Drug Development

The substantial developments in therapy over the past 50 years have meant that there are few major diseases prevalent in developed societies for which there is no effective therapy. Recent advances have tended to represent an incremental improvement over the current standard of care. The challenge facing all healthcare systems is how to prioritise and pay for these new therapies. Despite the fall in the number of new medicines being registered, no healthcare system has sufficient funds for all the therapeutic options available today. The UK National Institute for Health and Clinical Excellence (NICE) was one of the first authorities charged with the comparative assessment of new therapies and estimation of cost-effectiveness (Fig 12). Bodies of this type are now found in most developed countries. A detailed critique of the successes and shortcomings of NICE is beyond the scope of this article but recent controversies over novel cancer therapies have brought the role of bodies that decide on cost-effectiveness of new drugs into sharp focus.

One of the key changes over the past 5 years is the realisation that novelty or innovation alone will no longer be sufficient to grant a new medicine access to reimbursement within healthcare systems. It is clear that new medicines will need to demonstrate that they represent a significant clinical advance. It is now critical that the development of new medicines includes measures such as comparative efficacy in relation to the current standard of care, or a clear value proposition based on hard clinical endpoints. Defining value can be difficult as different stakeholders in the process (doctors, patients, and payors) may have different perspectives. For example, patients with hay-fever are interested in whether a new treatment improves their symptoms faster or more completely than their existing treatment, whereas a payor might be interested in whether it means they can return to work more quickly.