harmful drugs is actually much higher than the cost of initially demanding better research. “.

The surrogate endpoint is drag It is more effective than the benefit of the patient and is rarely discussed in the patient’s circle, Roger Wilson wrote in a linked opinion. As a sarcoma patient, he experienced “misplaced enthusiasm” for treatments that were tried on surrogate endpoints and approved based on secondary endpoints. If subsequent studies did not confirm the results, it was withdrawn.

“We need to ensure a balance between two different perspectives: the medical story and the patient’s story,” he says. Therefore, he argues that surrogate endpoints need complementary patient reporting results (PRO). This is a way to help build a complete picture of the condition from the patient’s perspective.

He writes that surrogate endpoints remain here, but patients want PRO to complement them as co-primary or sole primary endpoints. “The merit is Patience Physicians who guide us in making treatment decisions while better understanding the outcome of the trial will have less uncertainty and stronger evidence to use. ”

Drugs that have received rapid approval in Europe may not provide the intended clinical benefit

For more information:
Analysis: Raising criteria for using surrogate endpoints in drug regulation and health technology assessment, BMJ (2021). DOI: 10.1136 / bmj.n2191

Quote: Experts, current standards for drug regulation acquired on September 15, 2021 from https: // (September 2021) I have a question about (15th).

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