The world is still struggling to find effective ways of assessing treatments for rare diseases, according to a new analysis.
Treatments struggle to gain permission from regulators because researchers are not able to find enough patients for convincing clinical trials, according to analysts.
Allie Nawrat of GlobalData, a data analytics company based in the UK, states that developing drugs to treat rare diseases “is fraught with challenges” - from trial recruitment from small patient populations to issues financing innovative and often bespoke therapies.
In an analysis published last week, Allie Nawrat identifies and draws lessons from the obstacles faced through three case studies of treatments for rare diseases. These include a proposed gene therapy for Fanconi’s anaemia, developed by a company called Rocket.
Nawrat states: “Patients with Fanconi experience bone marrow failure as they are unable to create new blood cells.
“Rocket wants to change this situation with its lentiviral vector gene therapy, RP-L102. It is specifically for Fanconi-A, which is the most common form of the disease.
“RP-L102 is currently in a global registrational Phase IIA study, which has been efficacious and safe in patients so far. Based on these promising signals, RP-L102 has received all accelerated regulatory tools from the US Food and Drug Administration and the European Medicines Agency. The company is hoping to complete its biologics licence applications and marketing authorisation applications to the two regulators within the next few years.”
She says: “Regulators and reimbursing agencies still view large, randomised, placebo-controlled trials as the gold standard for measuring safety and efficacy.
“However, in rare diseases, because of the small patient populations, multiple disease sub-types and diagnosis challenges as clinicians may never have seen this disease before, it is difficult to identify and recruit enough patients with similar disease states into a randomised clinical trial.
“It is also common for regulators to want to see clinically appropriate endpoints that can be compared to placebo groups, but because there have been few previous clinical trials in certain diseases, there is a lack of established endpoints.”
All of these challenges mean that about 90% of rare diseases still have no effective treatment.
Nawrat adds: “There has been a gradual improvement in the biological understanding of the largely genetic causes of these complex conditions. Cell and gene therapies can bring long-term, durable, and potentially curative benefit to patients, but they are incredibly expensive to manufacture and complicated to administer.
“This causes challenges for reimbursement agencies who need to weigh up safety and efficacy factors against value for money in terms of improvement in patients’ quality of life and the practicalities of rolling out these therapies into routine commissioning.”
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