Today’s global infrastructure for drug development presents many opportunities as well as challenges for CROs. Current development trends around biosimilars, orphan drugs, as well as the conduct of clinical trials globally, are among the key areas regulators are focused on.
 
On behalf of its members, the Association of Clinical Research Organizations (ACRO) engages regulators around the world promoting patient safety for the conduct of clinical trials. The association also plays a key role in advocating for increased appropriations for the FDA’s international activities and frequently meets with FDA officials to discuss a variety of regulatory issues of importance to the CRO industry. Karen Noonan, vice president of Global Regulatory Policy, ACRO, discusses key regulatory issues, biosimilars, orphan drug development, and supportive efforts aimed at a stronger FDA. –KB
 
Contract Pharma: What progress is being made with regulatory initiatives for the development of biosimilars in the U.S.?
 
Karen Noonan: ACRO members have been actively involved in biosimilar development globally, outside the U.S. While Europe has been approving biosimilars since 2006, the U.S. is still implementing the pathway created by Congress with the passage of the Affordable Care Act in 2010. But, we do see progress. On May 14, the FDA published the first of five draft guidances anticipated this year. This adds to the trio of guidances issued in February 2012 and the March 2013 procedural guidance. We are hopeful that we will see final guidance from the FDA this year and perhaps even a biosimilar or two approved for use in the U.S.
 
From ACRO’s perspective, this is not only a question of the U.S. maintaining a robust research environment, but also issues of patient access and healthcare costs. ACRO is an active member of the Alliance for Safe Biologic Medicines, which is focused on biosimilar development.
 
CP: What major regulation hurdles remain in India and China for the conduct of clinical trials?
 
KN: In China, the primary issue for our members continues to be the amount of time it takes to get approval to begin a new clinical trial. A year or so ago this might have taken as long as 18 months, on average, but we are seeing some significant improvement in this timeline.
 
In India, currently, the hurdles are numerous with an unsettled political situation, a very difficult business environment and an uncertain regulatory structure. ACRO is continuing to reach out to the regulatory authorities and the Ministry of Health to voice our concerns and are working with a number of organizations on the ground in India as well. Everyone wants a safe, ethical, stable environment to conduct clinical research but the current state of the regulatory system makes this extremely difficult. 
 
CP: We see significant investment in the orphan drug market among pharma/biopharma. What are some recent changes within FDA regarding support of orphan drugs?
 
KN: The FDA has been very supportive of orphan drug development, we think, both in its approaches to clinical trial design and data analysis, as well as in risk/benefit assessment. In 2013, the FDA approved 27 new drugs, nine of which were for orphan disease, so one-third of the total. As you might expect, our members are doing a lot of work in the orphan drug area. In addition, in March 2014, the FDA held a joint EMA/FDA/PMDA orphan designation workshop and in January the Agency held two public workshops on complex issues in drug development for rare diseases.
 
We are also seeing very encouraging signs from the FDA around “Breakthrough Therapies.” Since late 2012 when the category was created, the FDA has received 156 applications for the “breakthrough” designation and has granted 44 of these. We see an opportunity for many of these innovative regulatory approaches to be applied more broadly, so that we can cut development time for many drugs that patients are waiting for, not only those with special designations. The ACRO members are very interested in bringing innovative ideas for drug development to the FDA and our sponsor companies. 
 
CP: In what new areas do you see greater appropriation of funds within FDA? What areas remain a concern for the industry?
 
KN: ACRO is very active in the Alliance for a Stronger FDA, which lobbies for increased appropriations for the agency. From the CRO perspective, we would like to see increased appropriations for the FDA in three primary areas: clinical trial innovation; regulatory science; and international programs. Our primary concern is that the FDA, like most regulators, is very risk averse unless provided specific direction by Congress. This is one reason why ACRO is very interested in participating in Representative Upton’s 21st Century Cures discussions around driving more innovation within the FDA.  
 
CP: Where does the FDA currently stand on inspections of foreign clinical trials?
 
KN: The FDA has an enormous task as a global health agency, which is not well understood. This is one reason why ACRO strongly supports increased appropriations for the agency. Remember, from a global perspective, the FDA is responsible for a large share of U.S. food imports as well as the import of APIs and finished drugs into the U.S. So, naturally, the agency’s priority on the drug side is going to be on manufacturing quality much more so than on clinical trials. That said, while we would like to see the FDA play a more active role in inspecting foreign clinical trial sites, the agency is doing a good job of coordinating with the host regulators in foreign countries to ensure that global clinical trials meet the agency’s requirements for data quality and patient protections.