Rarer Cancers Forum: new report reveals weaknesses in system to approve off-label treatment funding requests for patients with rare cancers

A new report published today by the Rarer Cancers Forum reveals huge variations in the way in which local NHS organisations process applications for using treatments for patients with rare cancer outside their licensed indication (off-label treatments).

Off-label treatments are an issue for people with rare cancers because there are usually very few – if any – licensed treatments for rare cancers. Instead, patients are prescribed treatments by their doctors which are licensed in more common cancers, but where the cancer which they have has a similar underlying disease process. The Rarer Cancers Forum has coined the phrase ‘near-label treatments’ to describe treatments which fall into this category.  

The issue is challenging for patients because – since the treatments they need are not licensed for the use for which they are needed – no mandatory national guidance can be issued. This means that patients are instead forced to apply for the treatments they need to their local NHS organisations.

The report, Off limits – an investigation into NHS organisations’ policies and processes for determining requests for the use of off-label treatments for people with cancer, is the first analysis of how this system impacts on patients with rare cancers. It finds that:

· The attitudes of NHS hospitals to near-label prescribing vary widely. Some trusts appear to actively discourage the practice whilst others acknowledge its necessity in high-quality patient care

· Over the last three years, over 3,000 patients have been forced to apply for funding for off-label treatments through their doctor to their local Primary Care Trust, and over 1,000 have had their requests rejected

· Patients with rarer cancers in the UK are less likely to receive off-label treatments if they need them than patients in France and Germany

The report makes 25 recommendations to improve the system, including that:

· Near-label cancer treatments used rarely should be funded at the national level

· Mandatory guidance should be issued to the NHS on the near-label treatments used most frequently

· The pharmaceutical industry should contribute to the costs of running the new system, provided that patients with rarer cancers benefit from improved access to medicines

Commenting on the report, Stella Pendleton, Executive Director of the Rarer Cancers Forum said:

“If a doctor thinks that a patient with a rare cancer should be given a drug, then the NHS should fund it. The NHS is forcing desperate patients into the cruel situation where the chances of their being given the treatment they need depend on where they live.

“No patient should be denied a treatment recommended by a doctor simply because the cancer it treats is too rare for the medicine to be licensed. We need these obstacles removed.

“Drugs companies, politicians and the NHS have a responsibility to patients to fix this system.”

Notes

The Rarer Cancers Forum’s public policy programme receives financial support from members of its healthcare industry reference group. The members of the group are Amgen Ltd, AstraZeneca, Bayer, Bristol-Myers Squibb Pharmaceuticals Ltd, Celgene Ltd, Merck Sharp and Dohme Ltd, Novartis Oncology, a division of Novartis Pharmaceuticals Ltd, PharmaMar, Pfizer Ltd, Roche Products Ltd and Wyeth Pharmaceuticals. The research contributing to this report was resourced from the Rarer Cancers Forum’s core funds as well as Charlotte and Emma Buckland's Reading Half Marathon sponsorship gift to the charity.

Editorial control rests with the Rarer Cancers Forum alone.

For media enquiries, please call Bill Morgan on 0777 965 5912. 

The report makes 25 recommendations to improve the current system, listed below:

1) All NHS bodies should be reminded of their responsibilities to respond in a timely and helpful manner to requests made under the Freedom of Information Act 2000. The 18 Primary Care Trusts (PCTs) which have failed to respond to our Freedom of Information requests on two consecutive occasions should in particular examine their processes for responding to requests under the Act.

2) As part of their obligation to meet competencies 2 and 3 of PCTs’ ‘World Class Commissioning’ assurance, PCTs should audit their performance in responding to Freedom of Information requests.

3) All NHS provider trusts should develop a policy or protocol for off-label prescribing as a matter of good practice. Progress in meeting this recommendation should be monitored by Strategic Health Authority (SHA) medical directors, and they should report on progress made to the National Quality Board.

4) NHS trusts should take urgent steps to implement electronic prescribing systems to improve their ability to audit prescribing patterns. Progress in implementing this should be recorded in the ‘Quality Accounts’ NHS trusts are now obliged to publish.

5) The National Chemotherapy Advisory Group should design the forthcoming chemotherapy dataset in order to ensure that off-label prescribing is clearly recorded.

6) The Department of Health should reiterate to PCTs the specific circumstances in which treatment requests should be treated as exceptional cases. This should make clear that funding requests for off-label treatments for use in a patient with a rare cancer are not to be treated as exceptional. Adherence by PCTs to the National Prescribing Centre’s guidance in this respect should also be inspected through the World Class Commissioning assurance process.

7) All PCTs should be encouraged to follow the welcome approach taken by some PCTs to date and develop clear processes for processing decisions on the use of off-label medicines.

8) The Care Quality Commission should be empowered to undertake spot checks of PCTs’ use of exceptional case processes, to ensure they correspond with the National Prescribing Centre’s guidance.

9) The Secretary of State for Health should issue immediate instructions to those PCTs which have a blanket policy of refusing to fund off-label treatments, instructing them to lift their bans on the funding of off-label medicines at once.

10) The Secretary of State should conduct urgent inquiries into how the PCTs which refuse to fund treatments off-label came to develop such arbitrary policies on the funding of cancer treatment. In the event that these inquiries establish that, as a result of such policies, patients with cancer were denied clinically-effective treatment, both the senior management of those PCTs and their boards should be held accountable.

11) All PCTs should adhere to the National Prescribing Centre’s guidelines, and record information as to whether the use of a treatment requested is on-label or off-label.

12) The Department of Health should conduct an investigation into the variation in the number of off-label treatment requests received by PCTs, to ascertain whether this variation adversely impacts on access to such treatments for NHS patients between areas of the country. This investigation should make recommendations to ensure greater consistency in approach in the future.

13) The Department of Health should examine the ‘service development plan’ approach to routinely funding off-label treatments cited in the response from Shropshire County PCT. If proved to be effective, such an approach could be implemented across the country.

14) The Department of Health should examine the causes of the variation between PCTs in the proportion of requests they approve and reject. Potential hypotheses to test on the causes of the variation revealed above might include:

PCTs with high rejection rates might routinely fund a large number of off-label treatments which have a volume of supporting evidence, meaning that the requests received are more likely to be those which lack evidence and are therefore rejected.

PCTs with high rejection rates might be controlling their budgets by rationing patients’ access to off-label treatments, using the absence of a licensed indication as an excuse to control their costs.

PCTs with low rejection rates might be those which demand that all off-label treatments – whether they are high or low cost – are applied for, and which approve higher numbers of treatments simply on account that many off-label requests they receive are older and have a minimal impact on their budgets.

15) The NHS Chief Executive should urgently publish an update on the progress made by SHAs in acting on his instruction to review, by April 2009, the way in which PCTs are collaborating to support effective decision-making on new drugs.

16) In implementing recommendation 13 of the Department of Health’s report Improving access to medicines for NHS patients, the Department of Health should prioritise the production of information for patients on the use of off-label treatments. This work should involve cancer charities and the outputs should be incorporated into the national cancer information pathway and information prescriptions.

17) In developing NHS Evidence, the National Institute for Health and Clinical Excellence (NICE) should consider how best commissioners can be made aware of the role that professional guidelines can play in supporting their decision-making process on off-label treatments.

18) The Department of Health, Medicines and Healthcare products Regulatory Agency (MHRA) and the Association of the British Pharmaceutical Industry (ABPI) should work together to produce guidance clarifying the circumstances in which pharmaceutical companies can provide information on the off-label usage of their products to clinicians, charities and policymakers. This should reaffirm that the promotion of medicines off-label is not acceptable, but make clear that there is a distinction between marketing an off-label treatment, and responding to legitimate requests for information about its use.

19) In taking forward the recommendation that further work should be undertaken to investigate the extent and causes of international variations in drug usage, the Department of Health should explore variations in the usage of off-label cancer treatments.

20) Treatments defined as ‘near-label’ should be reimbursed from a national fund and be subject to a comprehensive national audit.

21) An ‘evidence escalator’ should be established, ensuring that professional guidelines and ultimately a health technology assessment are available for those treatments which are used most frequently off-label.

22) Strict eligibility criteria should be established for reimbursement through the national fund.

23) The precedent set by the Office of Life Sciences’ recommendation earlier this year for an ‘innovation pass’ should be replicated in establishing the national fund.

24) The pharmaceutical industry should contribute to the costs inherent in running the national audit and in the commissioning of professional guidelines, provided that patients with rarer cancers benefit from improved access to medicines.

25) The MHRA should work with the European Medicines Agency (EMEA) to explore the feasibility of some form of ‘third party licensing’ of off-label treatments.