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Precision medicine: new trial designs and approaches to assessing comparative benefit


Precision medicine is rapidly evolving, so evidence generation strategies also need to evolve. Regulatory and health technology assessment (HTA) agencies are struggling to keep up. In this age of precision medicine, manufacturers must think about more factors than ever before to achieve market access success and consider—at an early stage—how they prioritize to establish comparative effectiveness.

This update, available to read in full in the free report “Precision medicine: positioning for market access success,” explores the need for new trial designs and variations in approaches to assessing comparative benefit.

PRMA Consulting is now Avalere Health

A need for new trial designs to achieve the same standards as traditional trials?

HTA agencies have acknowledged that new trial designs, such as basket trials, are required in situations where the frequency of the biomarker within a certain tumor is very low and/or where the tumor type itself is very rare (since these situations would not be conducive to traditional trial design).
However, there is minimal guidance or methodologies around this, so there is a need for such trials to achieve the same statistical or methodological standards as traditional trials.

The default is to enter into managed entry agreements with further evidence generation, if in fact the product is recommended for reimbursement at all.
Examples are shown in the table below. HAS and PBAC have not suggested any changes in approach for how they assess tumor agnostic medicines, while CADTH and NICE have been more forthcoming.


Source: “Precision medicine: positioning for market access success.”

Precision medicine: variations in approaches to assessing comparative benefit

Key considerations from the few assessments that have been made to date, are outlined in this table:

Source: “Precision medicine: positioning for market access success.”

In summary:


No clear changes have been made to support the assessment of tumor agnostic drugs.

  • Pembrolizumab: no tumor agnostic assessment has been undertaken by either agency.
  • Entrectinib: HAS awarded entrectinib an SMR of insufficient, meaning no reimbursement while, again, there is no PBAC assessment available.
  • Larotrectinib: where the HAS restricted reimbursement only to pediatric patients with relapsed refractory fibrosarcoma or other soft tissue sarcoma.


Have been more adaptive with recommendation for one or two of the tumor agnostic drugs without restriction. However, both have required cost reductions or manufacturers to enter into MAAs to reduce the uncertainty around the data.

Considerations for future-proofing the positioning of biomarker driven therapies

To understand where your biomarker driven therapy will be best positioned from a pricing and access perspective, you may want to consider the questions that are examined in our free report: “Precision medicine: positioning for market access success”.


Source: “Precision medicine: positioning for market access success.”

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