Patient groups have a fundamental role to play in the rare disease world. They are a vital lifeline to patients and their families, offering support and useful information about living with the condition. They are experts in their rare diseases, with valuable insight into the disease progression and impact. They represent patient voices and they understand patient needs for therapy and treatments. As a result, they are a key partner in patient care and research development.
Yet, only half of all rare diseases have a disease-specific patient group. Where patient groups do exist, they usually come in the form of ‘kitchen-table’ organisations, set up by people living with rare conditions or those who have an affected family member. They tend to be small, either entirely volunteer run or with a couple of members of staff. While they have the ambition and passion needed, they may not necessarily have the experience or knowledge to achieve their goals.
Findacure addresses this need through programmes for rare disease patients and advocates. We run training workshops and webinars, a peer mentoring scheme, and an online portal to facilitate training and learning for patient groups. We have also published two books supporting rare disease entrepreneurs and patient organisations. Our ultimate aim is to empower and upskill patients and advocates to build the capacity of their groups and communities, allowing them to effectively represent patient voices and become valuable partners in research.
Though there are over 7,000 rare diseases, only around 400 have licensed treatments. This small number of treatments is due to the small number of patients in any given disease. This makes it hard to generate research interest, and hard for pharmaceutical companies to recover the huge investment they have to make to develop a new drug. When they do develop new drugs for rare diseases, the price is usually very high – a 2014 pharmaceutical report found the average cost of rare disease drugs as almost $140,000 per patient per year, compared to a cost of $21,000 for common diseases.
For patients, this lack of treatments means that millions are left to manage their symptoms, rather than treat the underlying disease. This makes research their main source of hope, and many patient groups aim to encourage and support research into their specific disease. Taking the results of this basic research and delivering it to patients in the clinic remains a huge challenge. Findacure’s drug repurposing programme aims to tackle this problem.
Drug repurposing is like recycling. It takes existing generic drugs, approved for human use, and identifies new illnesses that they could treat. As the drug has already been used for many years to treat patients of one or more diseases, doctors and scientists already know a lot about how it works and its side effects. This extra knowledge makes drug repurposing faster and cheaper than traditional drug discovery – ideal for underfunded rare diseases!
Unfortunately, such projects are unlikely to be taken on by the pharmaceutical industry unless they can find something to patent – a challenge when working with generic drugs. Findacure’s drug repurposing programme aims to provide a new, non-profit route for patient group- or clinician-led clinical trials of repurposed drugs to be financed. We are using social financing to fund drug repurposing clinical trials into rare diseases, with the aim of delivering low cost generic treatments through the NHS, which will improve the health of rare disease patients and save the NHS money.
To learn more about or drug repurposing programme, and our innovative social impact bond, visit our rare disease drug repurposing social impact bond page.
The rare disease world is complex. Delivering new treatments to a dispersed, relatively small, and poorly understood patient population is hugely challenging. Establishing an effective group or charity to support these patient populations is no more straight-forward.
Success relies on collaboration. The importance of identifying the right people with the right skills or knowledge for any task cannot be understated. Clinicians, patient groups, the pharmaceutical industry, charities, academic researchers, the biotech industry, and biomedical support services all have a key role to play in meeting the huge unmet need of the world’s 350 million rare disease patients.
Findacure’s scientific conference and networking events aim to bring all of these stakeholders together in the same room, to share ideas and experience in the rare disease field. Our ultimate aim is to promote new collaborations that will help to drive rare disease projects forward, whether in the field of scientific research, or patient support and empowerment.
We also organise an annual student essay competition to get medical and biology undergraduates thinking and discussing rare.