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How will new most cancers medicines be authorized sooner or later? – Most cancers Analysis UK

Thanks to research and innovation, our knowledge of cancer is rapidly evolving, as are the drugs used to treat it.

Even in the past decade, many more cancer drugs have become available than in the past few decades. They are also becoming more and more advanced and expensive. And they are assessed earlier, sometimes before their overall ability to extend or improve patients’ lives is fully determined.

While this has resulted in more cancer treatment options, it has also increased uncertainty about the clinical benefits and cost-effectiveness of drugs. But new approaches to using real world evidence in the NHS can play a role.

What’s the value for money?

In the UK, the Medicines and Health Products Regulator (MHRA) licenses new medicines. The National Institute for Health and Care Excellence (NICE) is examining whether NHS patients in England should be offered drugs approved. Based on estimates of the drug’s cost-effectiveness, NICE will determine whether it is good value for money at the price negotiated between the NHS and the manufacturer. This is not an easy task, especially considering how cancer drugs are evolving.

First, cancer drugs are becoming more complex, sometimes with high up-front costs, but with benefits that can only be properly observed over the long term, such as with CAR-T-cell therapies.

This means that the overall picture of a drug’s cost-effectiveness is not always clear at the time of evaluation.

Second, cancer drugs are becoming more expensive. In 2020, oncology accounted for 15% of total drug spending in 11 major countries, up from 3% in 1995. And while the NHS and the pharmaceutical industry have agreed on measures to control the NHS’s drug spending, the UK pays relatively low prices compared to In in other countries, rising prices may prove unsustainable and make long-term cost-effectiveness estimates difficult.

Third, cancer drugs are rated earlier, in part because some promising cancer drugs are fast-moving through government approval.

This is certainly positive, but it has raised questions about drug effectiveness in later studies or in real-world patients versus clinical studies – particularly in the US, where the Food and Drug Administration (FDA) is a frequent occurrence uses quick approvals.

And research has found that in recent years, more drug filings with US and EU regulators are using non-randomized study designs. While there can be many good reasons why randomized controlled trials may not be feasible for some drugs, the lack of randomization can increase uncertainty.

So how do the NHS and NICE reconcile the need to bring promising new drugs quickly to patients with the confidence that they will be cost-effective?

Real-world evidence has proven successful.

The Reformed Cancer Drugs Fund (CDF) is temporarily promoting certain promising cancer drugs to ensure they are available more quickly to patients in the NHS in England. During this period, the manufacturer is expected to address the initial uncertainty by either conducting further clinical studies or collecting real world data on the drug’s effectiveness in the NHS. This data then feeds into a new assessment of the drug’s cost-effectiveness and sometimes into new price negotiations between the NHS and the manufacturer.

But the process of regulatory approval and cost-benefit assessment continues to evolve.

UK Post-Brexit Initiatives

Post Brexit, the UK government is exploring new ways to get cancer drugs to patients faster while removing some of the inherent uncertainties about cost-effectiveness.

Post-Brexit regulatory reform is part of the UK government’s agenda to make the UK a life sciences superpower and UK regulator MHRA is taking steps to work more closely with the FDA and speed up the regulatory process.

But will this lead to more uncertainty about the effectiveness of drugs after approval, as we have seen in the US? It’s too early to tell.

For example, the new Innovative Licensing and Access Pathway (ILAP) aims to reduce time-to-market and make it easier for patients to access innovative drugs by involving developers in the preclinical study phase and improving the overall assessment path.

The MHRA has also joined Project Orbis, an international program coordinated by the US FDA to review and approve promising cancer drugs.

If these trends and initiatives continue to facilitate earlier assessment, new and innovative mindsets may be required.

Link prices to treatment success

At Cancer Research UK, we believe that flexible drug pricing mechanisms can help alleviate some of the growing uncertainties surrounding the cost effectiveness of cancer drugs.

In collaboration with the Greater Manchester Health and Social Care Partnership, we are investigating whether the price the NHS pays for a drug depends on how well individual patients respond to the treatment – so-called outcome-based payments (OBP).

And the potential benefits are great. This approach can improve and speed patient access to some new cancer drugs, and ensure that the NHS only pays for results that are actually achieved for individual patients. The NHS is currently negotiating a price with the drug manufacturer that will be set regardless of patient outcomes.

And instead of funding a drug as more data is collected to produce a new cost-benefit assessment and fixed price – as the CDF does – OBP would dynamically adjust the price of a drug based on how good it is for the patient individual patient works.

So far, through our research, we’ve found that cancer treatment patients value four core outcomes: survival, disease progression, long-term side effects, and return to normal activities.

But OBP poses a number of challenges.

To collect data on these core results, hospitals need improvements to the data infrastructure and staff need more time and capacity. All of this needs to be considered prior to implementation in the NHS.

While OBP is by no means a substitute for NICE’s rigorous regulatory approval process and cost-benefit assessment, it can be useful in situations where data from completed clinical trials or the CDF are unlikely to resolve the uncertainty.

At Cancer Research UK, we hope to further develop our understanding of OBP and its implications. We also encourage the pharmaceutical industry, the NHS, and other decision-makers to continue to review this approach for the potential benefit of cancer patients.

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