Patient-centricity
in rare diseases

Putting patients at the centre of everything you do is not a new concept for pharmaceutical and biotechnology companies.

Virtually all companies in our industry lay claim to tailoring their offering to the needs of those who use their products or services and, in most cases, this is not without foundation. For those companies operating in rare diseases however, the principle is of overriding importance.

In Europe, a rare disease is defined as one affecting fewer than one in 2,000 people. In practice, many conditions have a much lower incidence, with total sufferers sometimes numbering in the low hundreds or even fewer. This means that, as compared to operating in disease areas which affect millions of people, such as diabetes or hypertension, you are closer to the people living with the condition. Much closer.

The respective rare disease communities are often very closely knit and comprise highly engaged and very well-informed people, with knowledge derived from a lifetime of experience living with the condition. Effective co-operation with patient representatives and patient advocacy groups (PAGs) is a critically important success factor when operating in this environment. There is immense value to be gained from supporting the aims of PAGs and tapping into the insight they are uniquely positioned to deliver.

The way Kyowa Kirin conducts our business is defined by the company’s ‘Commitment to Life’ philosophy. This revered mission statement defines our very purpose and guides us as we keep in mind the interests of the people our medicines are designed for, in every aspect of our operation.

Adhering to strict standard operating procedures to ensure compliance with local regulations, Kyowa Kirin has developed engagement programmes to help inform our actions in many different areas. The process is very much a two-way dialogue throughout, there is no setting of predetermined agendas.

The exchange ideally starts as early as possible in the drug development process. Discussions with the relevant PAGs can be used to help make sure that clinical trial programmes are appropriately designed to deliver results that provide relevant assessment of therapeutic benefit.

It is often the case that a new treatment for a rare disease will be addressing a previously unmet need. This naturally means that as the treatment nears launch, there are many challenges that need to be anticipated and appropriate solutions designed. Our experience has been that close consultation with PAGs enables this process to be handled very efficiently. Gaining this understanding can help to influence such diverse areas as supply chain, homecare services, product formulation and contribution to health policy debates.

The international nature of our business can mean that opportunities arise to facilitate introductions to link PAGs in different countries. This is of particular value in rare disease where the very low numbers can sometimes cause resourcing problems, so joining forces internationally can be beneficial.

Keeping the needs of the people our rare disease products treat as our guiding principle has been the single most important factor in our success in recent years.

KKI/UKIRE/NPR/0048 August 2020