The regulatory environment for clinical trials of medicines to treat rare diseases poses an increasing challenge for companies working in the area, according to Boehringer Ingelheim.
Speaking at an R&D conference in London this week, the company’s respiratory medical manager Dr Aruni Mulgirigama acknowledged the introduction by the European Medicines Agency (EMA) of an orphan drug process in 1999 was a key success, but she noted the number of marketing authorisations granted to such products has been falling.
“That decline shows how difficult it is for companies to show the value of medicines and develop them for rare diseases,” said Dr Mulgirigama, who is working to develop Boehringer’s nintedanib for idiopathic pulmonary fibrosis (IPF) – a rare lung disease that has nearly as bad a prognosis as lung cancer.
Among the key challenges is developing trials within the current regulatory set-up, with agencies requests data from randomised clinical trials involving thousands of patients.
“That is simply not achievable in rare diseases,” said Dr Mulgirigama. “That poses questions in innovative trial design and how we demonstrate efficacy.”
In addition, any company trying to get a drug for a rare disease through development also faces the problem of patient recruitment, explained Dr Mulgirigama.
This can be as simple of a lack of ICD-10 classification code for a rare disease, as is the case with IPF, making it hard to pull patient data from the databases of healthcare systems.
“That makes it difficult to understand what the burden of a disease is and where the patients are to be able to go and recruit in countries across the world,” said Dr Mulgirigama.
Despite these challenges, Boehringer had been able to push forward its development programme for nintedanib in IPF, where it is in phase III testing, alongside other trials investigating the drug in oncology indications.
“While it is challenging, I think Boehringer has done a phenomenal job,” said Dr Mulgirigama.
“Most rare diseases have maybe 100 or 200 patients in their global clinical trial programme at phase III, but we managed to recruit more than 1,000. I think that’s in recognition of the fact that there is a large unmet need out there and lots patients who are willing to participate.”
Boehringer is already involved in wider aspects of care for people with IPF, working with PatientsLikeMe to develop an online community for patients with the condition that will allow them to monitor their health and progress over time and connect with others.
