Process and methods
4 Patient access schemes, commercial access agreements and flexible pricing
4.1 The Pharmaceutical Price Regulation Scheme (PPRS) 2014 allows companies who are members of the scheme to submit proposals for patient access schemes and flexible pricing proposals as part of an ongoing or published NICE technology appraisal.
4.2 In the context of the Cancer Drugs Fund, companies can also agree commercial access agreements with NHS England. Such arrangements will be considered in the NICE technology appraisal.
4.3 A patient access scheme is a scheme proposed by a company that is a member of the 2014 PPRS. Up to January 2018, these were approved by the Department of Health and Social Care, but from January 2018 onwards they are approved by NHS England. Patient access schemes allow patients to have a technology when NICE's assessment of value, on the current evidence base, is unlikely to support the list price.
4.4 Flexible pricing recognises that the initial launch price of a technology may not fully reflect its longer-term value to patients in the NHS. It therefore allows a company to propose an initial price for a technology that reflects value that can be demonstrated at launch, while retaining the freedom to apply to increase or decrease this original list price either as further evidence or as new indications emerge and change the effective value that the technology offers to NHS patients.
4.5 A commercial access agreement between a company and NHS England supports use of a technology for which at least 1 indication is currently, or has been, considered as part of the Cancer Drugs Fund.
4.6 NICE can only consider patient access scheme proposals, flexible pricing proposals and commercial access agreements after NHS England has formally approved them (see figure 6).
4.7 The Commercial and Managed Access Programme at NICE will provide companies with opportunities to engage in commercial and managed access conversations with both NICE and NHS England. The relevant stages for commercial dialogue are:
4.8 The 2014 PPRS identifies 2 types of patient access scheme (see chapter 5 of the 2014 PPRS for more details):
simple discount schemes and
4.9 The Patient Access Scheme Liaison Unit (PASLU) at NICE advises NHS England on the feasibility of implementing patient access scheme proposals. When assessing a patient access scheme proposal, the PASLU considers the key principles for implementing patient access schemes in England as outlined in the 2014 PPRS. The PASLU process is not part of the appraisal process. Changes could be made to a patient access scheme proposal after NHS England has referred it to NICE, however, these must be discussed and agreed with NHS England.
4.10 The appraisal committee considers the effect of a patient access scheme proposal on the clinical and cost effectiveness of the technology and clarifies relevant points with the company (see section 3.3). The ERG or the NICE team assesses the impact of the proposed scheme on clinical and cost effectiveness.
4.11 The process for reviewing the impact of a patient access scheme proposal on the cost effectiveness of a technology depends on when the proposal is submitted to NICE. When companies wish to propose a patient access scheme in the context of a NICE technology appraisal, they should follow these rules:
As a general rule, companies should include a patient access scheme when making their initial evidence submission to NICE. This means that any patient access scheme proposal should be sent to NHS England long before the evidence submission for the NICE appraisal. This allows sufficient time for the patient access scheme to be approved before the first appraisal committee meeting.
In exceptional circumstances, a simple discount patient access scheme may be accepted at other times in the NICE process. A simple discount scheme can be proposed:
in response to the technical engagement step
in response to the ACD
at the end of the appraisal process, once any appeals have been heard and NICE's final guidance has been issued to the NHS, in a rapid review of the guidance.
The appraisal process could accommodate approval of a complex patient access scheme, particularly when introduced in response to technical engagement or the ACD. It is the company's responsibility to ensure that NHS England has sufficient time to complete its consideration of the proposed patient access scheme in time for the appraisal committee meeting.
4.12 If the appraisal committee recommends a technology with an outcomes-based patient access scheme or commercial access agreement, it is essential that arrangements are in place to collect and analyse the relevant outcomes. If the actual outcomes differ significantly from those assumed during the original appraisal, NICE may decide to bring forward a review of the recommendations.
4.13 For fast track appraisals (this is an exception to the statement in section 4.11) a patient access scheme proposal must be included in the company evidence submission.
4.14 Any significant new proposals for, or structural changes to, a patient access scheme or commercial access agreement after release of the final appraisal document (FAD) will not be accepted, but minor changes to an agreed commercial arrangement, such as a change in the level of discount could be accepted. At this point an update to the guidance will only be considered in a rapid review of the guidance. See sections 4.21–4.25 for further details.
4.15 The appraisal committee can consider a patient access scheme or commercial access agreement proposal before formal approval from NHS England when the risk of non-approval is considered low (for example when the PASLU advice to NHS England supports the proposal). NICE must not release an ACD or FAD until approval of the patient access scheme is received from NHS England.
4.16 If, in exceptional circumstances, the company wants to submit a proposal for a simple discount patient access scheme at a different time in the appraisal process, that is, after their evidence submission, the following conditions apply:
The company must inform the NICE Technology Appraisal Programme in writing of its intention to submit a simple discount proposal, as early as possible.
The simple discount proposal must be submitted to NHS England in sufficient time for it to complete its consideration of the proposed scheme and notify NICE at least 14 calendar days before the next committee meeting, to allow sufficient time for ERG or NICE review.
The company must provide information about the simple discount proposal in a separate submission, using NICE's patient access scheme submission template.
The patient access scheme submission must be submitted to NICE by either the technical report or the ACD consultation closing date, and if possible earlier.
4.17 When a simple discount patient access scheme proposal is submitted after the ACD is released, NICE may choose to reschedule the subsequent committee meeting to allow sufficient time to consider and review the proposed scheme.
4.18 When NHS England approves a simple discount patient access scheme proposal after the release of an ACD, the impact of the proposed scheme on the cost effectiveness of the technology may lead the appraisal committee to revise its recommendations. If the technology is recommended, a FAD will be issued for appeal (see section 3.5.44 onwards). Information will be released so that the proposed scheme and its impact on the cost effectiveness and the recommendations can be understood. Unless there are any appeals by consultees, the FAD forms the basis of NICE guidance on the technology. In certain circumstances, the centre director or programme director and the chair of the appraisal committee may decide that it is necessary to produce another ACD. If so, the consultation process will be repeated. The decision to produce another ACD will extend the timelines for the appraisal.
4.19 When the appraisal committee decides to recommend a technology for use within the Cancer Drugs Fund, the company will be invited to propose a commercial access agreement, or vary an existing agreement.
4.20 For a cancer drug to be recommended for use through the Cancer Drugs Fund, it must display plausible potential for satisfying the criteria for routine use, taking into account the application of the end-of-life criteria when appropriate. Companies should work with NICE and ask for advice about the assumptions used in the appraisal committee's consideration of clinical and cost effectiveness, which must form the basis of their proposal for a commercial access agreement.
Figure 6 Process for considering a proposal for a patient access scheme or commercial access agreement
4.21 Patient access schemes are designed to maximise the opportunity for cost-effective access to a new technology. Therefore, within 12 weeks of publication of the final guidance, companies can request a rapid review to consider new patient access scheme proposals. The rapid review of the guidance is planned, as a priority, into the work programme after final guidance publication. NICE can only consider a new proposal with NHS England's agreement. The appraisal committee will usually consider the proposal within 6 months of the company request.
4.22 The rapid review of guidance will be used for the consideration of a new patient access scheme proposal only. If the company wishes to submit additional new evidence other than for a patient access scheme proposal, NICE will consider whether this would be acceptable in the context of a rapid review or whether it would trigger a full review proposal (see section 6).
4.23 The company must use the patient access scheme submission template to provide details of the proposed scheme, a revised economic model incorporating the patient access scheme proposal, and an updated checklist of confidential information, if necessary. This is in addition to the information that must be submitted to NHS England as part of a submission for a patient access scheme proposal.
4.24 Although NICE will include patient access scheme proposals submitted for rapid review on the relevant committee meeting agenda, NICE makes no public announcement about the specific topics. Scheme proposals submitted as a rapid review are treated by NICE as commercial in confidence and all matters about the proposed scheme (except the existence of the scheme proposal) will usually remain confidential unless consideration by the appraisal committee results in a change to guidance recommendations. In this situation, NICE will issue a FAD for appeal (see section 3.5.44 onwards). NICE releases information during the FAD appeal stage so that the proposed scheme and its impact on the clinical effectiveness, cost effectiveness and the recommendations can be understood.
4.25 If, in exceptional circumstances, NHS England were to approve a patient access scheme proposal more than 16 weeks after guidance publication, the topic could be considered under the rapid review arrangements but it would not be prioritised in the schedule and NICE would need to be assured that the principles of rapid review apply.
4.26 The 2014 PPRS identifies 2 circumstances in which flexible pricing may be relevant:
when significant new evidence is generated that changes the value of an existing indication and
when a significant new indication is proposed.
4.27 Requests to consider a flexible pricing proposal for an existing indication of a technology must be linked to new evidence emerging. The company therefore needs time to gather the additional evidence necessary to justify a price change. NICE will consider reviewing the guidance only in the light of significant new evidence that is likely to have an impact on the clinical or cost effectiveness of the technology. This could include: new clinical trial evidence, new evidence on identified subgroups of patients, or significant new evidence supporting additional benefits previously unaccounted for (for example, long-term outcomes). New evidence does not include new analyses of existing data. Flexible pricing proposals that are not supported by new evidence will not be considered.
4.28 For technologies launched after 1 January 2009, if NICE receives a flexible pricing proposal for an existing indication within 12 months of guidance publication, NICE will consider the impact of the new evidence and the flexible pricing proposal on the clinical and cost effectiveness of the technology. NICE will clarify relevant points with the company before the ERG reviews the proposal. The appraisal committee will then consider the proposal together with the ERG's independent review.
4.29 NICE considers flexible pricing proposals for an existing indication submitted more than 12 months after guidance publication by the standard review process (see section 6).
4.30 All flexible pricing proposals for technologies launched before 1 January 2009 are considered through the standard review process (see section 6).
4.31 When the appraisal committee considers a flexible pricing proposal for an existing indication, the committee will review the original guidance in light of the new evidence and the proposed new price. The committee's assessment of cost effectiveness will be consistent with that used in the original appraisal.
4.32 Although NICE includes flexible pricing proposals under consideration on the relevant committee meeting agenda, NICE makes no public announcement about the specific topics. NICE considers it essential that such proposals can be received and considered in confidence. NICE also understands that companies may suffer commercial and other harm if information on the proposals were to be made public at this point. Therefore, NICE treats all flexible pricing proposals for existing indications as confidential and will not normally release any information about these schemes under the Freedom of Information Act, or for any other purpose at this stage (including during the public part of appraisal committee meetings), unless the company has agreed to this.
4.33 When the appraisal committee has reviewed the existing guidance on the technology in the light of the new evidence and flexible pricing proposal, an ACD will be published for consultation (see section 3.5.26 onwards). Detailed information will be released as part of the ACD consultation so that the proposed new price and its impact on the clinical effectiveness, cost effectiveness and the recommendations can be understood. As with the normal appraisal process, the appraisal committee will review consultation responses on the ACD and develop a FAD. NICE will issue the FAD to consultees, along with the consultation response to the ACD, for appeal. Appeals will be accepted only on points relating to the flexible pricing proposal. They will not consider points previously raised or points that could have been raised at an earlier appeal. Subject to any appeal by consultees, the FAD forms NICE's updated guidance on the use of the technology.
4.34 Flexible pricing proposals for new indications of existing technologies are also covered in the 2014 PPRS. New indications are potential new appraisals. Consideration of their suitability for technology appraisal is therefore covered under topic selection (see section 2).