Shortening development times for drugs is seen as a key way to tackle the problem of declining R&D productivity in the pharmaceutical industry, witnessed by a steady decline in the number of new active substances coming onto the market since the mid-1990s.
The pilot programme - known as Pilot 2 - is designed to test the idea of continuous marketing assessment (CMA) for drugs in the Investigational New Drug (IND) application phase, i.e. those that are in full development and have either completed a Phase I trial in the US, or have had a pre-IND meeting on the basis of development outside the US.
It is one of two exploratory pilot programmes to be conducted to allow for a comprehensive assessment of the added value, costs, and impact of more extensive feedback during drug development and early review of parts of marketing applications.
The second scheme, Pilot 1, allows developers to submit portions of their marketing applications before the full dossier is ready. The FDA can then give feedback on the likelihood of the data being successful, suggest improvements to the file and increase the chances that the full application will eventually be filed.
These so-called 'rolling' applications are only allowed for drugs that have been earmarked for priority review at the agency, meaning that they answer an urgent, unmet medical need.
Pilot 2 also has this proviso, and is designed to see whether the approach can improve the efficiency of the drug development and review process, and bring innovative products to patients more quickly. During the pilot phase, just one IND product will be considered for each of the 16 divisions of the FDA's Centre for Drug Evaluation and Research (CDER) and the four units in its Centre for Biologics Evaluation and Research (CBER).
The programme will evaluate the cost of such enhanced interaction between the FDA and applicants and whether it improves the efficiency and effectiveness of development. If so, it may be extended to other new treatments.
One early entrant into the scheme is US biotechnology company Neopharm, whose brain cancer treatment IL13-PE38QQR will be one of the first to benefit from the FDA's early input. The company is in the process of designing a Phase III trial of the drug in a rare brain cancer called recurrent glioblastoma multiforme, which affects around 20,000 people in the US.
Called PRECISE, the trial will compare IL-13-PE38QQR to the only approved drug for glioblastoma, Schering-Plough's Gliadel (carmustine) Wafer, which takes the form of a series of coin-sized disks that are placed into the cavity left by surgical removal of the tumour. Neopharm's drug will be delivered by catheter into the brain after surgery.
The aim of the study is to show a 50 per cent improvement in survival with IL13-PE38QQR compared to Gliadel. Patients who received the latter treatment still only have a median survival of around 28 weeks.