INTRODUCTION

  1.  The Association of the British Pharmaceutical Industry represents more than 70 companies in the United Kingdom producing prescription medicines. Its member companies are involved in all aspects of research, development and manufacture, supplying more than 80% of the medicines prescribed through the National Health Service. The ABPI also represents companies engaged solely in the research and/or development of medicines for human use. In addition, there is general affiliate membership for all other organisations with an interest in the pharmaceutical industry in the United Kingdom.

  2.  The ABPI welcomes this inquiry into top-up fees.

OVERVIEW

  3.  The ABPI submitted responses to both Mike Richards and the Conservative party on 21 August and 4 September respectively.

  4.  The review of the "top-up fees" carried out by Mike Richards was a sensitive task and the document "Improving access to medicines for NHS patients" is well balanced with sensible recommendations. In our view, Mike Richards understood well the sensitivities associated with top-up payments. We agree that patients confronting the difficult choice of having to pay for innovative medicines that are not funded by the NHS should be a very small group. Indeed, Lord Darzi's review and the draft Constitution both stipulate that patients should be entitled to NICE-approved medicines. Patients will benefit if the NHS moves to a more innovation-embracing culture, providing them with access to modern medicines that can improve quality and quantity of life and enable many to continue to work thereby helping the economy.

BACKGROUND

  5.  UK patients do not enjoy the full benefit of the innovations coming from the R&D investments made by pharmaceutical companies. Healthcare spending on medicines has been low and the uptake of innovative treatment slow compared to other European countries. The discontinuation of the 2005 PPRS has destabilized the market and further price cuts have undermined confidence in the UK within the industry. The ABPI could see no reasons for these further cuts given that UK medicines prices are lower than in other comparable European countries. There should not be any reason, therefore, why patients should be refused treatments on the ground of cost if their clinicians deem such treatments appropriate.

  6.  The NHS tends to look at the budget impact of a medicine rather than the value it brings to the NHS and to society. A broader definition of value should be adopted both in Health Technology Assessment processes and by local NHS organisations when decisions are being made on whether patients should receive treatment.

OUR REVIEW OF MIKE RICHARDS' RECOMMENDATIONS

Recommendation 1: The measures the government is already taking to improve the timeliness of the NICE decision making process are extremely welcome and should be strongly supported. The Department of Health and NICE should publish an update on the timelines for delivering these important commitments.

  7.  The ABPI agrees with this recommendation. It is important to remember, however, that at the time of launch clinical and economic data derived from use in a "normal" clinical setting are by definition limited and that such limitations should be recognised via a pragmatic approach to decision-making with decisions open to review once additional information becomes available.

Recommendation 2: The Department of Health should urgently consider how PCTs can be encouraged to work together to make proactive commissioning decisions. Consideration should be given to whether collaborative processes already developed, such as in the North East for cancer drugs, could be used as a model.

  8.  Whilst collaboration by PCTs with similar populations makes sense to avoid unnecessary bureaucracy and duplication of work, the processes they use to evaluate innovative medicines and make funding decisions for populations or individual patients should be transparent, robust and according to accepted best practice. Such collaborations should ensure that the companies that have developed the medicine are able to provide up-to-date information and critique factual information on their products.

Recommendation 3: The commitment made in the draft NHS Constitution to ensure transparency in PCT decision making, and the resulting work being undertaken by the Department of Health to support PCTs in delivering this, is extremely welcome. The government should set out as soon as possible more detailed plans for how it will achieve the commitment in the NHS Constitution, including the timescale for this work.

  9.  The Department should ensure that best practice principles and processes are adopted by local NHS organisations when making decisions on the funding of medicines; transparency on how those decisions have been made is essential for patients, clinicians and other stakeholders in order to limit unnecessary postcode prescribing and engender confidence in the system.

Recommendation 4: In developing collaborative arrangements for decision making, the government should also consider how PCTs can be better supported to make decisions on funding off-label drugs, whether as a matter of policy or on an exceptions basis.

  10.  Again, consistency in decision making will be welcomed.

Recommendation 5: The Department of Health should work:

—  with NICE to assess urgently what affordable measures could be taken to make available drugs used near the end of life that do not meet the cost-effectiveness criteria currently applied to all drugs; and

—  with the pharmaceutical industry in the context of the current Pharmaceutical Price Regulation Scheme (PPRS) negotiations to promote more flexible approaches to the pricing and availability of new drugs. This will require partnership working with the pharmaceutical industry and greater flexibility in approach from all parties.

  11.  The ABPI welcomes the consultation from NICE on the appraisal of end-of-life treatments. Our response is attached and brief commentary given below.

  12.  We also welcome the new flexible arrangements, agreed as part of the PPRS, which will enable the industry to re-evaluate the price of their medicines when new information or data become available.

Recommendation 6: The Department of Health should urgently undertake further work to investigate the extent and causes of international variations in drug usage.

  13.  The DH and ABPI are working together to make robust comparisons of uptake in the UK vs other countries; these are in the planning stage. Industry values this dialogue.

Recommendation 7: The Department of Health should clarify the policy on how the NHS should handle situations where a patient wishes to purchase additional treatment. The objective should be to ensure consistency in practice across the NHS.

  14.  Consistent adoption of DH policy will be essential to ensure equity of decision-making and confidence in the system of top-up payments. We indeed welcome the consultation issued by the Department of Health on their Guidance on NHS patients who wish to pay for additional private care. The ABPI will respond to this and gives a brief commentary below.

  15.  We believe clarity should be given by the DH on dispensing fees for these medicines; it is our understanding that currently dispensing fees for private prescriptions vary widely.

Recommendation 8: The Department of Health should make clear that no patient should lose their entitlement to NHS care they would have otherwise received, simply because they opt to purchase additional treatment for their condition.

  16.  The ABPI agrees with the decision not to withdraw patients' right to NHS treatment when purchasing additional treatment. If NHS funding is regrettably refused because of a negative NICE recommendation or a local PCT decision, patients should have the option to top up their NHS care by paying for the medicine—without losing the remainder of that NHS care. The clinician should additionally ensure that the top-up payment will not seriously damage the lifestyle of the patient and/or their family, for example through sale of the family home.

  17.  The reasons for any decision to refuse funding should be made clear to the patient and be subject to public scrutiny. Top-up payments for medicines should be a rare event and the industry does not anticipate that they would represent a major source of revenue.

Recommendation 9: The government should make clear that:

—  clinicians should exhaust all reasonable avenues for securing NHS funding before a patient considers whether to purchase additional drugs;

—  patients should be able to receive additional private drugs as long as these are delivered separately from the NHS elements of their care; and

—  providers should establish clear clinical governance arrangements to ensure that patients who do elect to purchase additional private treatment receive good continuity of care.

  18.  These recommendations represent a pragmatic and practical route to enabling patients to choose additional private care. The dialogue between the clinician and patient will be critical in establishing clearly to the patient what their options are. As said above, it is important that decisions not to fund a medicine are made under transparent and robust processes that follow best practice principles and operating procedures. For rare disease treatments, funding could possibly be derived from a central fund, using consistent criteria to avoid unfair decisions.

Recommendation 10: Strategic Health Authorities, working where appropriate through cancer networks, should ensure that local policies are developed to ensure that any revised guidance issued by the government is implemented properly. This might include using a designated hospital with private facilities for all patients wishing to purchase additional drugs, making use of homecare provision or designating an area of an NHS hospital for the delivery of privately funded treatments.

  19.  Government guidance will contribute towards a consistent approach across England. Consistent adoption of the guidance will be essential to ensure equity of decision-making and confidence in the system of top-up payments.

Recommendation 11: The Department of Health should take a lead on commissioning a national audit of demand for unfunded drugs and on the outcome of treatments, working closely with professional organisations and NHS managers.

  20.  As we are moving into unknown territory, data collection will be essential to evaluate the new policy and inform future decisions. It would be sensible to work with companies on data collection on treatment outcomes, to share conclusions and avoid unnecessary duplication.

Recommendation 12:

—  Doctors who are likely to have conversations with patients about treatments that are not routinely funded on the NHS should ensure that they have the necessary knowledge and skills to communicate complex information effectively and in a balanced way. This will help patients to make informed assessments about the balance of risk, cost and benefit involved in any potential treatment.

—  The Department of Health should commission a training programme for clinicians to enhance the quality of discussion about these difficult issues.

—  Relevant Royal Colleges should consider how assessment of communication skills could best be incorporated into recertification processes.

  21.  Providing information to patients is an extremely important task, the more so when patients are required to make important decisions about whether to pay for their treatment. Ideally, decisions should be made by the patient and doctor together and doctors need excellent communications skills to convey complex information at a very difficult time in the patient's life.

  22.  The pharmaceutical industry, as an important source of information on the medicines that it has discovered and developed, can help through the provision of factual, evidence-based information for patients. The ABPI welcomes the recent European Commission's proposals as part of the pharmaceutical package that industry should be allowed to provide limited information to the public on websites and in response to enquiries. Companies will also play their part in supporting health professionals to communicate effectively with their patients.

Recommendation 13: The Department of Health should consider how patients could best be given access to balanced written information on the benefits, toxicities and, where appropriate, costs of novel treatments, especially those given to patients near the end of life.

  23.  As said above, the pharmaceutical industry is an important source of information and would be happy to engage in discussions with the Department of Health to support implementation of this recommendation.

Recommendation 14: In responding to this Review, the government should confirm how situations where patients wish to purchase additional non-drug interventions should be handled.

NICE CONSULTATION ON APPRAISING END OF LIFE MEDICINES

  24.  The ABPI welcomes NICE's consultation on the appraisal of end of life medicines. The consultation is an important recognition that society places different values on different health interventions and whether patients should have access to treatment. The use by NICE of a one-size-fits-all application of a set cost per QALY threshold fails to reflect the complexity of evaluating the vast array of interventions that NICE has to assess, and whilst we understand that the NICE Social Value Judgements have some part in decision-making, we do not believe that factors other than the cost per QALY are sufficiently taken into account by the Appraisal Committee. The methods used to derive a cost per QALY are generally prejudicial to patients at the end of life, the elderly and those with long term conditions where small incremental improvements in treatment can make a big difference to daily life.

  25.  Overall comments are as follows:

  26.  It is not clear why the guidance to the Appraisal Committee is limited to end of life medicines only and not to other rare and debilitating conditions. This seems to discriminate unnecessarily against patients with life-threatening conditions for whom there is no alternative treatment.

  27.  The additional guidance focuses on extension to life only and appears to give no consideration to patient quality of life. This is unlikely to be consistent with the consideration taken by the patient and clinician in actual clinical practice of options and trade-offs for the patient.

  28.  Whilst we have no issue with data collection by NICE following appraisal, we would caution against overlap with existing studies and against under-estimation of costs where there is no existing disease registry. We believe that the focus of the data collection should include appropriate use of the medicine by the NHS as well as survival gains and that the manufacturer should be given the opportunity to comment on the protocol to ensure that their expertise relating to the product and therapy area is maximised.

  29.  It is not clear from the consultation document how NICE will report on how the additional guidance has been applied by the Appraisal Committee. Clarity in ACDs, FADs and final guidance will be essential to stakeholders' understanding of the decision making process.

EARLY THOUGHTS ON THE DH CONSULTATION "GUIDANCE ON NHS PATIENTS WHO WISH TO PAY ADDITIONAL PRIVATE CARE"

  30.  The DH guidance appears to be clear, useful and pragmatic. The challenge will be in local implementation.

  31.  The consultation makes the point that provision of information to patients on their options will be an extremely important aspect of making the new scheme work. As we say above, the pharmaceutical industry has an important role to play in the provision of information on its medicines.

  32.  The guidance states that "clinicians should exhaust all reasonable avenues for securing NHS funding before suggesting a patient's only option is to pay for care privately". In the case of end-of-life treatments, timely decisions are essential and delays should be avoided to enable patients for whom days count to receive urgent treatment, should it be funded by the NHS or paid for privately.

  33.  We believe additional guidance should be given to the NHS on the dispensing fees for these medicines, as we understand that these can vary widely.

December 2008