I was pleased to be able to take part in a recent Twitter Q&A on patients and digital media. John Pugh, Digital Innovation Team Leader at BI, and Dave Pinnington , Digital Lead at Pfizer, which was coordinated by Eye for Pharma. I asked how the advent of digital media would affect trial recruitment and design, now that patients, and indeed health care providers, can access detailed information on trials and new treatments. I was particularly interested in the implications for expanded access programmes, for patients for whom Phase III results will be too late.
Digital media’s impact on trial recruitment within the specified indication can certainly be seen in the popularity of Patients Like Me and other initiatives. As for trial design, the impact of increased numbers of informed patients is ‘inevitable’, according to Pugh. However, Pfizer’s REMOTE trial, designed to evaluate an overactive bladder drug and conducted entirely online, was recently halted due to a lack of participants. While disappointed, Pfizer for one aren’t abandoning the concept of virtual trials just yet. REMOTE also generated useful experience in terms of regulatory approval and data capture.
Expanded access is now the preferred term for the use of investigational drugs in patients who have exhausted all other treatment options, and who do not necessarily meet all of the inclusion criteria for ongoing trials. It’s less equivocal and certainly less emotive than ‘compassionate use’, but both will be found in use. However, the implementation of such programmes is less streamlined. A survey conducted in 2010 by ECRIN found discrepancies in the regulation and interpretation of expanded access between different countries. More recently (May 2013), the FDA released draft guidelines for industry on expanded access, which are currently under review. It would seem sensible for companies to adopt an expanded access policy as part of the design and protocol of clinical trials, particularly at the Phase III level.
Despite these advances, an unfortunate example of how not to handle expanded access featured in the Twitter feed. Biomarin’s treatment of Andrea Sloan, who requested access to an experimental drug for ovarian cancer, is beyond any parody of bad Pharma. The absence of any concrete policy, coupled with the painful leaked emails from the CEO dismissing Ms Sloan and her supporters, were indicative of an ingrained failure to address expanded access.
Widening participation in expanded access programmes seems likely as companies come under increased pressure from informed patients. To quote Pugh and Pinnington – ‘any business ignoring the expectations of customers is doomed’, and ‘democratisation of information is changing the patient dynamic and how we should engage’.
There are some caveats to this, both procedurally in that expanding access can hamper recruitment to earlier phase studies, and in terms of unknown risks to patients. There may also be risks inherent in switching to a new drug as monotherapy, such as resistance development in HIV. How best to handle the data arising from expanded access, and how to avoid compromising current and future drug development, could be the next topic for discussion.