Doctor shows patient chart

In this post, I will examine outcomes evaluations and how they can help biopharma demonstrate value and outcomes at a local level in the UK. For biopharma, proving the value of a drug is key to its success in the local health economy. During the process of determining a drug’s effectiveness within a local heath economy it is important that all potential data sources are utilised when developing a case for funding with decision makers and a local outcomes assessment can be an important element of this case presentation.

With this in mind, outcomes evaluations and outcomes audits are suitable methods for proving a drug’s value and measuring treatment effectiveness using observational data.  We have previously discussed outcomes audits in detail, however this approach looks at data retrospectively to view a patient’s progression since receiving a drug treatment.

An outcomes evaluation is more of a prospective process – comparing initial measurements from a patient with measurements taken at appropriate intervals during the patient’s treatment course, which allows for real-time reporting of the patient experience and Patient Reported Outcomes Measures (PROMs).

Both methods take a similar approach to measuring patient outcomes, and both are powerful market access tools when used at the right time.

"An outcomes audit..."

Challenges facing biopharma

There are a number of challenges facing biopharma that may lead to supporting the NHS with outcomes evaluation. Biopharma has traditionally focused on gathering evidence to support regulatory approval rather than demonstrating real-world value and outcomes to a local health economy. While they may have access to strong data from a clinical measure perspective, there must also be data made available from a patient outcomes perspective in order for it to fully prove a drug’s value. If this data is not available, NHS payers will find it hard to differentiate the drug from alternative therapies.

Another challenge facing biopharma when introducing a new treatment is the likely higher cost of their treatment compared to established and generic treatment options.  Payers will need reassurance that the new therapy has distinct advantages over other options and that additional costs may be recouped at other stages of the treatment pathway.  If there is a high cost differential they are also likely to demand a clear positioning of the new treatment to ensure that it is prescribed for those patients that will experience most value.  An outcomes evaluation can support gathering of this information at a local level.

To address these challenges, an outcomes evaluation provides biopharma with two powerful insights to support effective decision making in the NHS: Informed decision making and change management.

Informed decision making

It is important for biopharma to support the NHS with all the relevant information it needs to make an informed decision and to prove that a drug has value within the local health economy.

Evaluation outcome data can be used alongside clinical trial programmes and other market real-world data as part of a business case presented to funding decision makers.  This local outcomes data can support further differentiation of a new drug when compared to other alternatives. It gives the NHS an understanding of how the drug was utilised within their own health economy, and reassurance that outcomes achieved within trial data are replicated in their local patient population. It can also support appropriate positioning of the treatment within protocols and guidelines as it provides funding decision makers with an understanding of the type of patient the drug is effective for. It might be that the patient has tried the generic example or another brand, yet has failed on that treatment or has had unacceptable side effects. If the new proposed drug is more costly, it must be proven that it can give value to a restricted patient cohort such as this as it will reassure the NHS that the costs of prescribing the drug wouldn’t escalate out of proportion.

Change management

If a treatment is likely to positively change the patient pathway, an outcomes evaluation enables the local health economy to trial that new pathway – and determine the scale of that positive change to both the service and patients. Change is notoriously difficult in the NHS, so an evaluation can help a local provider or clinical commissioning groups better understand the implications of how a new improved pathway may differ from the old one, and the scale of change and resource re-investment that is required. By offering this insight in a small cohort of patients, biopharma can support the NHS in preparation for any pathway change.


With a number of market access challenges facing biopharma, outcomes evaluations can help differentiate a drug from its competitors when utilised to complement other market real-world data and published clinical data. By supporting the NHS to evaluate outcomes within a specific cohort of patients, biopharma can prove a drug’s value within a local health economy and demonstrate benefit in a patient population currently not effectively using existing treatments.

An outcomes evaluation can help biopharma to reassure the NHS around associated costs of prescribing a treatment whilst providing further evidence of its place in therapy and its potential impact on evolving treatment pathways. By working jointly with the NHS in preparation for change as a result of introducing a new therapy, biopharma can demonstrate a stronger commitment to that treatment, to the NHS and to the patients it will benefit.