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Table 1 Suggested actions, based upon discussion heard at the Sydney Workshop

From: Challenges in valuing and paying for combination regimens in oncology: reporting the perspectives of a multi‐stakeholder, international workshop

Bucket

Action

Description

1

1

Thought should be given to the treatment regimens tested in clinical trials, particularly with respect to stopping rules and targeting treatments to those patients most likely to benefit. Adaptive and platform trials may be useful practically and from an ethical perspective. Combined scientific advice processes should be considered to enable this, including all relevant stakeholders (e.g. different HTA agencies, payers, regulators, patients, clinicians, ethicists, academics).

2

Research is required to identify the most patient-relevant outcome measures to be included in clinical trials. This requires input from all stakeholders.

3

Manufacturers and HTA agencies/payers should work to make more use of post-launch studies and real world data to provide information on alternative treatment regimens including the use of stopping rules.

2

4

HTA agencies should ensure that assessments of combination therapies (like assessments of any treatment) capture the full value of the therapy. There is not currently a case for altering HTA decision rules or deliberative frameworks specifically for combination therapies.

3

5

When combination therapies are assessed by HTA agencies/payers and issues arise regarding the value for money of existing backbone therapies, this should trigger a re-assessment of the backbone therapy by the HTA agency/payer.

6

Manufacturers should not develop combination therapies as single products (except where there are clinical benefits from doing so). This is inefficient and often impossible. Similarly, all stakeholders should work to remove incentives for manufacturers to develop their own “me too” versions of backbone therapies simply to achieve value for an effective add-on technology.

7

Manufacturers should be prepared to revisit the price of backbone therapies (in respect of their use within the combination) when add-on therapies are combined and provide clinical benefit, with a view to ensuring that the price of the combination therapy is commensurate with its value whilst also allowing prices for constituent parts that are acceptable to their manufacturer. Similarly when existing monotherapies are combined.

8

It is important for HTA agencies and payers to communicate clearly with manufacturers what type of multi-use flexible pricing models are implementable to achieve price adjustments in their jurisdiction.

9

Health systems need to have in place appropriate systems to collect data on the actual or likely use of cancer treatments, if flexible pricing and payment mechanisms are to be used.

10

It is important for all stakeholders to consider how to incentivise companies (particularly manufacturers of backbone therapies) to participate in price negotiations.

11

There is a range of views on who (manufacturer, HTA agency, payer) should be responsible for attributing value to the constituent parts of a combination therapy. This needs to be discussed and agreed within specific jurisdictions.

12

Research – involving all stakeholders – into how value could be attributed between constituent parts of a combination therapy would be valuable.

13

There is an urgent need for manufacturers, payers and HTA agencies to explore the legalities of price negotiations between companies in different jurisdictions around the world. HTA agencies/payers have – at least – an important facilitation role in enabling price negotiation.

14

It is important for pharmaceutical companies, regulators, HTA agencies and payers, to further consider how to provide patient access to new low-cost combination therapies. These typically involve repurposed drugs that are found to be newly clinically effective in combination uses. As they are off-patent, there is no manufacturer sponsor to take them through the regulatory and HTA processes. This is not an issue relating directly to high cost combination therapies, but one that is potentially important to improving the quality of cancer care through new uses for off-patent medicines.