Successfully introducing a new rare disease therapy

Richard Johnson

Country General Manager, UK & Ireland

The extensive research and development needed to produce new treatments targeting rare diseases is only half the battle. Before marketing, some unique challenges have to be navigated.

Pioneering biotechnology, that is focused on antibody engineering technology, is being used to develop treatments for some of the many rare diseases lacking effective therapies.

To date, this has delivered a number of innovative therapies in the pipeline, spanning conditions in disease areas including oncology, immunology/allergy and the central nervous system. All have the potential to deliver disease-modifying and life-changing results.

Richard Johnson, Country General Manager for the UK & Ireland at Kyowa Kirin, explains how the success of the company’s research and development presents a set of challenges that need to be overcome in order to bring the products to market.

“In many rare diseases, there has never been an effective treatment. This means any company that has developed a drug will nearly always be new to the therapeutic area and so facing a steep learning curve. In marketing treatments for rare diseases, not only is the prescribing physician important, the entire multi-disciplinary healthcare professional team must be fully engaged.  

“The challenge is defining exactly who is managing patients with a rare disease. The nature of rare diseases means that there are often few healthcare professionals with specialist experience and sometimes even the medical discipline involved in treatment varies from one Trust to another.”

This lack of familiarity with the relevant therapy area means it’s important that the learning process begins very early on in the development of a new medicine.

Gathering patient and carer insights is criticalin drug development for rare diseases

Keeping the needs of the patient in focus is critical to the success of any treatment in rare diseases. Patients and their caregivers can provide valuable insight into the challenges of living with a condition.

Their experiences help to define the various referral pathways through the health system and provide an understanding of the inadequacies of current treatments.

Developing partnerships with patient groups can be helpful in reaching patients, although the low numbers of affected individuals can mean that no such groups exist. In this landscape then, the challenge can be to shape and create things.

Market access

The primary challenge is generating a robust evidence package that demonstrates the value of the technology to payers and other stakeholders.

There are a number of drivers of uncertainty in rare disease, including the low number of patients in clinical trials and the lack of data on existing disease burden.

A robust value story requires good understanding of the disease burden for patients and carers, an area where patient groups can give invaluable insight.

Gathering and disseminating real world evidence is key in preparing reimbursement dossiers for medicines targeting rare diseases.

Challenges include the fact that there are usually no disease-specific registries in place, meaning these have to be set up by working in collaboration with all stakeholders.

It can also be the case that even if an early access programme exists, data extraction is understandably restricted and controlled for ethical reasons.

Engaging with payers

Building partnerships results in the development of working relationships with regulatory and health technology assessment bodies.

Experience has shown that the best way to tackle the challenge of securing reimbursement for medicines in the UK is to align closely with health authorities and engage with the existing framework as early as possible.

“The primary objectives of these bodies are to support patient care and improve outcomes,” says Richard. “We market our therapies with the same objectives and so it’s to our mutual benefit to work together to achieve these. Our pipeline includes therapies for a diverse range of conditions; it follows that we are going to touch on lots of different funding routes within the NHS, so we must work in partnership to understand how patients can access our medicines. It is in the best interests of all parties to understand each other’s needs to make sure that patients get the benefits of life-changing treatment.”