As always at this point in the political cycle, there is a great deal of uncertainty about future policy directions, funding sources and the longevity of certain programmes.
Uncertainty has a habit of making people feel anxious, insecure and stressed. It is often cited anecdotally – personally, the uncertainty over planning permission for my recent house extension has undoubtedly been stressful – and there are many academic studies showing a link between uncertainty and stress. Uncertainty is sometimes more impactful than negative news. It wears you down, uses up precious thinking time and energy and paralyses decision-making. Perhaps that’s why being able to deal with uncertainty, to hold many options open or make decisions on incomplete and uncertain information, is a prized trait of great leaders.
Healthcare suffers from uncertainty as much as any other walk of life. For patients, it’s the unknown pre-diagnosis, the uncertainty over whether existing treatments will work for them or the risk and potential benefit of entering a trial for a new treatment. Clinicians and payers share these concerns, and also have to try and predict how many people will become ill, of which diseases, and what the next big public health threat will be. Our overstretched NHS has to make investment decisions based on uncertain information, and may even be unsure about its own longevity as an organization.
Our regulatory and reimbursement structures – in the UK, NICE, the MHRA and NHSE – are mechanisms for reducing uncertainty and making judgments about how much risk is appropriate. But are we, in emphasizing a reduction in uncertainty before medicines are offered to patients, missing out on getting those medicines to patients faster and more cost-effectively? Can some patients, in fact, handle far more uncertainty than we are willing to give them, in return for a potential upside?
To be great leaders and great healers and to lead the world in medical innovation, we have to become comfortable with uncertainty, and learn how to use it to our advantage. That means radical changes to our existing structures for drug discovery, regulation of medical treatments, cost effectiveness analysis and appropriate evidence-gathering mechanisms.
CASMI sits on the Early Access to Medicines (EAMs) taskforce and is a leading player in the Medicines Adaptive Pathways for Patients (MAPPs), two attempts at a more flexible and adaptive approach to new medicines development that embrace uncertainty.
For both approaches, medicines will be licensed for use by specific subgroups of patients much earlier in the process than for the standard pathway. This means that these patients will gain access to medicines that would otherwise take years to finish phase three trials. However there will inevitably be a greater degree of uncertainty in decision making at this earlier stage and it will be necessary for regulatory authorities, drug companies, patients and clinicians to be open to revising their views on a particular treatment as more evidence is gathered. The strong leaders who will take these approaches forwards need to embrace uncertainty and use it to give greater opportunity for risk benefit trade-offs that accurately reflect the needs of patients.
These and other initiatives like them are an important step towards a more mature approach to medicines development that engages all stakeholders, but especially patients and the health service, in making more nuanced choices about risk and benefit and the degree of uncertainty that is acceptable.
Megan Morys, Deputy Director of CASMI