Off-label rules for cancer need to be clarified
pharmafile | August 18, 2009 | News story | Research and Development, Sales and Marketing |Â Â Cancer, NHS, off labelÂ
A new report is pressing for the off label prescribing of drugs to be made clearer and more fair for patients with rarer cancers in the UK.
The Rarer Cancers Forum says its research shows patients with rarer cancers often struggle to gain access to treatments, and that the current system has numerous weaknesses.
Many of the latest cancer drugs launched in the last few years are likely to benefit a wide range of cancers, but they need a specific licence for each individual cancer type, a process which can take years, or may never be commercially viable.
Oncologists and their patients are often willing to try using unlicensed medicines, but the interpretation of NHS rules about off-label prescribing varies widely.
The Rarer Cancers Forum (RCF) says this variation between hospitals to so-called ‘near-label’ prescribing means some trusts appear to actively discourage the practice, while other acknowledge its place in high-quality patient care.
The charity says the last three years has seen over 3,000 patients made to apply for funding for off-label treatments through their doctor to their local PCT, with around one in three having their requests rejected.
It says that patients with rarer cancers in the UK are less likely to receive off-label treatments if they need them, compared to patients in France and Germany.
The report makes 25 recommendations to improve the system, including:
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.”
She adds: “Drugs companies, politicians and the NHS have a responsibility to patients to fix this system.”
Another key change proposed would be for all NHS provider trusts to develop a policy or protocol for off-label prescribing as a matter of good practice. The RCF says Strategic Health Authority medical directors should monitor this progress, and should provide reports to the National Quality Board.
A further recommendation is for the Department of Health, the MHRA and the ABPI to produce new guidance to clarify when pharma companies can provide information on the off-label use of products to clinicians, charities and policymakers.
This is a key problem, as it is strictly prohibited under the ABPI Code of Practice for companies to promote medicines outside their licence. The charity says new rules would reaffirm this existing rule, but would make a distinction between marketing an off-label treatment, and responding to legitimate requests for information about its use.
Funding
The Rarer Cancers Forum has made clear in publishing its Off Limits report that it does take funding from pharmaceutical companies, but stressed that it has sole editorial control of its publications.
The charity receives funding from its healthcare industry reference group, which is made up of Amgen, AstraZeneca, Bayer, Bristol-Myers Squibb, Celgene, Merck Sharp & Dohme, Novartis, PharmaMar, Pfizer, Roche and Wyeth.
It says the research contributing to this report was resourced from the Rarer Cancers Forums core funds as well as sponsorship gift to the charity raised by Charlotte and Emma Buckland in running the Reading half marathon.
The Report’s 25 recommendations
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 Centres 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 Centres 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 Centres 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.
Rarer Cancers Forum – Off limits report on off-label prescribing
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