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Real-world data to assess value

With a systematic approach, this intelligence can be used to augment the clinical argument for drugs' benefits in the longer term
real-world-data

The pharmaceutical industry is increasingly being called upon to demonstrate the value of its medicines. Funding for health systems across Europe, whether state-funded or insurance-based, is under enormous pressure as the threat of further economic crises intensifies. And as a result, the price of pharmaceuticals is again under scrutiny as governments look for cost-savings in the delivery of healthcare. It was ever thus.

The concept of demonstrating value is currently enjoying a high profile in the UK, where new policy in the guise of value-based pricing (VBP) forms part of radical proposals to reorganise the National Health Service (NHS). However, the philosophy predates the policy. Demonstrating the value of medicines has always been a key objective. At present, it remains the single biggest challenge the global pharmaceutical industry faces. But how can marketers prove value?

The NHS has a long-standing tendency to regard medicines, and indeed all technologies, as a cost item. This has ultimately led to decisions that give undue weight to cost-control and fail to consider downstream impact or take into account the potential benefits of using more effective medicines. As a consequence, there remains not only a high resistance to the introduction of new technologies in the NHS, but also a variation in uptake among clinicians across health economies and weak transmission of innovation to other health economies.

This is all clearly to the detriment of patient care and sound financial management in the medium- to long-term.

Establish true value
Across the board, there needs to be a shift in perception that means the commissioners of care regard medicine as adding value, not cost. Health stakeholders must collaborate to become more assiduous in establishing the true value of any given innovation. And pharma companies must play their part in developing technologies that deliver relevant value, rather than merely suit their own agendas.

The concept of demonstrating value is currently enjoying a high profile in the UK

But shifting the paradigm of 'cost not value' is not an easy task. How can pharmaceutical companies prove value when the existing clinical trial system does not appear to support it? The traditional template for the development, evaluation and adoption of medicines is not configured to generate value-based evidence in a real world setting.

Historically, randomised controlled trials (RCTs) have long been considered the gold standard for generating data to inform the registration and, subsequently, promotion of new medical innovations. The extrapolation of RCT data is an academic exercise that has worked well for many years – but where cost control, widespread financial pressure and ambitions to improve quality of care are the reality, it is no longer enough.

Increased emphasis on cost-effectiveness has driven the need for more sophisticated value-based evidence – much of which is not possible through a conventional RCT. How can RCT data be translated into a real-life setting to help commissioners make value-based decisions about managing disease with public money? On its own, the existing methodology plainly fails to deliver.

Different thinking
Such limitations are forcing the industry to explore new ways of obtaining relevant outcomes data to support the reimbursement of new innovations. Pharma has become more adept in its use of budget impact models and how to cost out benefits along the patient pathway using data captured during RCTs, but there is a limit to how far traditional methodology can go.

There is now increased importance being placed on the development of real-world (RW) data to augment the clinical argument. RW data is defined as data that is collected outside the normal constraints of RCTs, to evaluate what is happening in normal clinical practice. It creates the ability to track the benefits of specific medication at patient level and also increases understanding of the value of improved compliance and adherence. Against current aspirations to develop an outcomes-based health service, RW data is essential.

Recently, the Association of the British Pharmaceutical Industry (ABPI) issued a paper outlining ambitions to establish the UK as a centre of excellence for the collection and analysis of RW data. It said this could help lead to a better understanding of conditions and treatments in a wider population, improve the management of chronic conditions and facilitate the avoidance of preventable hospitalisations. Its report presents a powerful argument for RW data and how it can provide an important platform for assessing the value of medicines – and calls for a concerted, collaborative effort between NHS and industry to make the transition towards RW data collection.

Current rhetoric suggests that the journey towards true RW methodology may be a long one but, in fact, much of what is needed to propel the new approach is already present in the system. A lot of the data for RW studies is already there; it just needs a systematic approach to joining it up. But the RW solution also requires some RW thinking; most notably a shift from the traditional adversarial relationship between the health service and pharma to one of true partnership.

Real-life solution
In today's climate, commissioning decisions, particularly those related to medicines usage, increasingly need to be based on real-life, real-time data that builds a value proposition beyond launch. Currently, decisions are largely based on traditional RCTs that have been geared towards product approval, launch and health technology assessments. Moreover, the huge effort required to make a change in the way care is delivered seems to stop at the moment a pathway is redesigned or a new service is implemented. Measuring the effects of that service often gets lost in the melee.

The solution is to collect and interpret longitudinal data sets that encompass primary, secondary and pharmaceutical care at the patient level, not just for one or two patients, but for large cohorts in a given population. First, data should be collated relating to current, standard treatment protocols. Second, the new product should be introduced to generate a comparative data set for interpretation. This data will provide the platform for a range of commissioning initiatives that can help the NHS optimise treatment pathways and help industry demonstrate the value of its products by isolating the outcomes that have been achieved. (See text box below.)

Such data already exists within the system. It is routinely captured, but needs to be connected and analysed in a systemic fashion. This is neither difficult nor expensive. Data sets can be linked using patients' NHS numbers, anonymised, aggregated and then interpreted by project stakeholders.

The benefits for marketers are significant. Well presented data drawn from local populations, propagated by clinical commissioning groups, can create an evidence pool that is likely to change clinical behaviour far more effectively than the publication of local and national guidelines.

The challenge is to achieve the backing of all stakeholders and this requires a change in mindset. It is all about partnership, but pharmaceutical marketers must play a vital role in driving the agenda. Marketers need to engage in more adult discussions with commissioners to demonstrate the benefits of collaboration. By working together, commissioners can make more robust, evidence-based decisions, pharma can better illustrate the value of its innovations and patients can gain access to high-value, life-enhancing medicines.

Sharing risk
The premise is, of course, about sharing risk as well as sharing benefits. Marketers need to ensure that, at launch, they have mobilised sites (primary care trusts, clinical commissioning groups) that are prepared to work with them and have agreed the parameters for the partnership. It is about saying 'this is what we think we can prove, so where can we share that risk in order to achieve it?' The industry is well placed to provide project management, technology and resources to help conduct the studies. The NHS can unlock access to patient data for the betterment of patient care.

Success will depend upon identifying customer needs and delivering a value proposition that aligns with them. It is those same needs that any cohort study will set out to prove. Customer need is the critical element of the partnership and one that drives the risk share. And it is in this area that, despite significant industry will to facilitate the development of RW data collection, pharma must pay the greatest attention, or risk missing out on its benefits.

Right questions
If the industry is going to use RW data, it needs to make sure it asks 'RW questions': those questions to which commissioners and heads of medicines management want answers and not simply those that pharma companies themselves may like to prove.

The two sides need to talk and agree the outcomes data that will enable decisions to be made and changes to happen. It is about getting in the real world. But for marketers, the solution may not be another world away. It could be right in front of them if they recognise the opportunity and build the partnership.

The marketers' guide to optimising real-world data

The context:
If you have got a new drug and want to charge more for it than the current preferred treatment option, you need to prove that a value equal to or better than that investment comes back as a result of using your product. But extrapolated RCT data alone cannot truly provide evidence of that value. Commissioners need proof, not promises, that your drug works.

The consideration:
Ask yourselves:

  1. Is the NHS going to be called upon to invest in my drug?
  2. Is my price going to be more than that of a treatment currently being used?
  3. Am I confident that I can prove the value of what I am asking?
  4. Could I partner with my customers to develop data that not only proves the value of my drug, but also provides commissioners with assurances if they use it?


The partnership:

Forward-thinking marketers are proposing partnerships with NHS customers to conduct cohort studies in their therapy areas, at population level within Primary Care Trusts (or in the future Clinical Commissioning Groups). Whereas most RCTs study relatively small numbers of patients, cohort studies can draw on huge populations and generate rapid and robust RW data. Longitudinal analysis of the cohort to assess what is happening using the current treatment, and then what happens when your intervention is introduced, can be a real indicator of value. Have hospital admissions decreased? Has adherence improved? What is spending like across the patient pathway?

If improvement occurs in areas that count for commissioners, they will ultimately support continued use of your drug.


Andy LeeTim JonesThe Authors

Andy Lee (left) is commissioning and NHS partnerships director, WG Group and Tim Jones is NHS commissioning consultant and NHS associate, WG Group

To comment on this article, please use the commenting feature below

18th November 2011

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