Balancing act

The new options in the UK of top-up payments and patient access schemes promise to improve access speed for patients in need of treatment. However, these schemes need to be managed carefully to ensure they don't become a burden to health systems, market access processes and healthcare players.

Defining the new schemes
Both top-up payments and payment access schemes were created to increase access to drugs for patients in need of specific treatments. They are part of a range of new measures that the Department of Health, NICE and the pharmaceutical industry are taking to improve drug availability for NHS patients.

The drugs that patients might seek to gain access to through the schemes fall into three main categories:
• Drugs on which NICE has yet to issue final guidance
• Drugs that NICE will not appraise or that a clinician wishes to use "off label"
• Drugs that NICE has declined to recommend for use in the NHS.

Top-up payments
A clinician may have reasons to prescribe a drug that has been declined funding by a Primary Care Trust (PCT). In this situation, patients may wish to purchase part or all of the recommended drug treatment themselves. There are a number of different terms for the mechanisms that allow patients to pay out of pocket. These include top-ups, co-payments, user charges and patient contributions.

Often, patients wishing to increase access to drugs in this manner are those nearing the end of their lives. The common perception is that the value society places on supporting patients in their last months or years is not sufficiently considered when assessing the cost-effectiveness of new drugs. There is a general view among stakeholders that drugs used to treat patients with life-threatening conditions should have a very high priority.

In addition, in a review by Professor Mike Richards CBE, released in November 2008, stakeholders called into question the way NHS organisations handle drug access for seriously ill patients. They expressed a strong desire for the NHS to continue providing comprehensive services, including offering a wide range of treatments similar to those available in other countries.

The review presented a broad spectrum of options for NHS patients wanting to purchase drug treatments themselves — from excluding them from the NHS entirely, to integrating them fully. Whichever option gets put into place — and whatever terminology is used to describe it — it is essential to have clarity on what it promises, to ensure the scheme can be implemented effectively when put into real-world practice.

Patient access schemes
Patient access schemes are intended to help NHS patients secure access to drugs that NICE might not consider cost-effective. Patient access schemes are proposed initially by a pharmaceutical company. The company then comes to an agreement with the Department of Health (with input from NICE) to improve the cost-effectiveness of a drug, so as to enable patients access to innovative medicines.

The latest Pharmaceutical Price Regulation Scheme agreement recognises several schemes that fall within two overall categories; financially-based and outcome-based. Financially-based schemes do not alter the list price of the drugs, but offer effective discounts or rebates that may be linked to criteria such as the number of patients treated, the response of patients to the treatment and the number of doses required, as examples. Companies also have the option to change the list price of the product.

Outcome-based schemes can be split into three sub-groups:
• Proven value, price increase: The company seeks agreement with NICE for a later increase in price, subject to a re-review of the drug in the light of additional evidence. Normally the company will be responsible for collecting the additional data.

• Expected value, rebate: The company seeks agreement with NICE to a price, subject to the collection of additional evidence. A rebate and a subsequent reduction in list price will result if the additional evidence does not support the current price. Normally the company will be responsible for collecting the additional evidence.

• Risk sharing: Outcomes are measured through patient reported outcomes or clinical outcome measures, and price adjustments and/or cash transfers made in one or both directions (between the company and the NHS) in light of the results. In other words, outcomes are assessed to ensure they are in line with the terms laid out in the scheme, and adjustments are made if results don't match the anticipated outcomes.

Both schemes should allow greater access to new medicines. In addition, they should help increase the speed of uptake of new drugs — an area often pointed out as an NHS failing. By definition, both schemes are value-based and will increase the transparency of outcomes. Their introduction, however, might be associated with a significant administrative burden to the health system, a burden that is complicated by the variety of funding mechanisms possible, each with potential implications for health service delivery.

The pros and cons
Patient access schemes and top-up payments offer strong potential benefits, but also raise some key concerns. One of the greatest opportunities offered is the fact that medicines will be made available in the global arena of linked market prices, but with localised cost-effectiveness targets. In addition, healthcare should improve as patients can be treated with the best medicines at the right time, and the system is value based. Outcomes of treatments will be in the public domain, and the uptake speed of new products should improve. Prescription drug sales should increase also, improving manufacturers' profitability, and there should be more opportunities for partnerships between industry and the NHS. All this will result in patients gaining more freedom and a stronger voice in their own treatment decisions.

Along with the benefits, however, comes a list of potential problems. The schemes may result in an administrative burden on the health system. Financial flows in the NHS are complex, and there is a need to reconcile the risk share schemes or the top-up schemes with the movement of funding between suppliers, providers, patients and commissioners. Schemes also require the transfer of individual patient data, raising confidentiality concerns, and auditing the schemes is a complicated process.

Extensive information
The schemes depend on patients providing top-up payments, with no guarantee that the treatment they are selecting will be effective. For patients to make informed decisions about the treatments they are willing to 'top-up', they need extensive information and education about their options. Patients' decisions on top-ups might be based on very subjective motivations, and it will be necessary that both schemes put in place agreements on how clinical criteria will be measured. As clinical expertise grows and patient response rates improve, the return to the NHS could decline.

Patient response rates will not be consistent across the NHS. A number of factors may influence responses, including patient differences in treatment resistance and compliance, variations in the quality of service delivery across providers and tertiary centres.

Finally, clinicians may treat more freely with medicines they perceive to be free. Implementing these schemes effectively requires a careful assessment of both benefits and risks. Successful schemes will strike the right balance, optimising the upside and minimising the chance of difficulties and complications.

Recommendations
There are several recommendations, both strategic and tactical, that will help all players make the most of the new schemes. While the application of these schemes is limited by the administrative burden on the NHS, however, industry and government can build on their experiences with the new, more flexible approaches to develop other ways of improving patient access to drugs.

The most important recommendations for helping flexible models succeed are:

1. Ensure timeliness and affordability
It is important that arrangements for the schemes do not jeopardise the timeliness of NICE appraisal guidance, which was designed to help the NHS provide cost-effective treatment to patients. NICE must assess urgently what affordable measures can be taken to make drugs available to patients nearing the end of their lives, even when those treatments do not meet the cost-effectiveness criteria used for other medicines.

2. Keep it simple and fast
Schemes should be patient-centred, with patient agreement, and based on easily-measured clinical outcomes. Information provided to patients to help them make top-up payment decisions should be easy to understand and enable them to make informed choices. Every effort should be made to speed up NICE decisions, so that all players have the opportunity to work together on patient access schemes or make drugs available through top-up payments.

3. Put one administrative model in place
To reduce administrative burdens, all schemes should comply with a single, preferred model for transfer payments. PCTs should be encouraged to work together on proactive commissioning decisions.

4. Make decision making transparent
NHS must live up to the commitment in its draft constitution to ensure transparency in PCT decision making.

5. Support open communications
Schemes should be jointly developed by the NHS, industry and patients, with patients given the information they need to make the best decisions.

6. Consider infrastructure costs
The costs required to administer the schemes must be factored in for all participants.

7.  Require financial clarity
Schemes must address the twin issues of patient outcomes and the implications for financial flows. Financial flows within Trusts must be considered and prices varied to accommodate differing needs.

Flexible models that improve access to drugs are critical to ensure all patients – including those nearing the ends of their lives – have the best choice of therapies. As stakeholders gain experience with these approaches and how to apply them, patients will benefit from increased drug availability, leading to better treatment.

The Author
Susanne Michel is global practice leader, market access, pricing and reimbursement at TNS Healthcare
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