Accessing Non-NICE-Approved Treatments: What You Need to Know

For patients living with EGFR+ lung cancer and navigating limited treatment options, we know (from first hand experience!) that it can be incredibly frustrating to hear about promising new therapies that aren't yet approved for NHS use through NICE (the National Institute for Health and Care Excellence). However, there are ways to potentially access these medicines—though success is never guaranteed, they’re certainly worth exploring if you're running out of options.

One potential route is through ‘compassionate use programs’, often governed by the Medicines and Healthcare products Regulatory Agency (MHRA). These schemes depend on where the drugs are in the approvals process. In the UK, new drugs first undergo clinical trials to assess their safety and effectiveness. If the results are positive, the company applies to the MHRA for marketing authorisation, allowing the drug to be legally prescribed. After this, NICE evaluates the drug’s clinical benefit and cost-effectiveness to decide whether it should be routinely funded and made available on the NHS. ‘Compassionate use programmes’ allow patients to receive medicines that haven’t yet gone through full UK approval for NHS prescription, and typically fall into two main categories:

1. Early Access to Medicines Scheme (EAMS)

· This scheme is designed for medicines that do not yet have full marketing authorisation (i.e. they do not yet have MHRA approval).

· Focuses on drugs that show a clear potential to address unmet medical needs.

· Applications must show the treatment is likely to offer significant benefit over existing options.

2. Named Patient Supply / Managed Access

· Applies to medicines that do have marketing authorisation but are not yet approved for NHS use (i.e. not NICE approved).

· Enables your treating doctor to request access on a case-by-case basis for a specific patient.

· Often arranged in collaboration with the pharmaceutical company and subject to MHRA guidelines.

The scheme you need to use will depend on the drug you are trying to access, But either way, the process is similar.

How to Get Started

This process must be initiated by your oncologist or treating physician. They will:

· Contact the manufacturer directly to inquire whether the drug is available one of the above schemes.

· Submit a request on your behalf, making the case for clinical need.

· Ensure all documentation aligns with MHRA guidance.

If the manufacturer agrees, they’ll typically coordinate delivery of the medication to your NHS Trust. In many cases, the company provides the drug free of charge. However, if not, additional approval will be needed from the NHS for funding support.

Things to Keep in Mind

· There is no guarantee the request will be accepted—each case is considered individually.

· The process can take time, so it’s best to start conversations with your medical team early.

· Your oncologist’s support is critical. If they’re unfamiliar with this route, you may want to share this information with them or ask them to consult with a colleague or the hospital’s pharmacy team.

While these pathways may sound daunting, they can offer us some hope while we campaign for more treatments to become available on the NHS. If you believe this may be right for you, speak to your care team and explore the possibilities—because sometimes, persistence can open the right doors.

Professor Gini Harrison, EGFR+ UK Research Trustee