Trial outcomes and value – Explaining contemporary clinical trial issues

Trial outcomes and value – Explaining contemporary clinical trial issues

Published on 8 April 2019
Author: Andy Smith

Andy joined the healthcare team in November 2017 after a period as a senior principal in ICON’s Pricing & Market Access consultancy. Prior to ICON he was chief investment officer at Mann Bioinvest and managed healthcare and biotech funds at AXA Framlington, SV Life Sciences, Schroders and 3i Group.


For recently approved drugs with premium prices, healthcare budgets are being stretched and are likely to be rationed in one form or another. To justify the value of the drug, companies have to invest more on measuring their drug’s benefit or outcomes. For companies developing one-off treatments such as gene and cellular therapies, there are expectations that a single price per treatment course will capture the value of a lifetime cure. Here we explore how some clinical trials are now measuring outcomes and endpoints for both small molecules and innovative therapies.

How does a clinical trial translate into value?

Until recently, a positive well-controlled Phase III programme conducted in a large number of patients would usually lead to commercial success, despite the patent position. These days, a positive clinical trial is far from guaranteed to translate into commercial success in many indications and drug sponsors are having to do things differently to demonstrate value to payers. This is even more acute as cheap generic drugs comprise the standard of care in many indications and the unmet medical needs are in smaller, higher value, more difficult to treat patient populations.

Innovative therapies mean innovative pricing

The approval of drugs with very high prices and innovative cell and gene therapies that are proposed to be priced as millions of dollars per treatment magnify this issue of demonstrating value. Innovative therapies are giving rise to innovative pricing mechanisms that invariably involve measuring to demonstrate value. This is easier said than done. Likely winners
  • Clinical research organisations (CROs) such as Parexel, IQVIA and ICON, which have added long-term outcome studies to their product offerings.
  • Pharma and biotech companies that able to provide positive outcome data.
  • Payers, which have to sit back and wait for outcome data before reimbursing.
  • Patients, who are more likely to receive drugs that provide them a benefit.
Likely losers
  • Pharma and biotech companies that cannot show positive outcomes.
  • Whoever collects the data: if small pharma and biotech companies have to collect outcomes data and follow patients in registries to be reimbursed, their costs will go up.
Winners and losers: The companies shown above do not translate into buys and sells as other themes (and valuation parameters) may conflict with this one.

Clinical trials, outcomes, value and pricing


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