Drug repurposing can help tackle Rare Diseases, says charity
pharmafile | February 29, 2016 | News story | Medical Communications, Research and Development, Sales and Marketing | rare diseases
On Rare Disease Day 2016, UK charity Findacure says existing drugs may hold the key to new treatments and repurposing could save the NHS millions of pounds in tackling the problem of rare diseases.
The charity is holding a conference today to bring together health professionals, researchers and patients to explore how emerging approaches to drug repurposing can give hope to families facing the tragedy and burden of living with rare disease.
Dr Richard Thompson, scientific officer of Findacure, says: “Only around 400 of the 7,000 rare diseases have licenced treatments, and few of these conditions receive research attention. This leaves patients with little hope of ever receiving a disease modifying treatment, and doctors to prescribe drugs that they believe could benefit their patients ‘off-label’, often with little clinical evidence.”
Dr Thompson explains that doctors prescribing drugs ‘off-label’ for other conditions on a fairly frequent basis, and that this practice could present new hope for people with rare diseases for which the treatment options are limited. He comments: “Off-label prescription of drugs is actually surprisingly common in the health system. There are elements of uneasiness around it, because monitoring of the treatment isn’t coordinated or widespread; however, when no other treatment option exists, as in the majority of rare disease, it can be the only hope for both patient and doctor. When properly monitored, it can lead to medical breakthroughs by finding new uses for existing drugs. This is known as drug repurposing.”
Where evidence from patients, clinicians, or researchers suggests that certain drugs are beneficial, Findacure is aiming to run clinical trials to provide a more robust body of evidence to support the off-label prescription of drugs in rare diseases. The charity says this is complicated, due to the fact that the rarity of these diseases can often mean there are insufficient patients to participate in a trial, as well as the problem that pharmaceutical companies frequently lack the appetite to invest in research when the potential market is so small.
One of the achievements of the 100,000 Genomes Project has been support for a consistent platform for genomic analysis in the UK, which is providing a means of identifying clinicians that might have candidates for trials. The Sapientia technology supports a more accurate clinical diagnosis for patients with rare diseases by highlighting the gene mutations associated with disease. By providing a greater understanding of the faulty pathway, it is easier to see how a drug can provide a benefit.
Dr Tom Weaver, chief executive of Congenica, the company behind Sapientia, comments: “On average it takes seven years to diagnose a developmental disease; sadly for many children this is too long. Sapientia provides a rapid diagnosis and – crucially – is building a knowledge base of rare diseases and their symptoms. This will help consultants to identify those who could benefit from new treatments, leading to increasing success rates in clinical trials.”
To fast track the search for beneficial drugs, Findacure is developing a new approach to funding trials using Social Impact Bonds.
Dr Thompson explains: “An effective treatment has the potential to both help patients, and save the NHS money. Using a social impact bond, a proportion of these savings can be reinvested to fund further research, creating a win-win situation.”
An example of the success of this approach is a trial funded by Findacure’s US-based partner ‘Cures Within Reach’, which found Pfizer’s drug Rapamune (sirolimus) could be effective in treating the deadly childhood disease Autoimmune Lymphoproliferative Syndrome (ALPS), which requires hospital confinement and expensive forms of treatment.
According to FindaCure, this research approach offers an estimated average saving of more than £50,000 per patient per year over current treatment, meaning that if only 10% of ALPS patients in the US benefited from sirolimus, it could still generate annuals savings of £3 million on a total investment of £150,000.
Dr Thompson continues: “Findacure’s development study is focusing on building cost of illness models for specific rare diseases, allowing us to quantify their current cost to the NHS, and the amount repurposed drugs could save the health service.”
Ten clinical diseases will be targeted and Findacure is in the process of setting up a proof of concept study for the first of these, congenital hyperinsulinism (CHI).
Findacure is confident that the treatments will be affordable. Dr Thompson comments: “As the current plan is to work on an off-label prescription model, the drug couldn’t be marketed for the new rare disease treatment.
“By targeting only generic drugs, the price point will already be low, and any given generic company would struggle to raise it. This should protect the price, ensuring that all partners get the benefit of any successful trials.”
Findacure says the NHS has been supportive of its approached and that it has encopuraged further development of the model, to prove it can deliver both cost savings to the NHS and improved healthcare to rare disease patients.
Joel Levy
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