There are many factors which determine how likely it is that a pharmaceutical company is patient-centric – but is specialising in rare disease one of them?

Intuitively, it makes sense that a rare disease company would be more connected with their patients and more focussed on their needs because of the rarity of the condition and size of the patient group. Or is it possible that we are being too idealistic? Could putting patients first simply be a business strategic choice irrespective of the therapy areas an organisation works within?

People make companies

When asked to comment, people who work in this field often say that the type of employees who choose to work in rare disease are naturally more patient-centric, because they are often drawn to the area from personal experience. This drives their motivation to make the biggest difference possible.

Single-minded about patients

Specialist rare disease companies are created with a clear ‘mission’: to truly change lives of patients with a specific disease. For companies who started with one or a few orphan drugs, often in one area, the focus is already on disease rather than brand, something their larger counterparts can struggle with.

The organisations themselves are small so often all the employees are outward facing, interacting directly with clinical and patient stakeholders. This hands-on approach has a single-mindedness about it, focussing on the patient’s needs over corporate or economic strategy.

However, in recent times the substantial rewards of orphan drugs have been noticed and big pharma has moved into the previously neglected space. Within these large organisations, smaller teams have had to work in entrepreneurial ways to gain success in the rare disease environment, and are now acting as the new players in the area.

Repurposing rather than inventing

It is common for rare disease treatments to start life as a repurposed product. The starting point for this development pathway is usually off label use, where doctors have been confronted with a difficult patient with very little hope of a true diagnosis or suitable treatment.

The discovery of this unmet need and potential solution is the starting point for rare disease companies to begin the research and approval processes. Does this more clinically driven path to market make a company less commercially driven and more patient focused? Or, is the recognition of this path as a viable and profitable commercial strategy outweighing the patient focus?

While valuable, this approach to addressing unmet clinical need has naturally an adhoc, haphazard quality to it, and needs to complement a more systematic and rigorous innovation programme, which larger companies can bring.

Not everything is about the bottom-line

With fewer blockbusters and the risks and costs associated with bringing traditional drugs to market, the appeal of orphan drugs for pharma has risen considerably. Inevitably, the question is what is the biggest driver, patient need or economics?

The real test of a company’s commitment to putting patients first is when treatments become less viable financially. Where some larger corporations may divest the business, smaller rare disease companies may continue to provide access, a sign that the bottom line does not dominate every conversation.

The complexity of growth

Does size really matter when it comes to how patient-centric companies are? Can patient-centricity be more about strategic choice than default hardwiring? Perhaps every organisation can set their own strategy and choose whether to work in a patient-centric way or not. We are seeing a change in the industry as companies make a move to a patient-centric strategy. Can larger organisations better afford the scale of this change? Or does their size and might cause rigidity due to complex structures and set procedures?

Even companies which have organically evolved around their commitment to meet patient needs will need to change as they grow in size. In the beginning, individual employees are instrumental in defining their company’s patient focus but as time goes on, a more coherent, coordinated and pervasive approach is needed to truly embed a patient centric strategy into every aspect of their business. Although rare disease companies may be naturally more on the side of patient-centricity, it may well be that as long as the commitment is there, all companies can strive to ensure the patient perspective informs their thinking.