Increasing the value of new medicines: utilising the patients’ perspective

Dr Paul Stuart-Kregor of The MSI Consultancy discusses how to demonstrate real value of new medicinal products by highlighting the value it brings to patients.

Payers and providers are increasingly demanding a much more thorough understanding of the 'true' value of medicines, to help inform decisions on what can and cannot be prescribed. Obviously for the industry a key step in that process is gaining regulatory and over-arching 'market access' approval – but this in itself does not guarantee commercial success, as even 'big Pharma' has discovered.

The key challenge therefore, is how the industry identifies meaningful 'value' that clearly differentiates their molecules. I believe the secret to this is a deep understanding and integration of the payer, provider and patient needs, that then allows companies to differentiate the asset or therapy portfolio, and help ensure the asset is something that people are willing to pay for.

We are all familiar with the standards expected of new medicines if they are to reach regulatory approval. Increasingly that approval is only forthcoming if the molecule shows some advantage over existing therapies on the usual parameters of clinical efficacy and safety / tolerability.

"...many new drugs do not offer such significant and marked advantages over their competitors."

However, proving the real 'value' of what the new molecule offers over existing therapies can be a real challenge. Not least when Pharma companies fail to complete head-to-head trials against current standards of care. Just look at Germany's Institute for Quality and Efficiency in Healthcare (IQWiG's) failure to award Boehringer Ingelheim's Trajenta premium pricing as "No such added benefit can be inferred from the dossier, as the drug manufacturer deviated from the G-BA's specifications and chose a different comparator therapy."¹

Increasingly, authorities are looking for real, positive effects on outcomes over the current 'standard of care'. As a tax payer, any one of us would agree that's sensible. Why should we pay more for something that is not 'better'?

We know that this can be achieved by demonstrably better clinical efficacy but many new drugs do not offer such significant and marked advantages over their competitors.

Alternatively, we can demonstrate how our medicine allows the healthcare system to do more or do more for less, due to its impact on the whole process of healthcare delivery.

As The Kings Fund famously said in 2010: "Despite a century of developments in medical technology, and vast improvements in the ability of medical science to prevent, diagnose and treat disease and ill health, attempts to measure the outputs of health care in terms of their impact on patients' health have not progressed beyond Florence Nightingale's time."

And yet patient outcomes have to be the best measure of the success of healthcare delivery, and within that, pharmaceutical- or device-based interventions.

Some people in the payer community certainly get it. As Donald Berwick, President, Institute for Health-Care Improvement, Professor at Harvard University and recently appointed to head President Obama's Center for Medicare and Medicaid Services (CMS) put it: "The ultimate measure by which to judge the quality of a medical effort is whether it helps patients (and their families) as they see it. Anything done in healthcare that does not help a patient or family is, by definition, waste, whether or not the professions and their associations traditionally hallow it."²

"Some people in the payer community certainly get it."

So, if we are to truly demonstrate the 'value' of our medicines which only show marginal benefits on efficacy, safety and healthcare economics, then we need to consider the benefit to patients beyond those traditional parameters.

It still staggers me that this is not fully understood. For example, in 2011 PHA Europe carried out a large scale pulmonary arterial hypertension (PAH) Patient and Carer Survey. In that survey the impact of this often fatal and debilitating disease on patients and their carers was brought to life. It's not just the clinical / medical impact. "Employment / work and income emerged as areas of particular concern for both patients and carers: 85% of patients and 29% of carers said their work was affected by PAH. Nearly three-quarters (73%) of patients said their household income was reduced by PAH, leaving most families with below average household incomes."

Yet if we were producing a medicine that significantly improved a patient's health so that they could go back to work, they then become less of a burden on government by not claiming benefits and contributing by paying taxes. So there is a real gain. Look at the impact biologics have on people with rheumatoid arthritis. Yet factoring in the positive consequences of treatment in financial terms seems to be lagging behind other areas.

It is obvious when you think about it but this is not normally built in to clinical research.

However, companies are starting to think about it. Novartis has set up a new partnership with the US pharmacy chain Walgreens. Novartis hopes to utilise the national network of Walgreen pharmacies, effectively turning them into local walk-in clinical trial centres. Pilot studies have already been completed and following their success the partners now expect to launch their first 10,000 + patient clinical study this year.

"...factoring in the positive consequences of treatment in financial terms seems to be lagging behind other areas."

Clearly there are significant cost advantages but also there is a positive impact on recruitment. It could well transform participation in clinical trials given it may only now involve stopping at the Walgreens on the way home rather than more time consuming activities.

The real opportunity for 'patient-reported outcomes' is this opens up the potential for 'real-world studies'; following patients outside the artificial confines of a clinical trial, which could prove to be extremely useful as regulators and healthcare systems seek ever-more robust data on patient outcomes.

What this focus allows is the possibility to pick up more patient-centric measures and patient-reported outcomes of living with chronic diseases which often go missing in normal clinical settings.

That should in turn help us support the choice of our brand in Donald Berwick's brave new world.

References

1. IQWiG

2. Berwick, 1997

The next article from The MSI Consultancy will be published on the 4th November

About the author:

Dr Paul Stuart-Kregor is a Director and Founding Partner at The MSI Consultancy, a CELLO HEALTH business. He can be contacted at pstuartkregor@msi.co.uk or visit the website www.msi.co.uk

Why should we pay more for something that is not 'better'?