Patients are in the driving seat delivering cutting-edge, patient-centric research the market needs (Guest blog)
19.05.16
From healthcare reform and the ascension of health technology assessment (HTA), to the empowerment of patients and the near-extinction of blockbuster drugs, the pharmaceutical industry is under intense pressure. The established commercial model is no longer fit for purpose and, as a result, it isn’t just incremental change that is needed, it’s seismic.
The whole healthcare system is speeding towards a tipping point. Policy-makers and payers are demanding action on prices and market access approaches that reward real-world outcomes and not merely the promise or hope of innovation. The traditional model of price-taking is being challenged by HTA, rapidly increasing demands on flat-lining health budgets, payer influence and increased patient advocacy, knowledge and information.
The customer has changed and suddenly patients are at the centre of the value equation.
Recognising that the landscape is changing, payers, seen as the traditional pharmaceutical ‘customers’, are adapting their models from buying costs to buying service, treatment and care outcomes. How decisions are made, what information is needed to make them, and importantly who makes them, has changed. Pharmaceutical companies have to change too.
At Myeloma UK, we believe that the future commercial model of successful pharmaceutical companies must have at its core the development of effective relationships. This means genuine partnerships with academia, medical research charities and patient advocacy groups, HTA bodies and payers as well as other key stakeholders involved in the delivery of improved patient outcomes.
It means moving away from the traditional global model of only generating regulatory ‘safety and efficacy’ data. This new model would recognise that regulators, whilst important, are no longer the final hurdle before market, and that a new model is emerging, one that is built on establishing early engagement and a broader but strategic approach to evidence development.
Patient-driven organisation’s such as Myeloma UK understood the need for change more than a decade ago, and have been working to facilitate the generation and collection of data which are important not only to regulators, but also to HTA bodies, payers and patients. Increasingly, the role of research charities and patient organisations such as Myeloma UK has evolved from a primary emphasis on grant funding to a driving force that is advancing scientific development and leading cutting-edge, patient-centred research.
While most stakeholders are only focused on post approval data collection, Myeloma UK is equally focused on generating additional real-world data, by running trials in parallel to the regulatory evidence development programme.
The Myeloma UK Clinical Trial Network (CTN), has been pioneering partnership approaches to strategic evidence development through a ‘hybrid trial’ concept, to generate local market data aimed at de-risking the approval, adoption and diffusion of new drugs into UK health systems.
We do this by working closely with pharmaceutical companies, stress testing their clinical development programmes, and identifying where their regulatory trial designs are likely to fall short from a UK perspective. In an attempt to address this, we propose an investigator-led trial design that will help to address these data issues. In a recent pilot study our CTN collaborated with NICE Scientific Advice, submitting a trial concept for myeloma treatment, ixazomib, which supplemented the regulatory trial with the aim of providing additional and complementary evidence to support the HTA and NHS Commissioning of the treatment in the UK.
Alongside this, through our Health Services Research Programme we are funding and facilitating research, to define approaches to collecting patient preference data on the benefits and risks of new drugs. Generating this data as part and parcel of clinical development programmes will shine a very bright torch on the value proposition of a new drug at every stage of its development and will help regulators, HTA bodies and payers to better understand the value of traditional clinical endpoints and quality of life data from the perspective of patients.
The UK is well placed to reinvigorate the way drugs are brought to market with a focus on what the patient needs and wants. Partnerships such as those enabled and encouraged in the UK by the National Institute for Health Research and the Medical Research Council, and a strong and clear policy direction from government, will help to fuel the role of patient-driven and medical research organisations. It will allow them to play a key role in drug development, generating the data needed by a new demand-driven market, to ensure a more compelling value proposition to regulators, HTA bodies and payers, leading to improved access to new drugs, the better utilisation of scarce resources and improved patient outcomes.
