Why is Patient Feedback Critical to Rare Disease Treatments?

Why is Patient Feedback Critical to Rare Disease Treatments?

Why is Patient Feedback Critical to Rare Disease Treatments?

A disease is designated as ‘rare’ if it affects only a small proportion of the population (roughly 0.05% in the EU). The term ‘orphan indication’ describes a disease that will most probably be rare, and has – as yet – has no convincing treatment. An ‘orphan drug’ is one that is being developed, or tested, specifically for a rare or orphan disease where no existing treatment is currently available to patients.

The LDA Research team is known for its tenacity and subsequent success rate when it come to medical market research in this area. Patient feedback is absolutely crucial to marketing rare disease treatments. Accessing those patients require a unique approach however, given the small patient numbers, the difficulty of accessing them, and the lack of available data. LDA patient feedback helps client companies to:

  • Understand the market size and referral networks
  • Provide key data for reimbursement and NICE appraisals
  • Understand, through the use of patient stories, how to market the product successfully
  • Locate potential clinical champions who can help with uptake and reimbursement, and foster relationships with Key Opinion Leaders (KOL) programmes.

The Voice of the Patient

Many research agencies are reluctant to even attempt the recruitment of hard-to-reach patients suffering from rare or orphan diseases. This is down to the small population numbers and the time-consuming nature of the pursuit. In order to overcome these challenges, LDA Research has implemented an innovative approach.

Our ‘Voice of the Patient’ Facebook forum opens up an ongoing channel for discussion with a huge variety of members who join in order to share their experiences as ‘patients’. We hold regular, moderated discussions on a wide range of topics to do with different aspects of healthcare. And we regularly provide the opportunities for followers to take part in paid surveys on behalf of sponsors in the medical industry.

The ‘Voice of the Patient’ has enabled us to build far-reaching and sustained links with patients and patient organisations.

Examples of LDA Market Research With Hard-to-Reach Patients

1. Lennox-Gastaut Syndrome

This is a rare syndrome that affects 1-2% of all childhood epilepsies. Onset is usually between 2-7. We contacted carers of children suffering with this syndrome as the patients tend to suffer from cognitive impairment. The carers we spoke to had lots of information that they were keen to share, and were pleased to be able to to help improve support for people living with Lennox Gastaut Syndrome.

2. Intermittent Self Catheterisation

The patients for this new product are designated ‘hard-to-reach’ because they may be suffering from spinal chord injury or urology related problems. This is a ‘sensitive’ area to discuss and we had to reach out to patients who are not members of particular patient associations owing to the differences in their underlying conditions.

“LDA contacted me to talk about intermittent catheterisation. Its a really sensitive issue for me and I was nervous about talking about it, But the recruiter and the interviewer really understood the issues I am facing. I felt that someone had listened to me properly for the first time.”

Additionally, we have recently recruited patients with Cystic Fibrosis, HIV, NSCLC, Von Willebrand Disease and Nephropathic Cystinosis. These are all considered hard-to-reach patient groups and LDA Research takes pride in being considered a trusted source for accessing these patients.


LDA Research team are experts when it comes to locating hard-to-reach patients for participation in medical market research. Give us a call to find out more – 01525 861436