Getting approval for regulators and payers

Once a pharmaceutical is approved by country regulators, how does one justify to payers that the product is worth the price.  An article at Real End Points identifies a number of key trial design aspects that are vital to proving to payers that the product is cost effective.

First, having having a “meaningful comparator is obviously important” states Roy Baynes, SVP Oncology Therapeutics at Gilead Sciences Inc.  Often, the correct comparator may not be clear, or the standard of care may change between the start of the trial design and when the trial finishes.  At One the one extreme is Germany, where it is critical to use the exact comparator requested by the G-BA reimbursement authority in one of the clinical trials.

Second, firms need to specify valid outcome measures.  For cancer treatment, overall survival and progression free survival are popular alternatives.

Many European Health Technology Assessment agencies, such as NICE, still consider overall survival (OS) as the gold-standard, but the reality is that in cancer in particular, “if you’re treating adjuvantly or in the front-line for recurrence in a treatment-responsive disease, achieving OS may be extremely challenging,” notes Baynes. It simply takes too long, and requires too large a trial, or is fundamentally not achievable because of effective and active salvage therapies.

Many regulators view progression-free survival (PFS), if of significant magnitude, as a reasonable surrogate for OS in a number of cancers, but “it’s not widely-accepted” by payers, acknowledges Ilana Widera, Director of Market Access, European Oncology Business Unit at Pfizer.

Quality of life improvements (e.g,. reduced side effects) can also be used to demonstrate value.  Many payers, however, do not place much value in reducing the side effects from a drug.

Is conditional reimbursement a solution? In theory yes.  Conditional reimbursement permits payers to reimburse for new drugs, but then later revoke reimbursement status if promised benefits do not hold up to real-world data.

“Usually [conditional reimbursement] just provides an opportunity [for payers] to lower price,” warns Widera. And this hits upon what some sponsors see as a somewhat contradictory stance among payers: they want proof that drugs work in the broader population, yet at the same time, says Baynes, firms “need to show how to narrow the relevant population, so the product is more likely to be covered” (since fewer people means lower cost).



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