Pharm Exec: Transparency is not simply a legal concept; it is a cultural mindset built on a willingness to communicate around things that are important—especially to others—and to build relationships that stretch beyond the sound bite. How well do you think the EMA is communicating its values and objectives?

Rasi: Communication starts with good institutional ties among our key stakeholders. The agency has built a very important coordination capability through the Heads of Medicines Agencies [HMA] group, which gives us a regular vehicle to promote our alignment goals with 44 national registration and review bodies throughout Europe. Earlier this year, I launched a new Scientific Coordination Board, composed of the seven heads of the committees and leads for a number of working parties, to develop a vision that integrates the expertise of all these groups. The board will not deal with issues of specific medicines but rather on identifying and resolving challenges to the overall drug evaluation process. Improving working contacts with patients is another priority, building on the working party we already have in place that gives patient organizations formal representation on our human medicines scientific committees, including pediatrics. And we are exploring ways to provide more support to help keep the patient groups independent and to allow them to invest in the greater expertise needed to contribute to the complex science our committees must deal with.

I am most excited about the initiative on disclosure of data from clinical trial results. This is where the EMA is playing a global leadership role in making the regulatory process transparent and trustworthy. Our credibility rests on the ability to communicate why we take a decision on a particular medicine, and that depends in turn on displaying in public the data driving that decision. It is also valuable in that disclosure, by encouraging more debate on interpretation of trial evidence, will enhance the rigor, integrity, and ultimately the reliability of our evaluation procedures. This is an important objective, as changes in the way clinical trials are conducted—today, many trials are now global in scope, and beyond the reach of any one regulator—raise many questions about how to keep the current system effective, honest and useful to reviewers seeking the best outcomes for patients.

Rasi: Disclosure is easy in principle, but defining a process can get complicated. We are holding a stakeholder discussion on our initiative on November 22, which will seek to resolve four transactional issues. These are (1) procedures on how to classify and when, or if, to release such data; (2) ensuring that those who receive data have sufficient expertise to analyze and apply it without manipulating or misrepresenting the results; (3) creating specific ground rules to protect patient privacy, including whether to sanction the release of large patient data sets; and (4) commercial confidentiality considerations, including data scrubbing to prevent disclosures that create a competitive advantage or disadvantage. I am confident we can work these issues out, especially as I see many companies moving in this direction, on their own initiative.

Pharm Exec: What is your assessment of the industry position?

Rasi:It is positive and constructive overall. Industry engagement around disclosure makes our efforts easier to execute. Although the industry's stance is not homogenous, I think we all agree that the trend to greater transparency in accessing trial data is irreversible. The challenge lies in making it work at a time when the basic model of the clinical trial is splintering, in the face of advances like biomarkers and other tools of personalized medicine. Regulators have an obligation to clarify standards for trial design and evaluation where there may be two, three, or even more options that companies can follow for a single compound. It simply reflects reality; some tested compounds will benefit from the availability of biomarkers that can serve as surrogate clinical endpoints, while others may not. We must work together with industry to determine how seriously we should invest in validating results based on these new high-tech tools. I would add that predictability from the regulator—on what factors are needed to make a good evaluation—is vital to controlling the soaring cost of these trials.

Pharm Exec: Are you coordinating your approach on trial data disclosure with the US FDA?

Rasi: The FDA is aware and supports our activities. But the US environment is different than here in Europe, where there is strong institutional pressure to take this forward. We will act on this initiative, after November 22. It is my most important priority at the moment.