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The EU's Innovative Medicines Initiative is helping to invigorate new drug development, but there are still many hurdles to tackle before its promise is realized.
Developing new medicines is hard enough. Accessing schemes to promote the development of new medicines can prove even harder.
European funding schemes for research have often been too cumbersome and unapproachable for researchers to cope with. When the European Union ran a check in preparation for its $100 billion research support program due to start in 2014, it was assailed with complaints about complex application procedures and overbureaucratic financing rules.
Those same concerns have been echoed in schemes aimed specifically at medicines. In early 2012, Europe's pioneering public/private partnership in drug research revised its own procedures in an attempt to allow researchers more time in the lab, by releasing them from excessive form-filling. The Innovative Medicines Initiative (IMI) is improving the financial conditions for scientific teams bidding for funding, and simplifying management requirements. It claims that the new rules ensure more flexible and efficient use of funding.
The details of the changes can be found on the IMI website (www.imi.europa.eu), but they are—as might be expected from a program with a heavy industry involvement—intensely pragmatic. For instance, actual indirect overhead costs can now be reimbursed, whereas previously the limit was a 20 percent flat rate. Funding personnel costs is being simplified, for instance by allowing the use of average personnel costs. Reporting and accounting procedures have been changed to cut the administrative burden and associated costs. And additional simplifications in scientific reporting procedures are in the pipeline, too.
The pragmatism also comes from hands-on experience and direct feedback. In its first two years of operation, IMI has backed 23 projects. This $2 billion program, with its unique blend of significant public finance from the EU and expertise from the European pharmaceutical industry, has already shown it can deliver. The latest evaluation, by a panel of independent experts, was very positive. "Europe has succeeded in establishing a new business model between public and private sectors which unites research strengths across European pharmaceutical industry, academia, and small to medium enterprises," it said. "To have formed and embedded this new, applied research environment is a significant achievement for Europe."
The compliments went even further. IMI's development "has been meaningfully enhanced by its engagement with the regulatory authorities and patient organizations," and "to have succeeded here is rare, and taken together with the scale of interest of research organizations is a tremendous illustration of Europe's strengths in creating consensus and collaboration."
The achievements are more than just political, however. This is a scientific program that is starting to show scientific results. Diabetes treatment is one area that stands to benefit; a project led by a team at the UK's Imperial College has provided the first demonstration in humans that the PAS kinase protein plays a key role in insulin secretion. Chronic pain is being explored in another project, and schizophrenia and depression are being investigated through new experimental models.
New tools are under development to test potential treatments of Alzheimer's disease, bringing together databases of previously conducted clinical trials and combining the results from blood tests, brain scans, and behavioral tests. Innovative testing methods to classify asthma patients are expected to play into new drug development, and new assessment techniques are emerging for chronic obstructive pulmonary disease. Work is under way on proof of concept that early biomarkers can reliably and robustly predict later cancer development.
The range of supported actions extends beyond specific therapeutic categories to cover the entire research process. The search is on for sensitive and specific clinical tests to diagnose and monitor drug-induced injury to the kidney, liver, and vascular systems in man, so as to overcome one of the major current hurdles in drug development. One project provides advanced training on subjects such as safety, pharmacology, ethics, and non-clinical safety assessment, or predictive cell culture systems. Another is creating a pan-European platform for education and training across the entire lifecycle of medicines research, from basic science, through clinical development, to pharmacovigilance. Doctoral-level training in pharmacovigilance and pharmacoepidemiology for specialists and non-specialists also started in late 2011.
IMI is promoting standardized management methods for clinical and medical research data through an agreement with the Clinical Data Interchange Standards Consortium (CDISC). As IMI executive director Michel Goldman observed, "Since IMI projects will be increasingly based on the analysis of large data sets, harmonization and standardization of data made possible through the collaboration with CDISC will be critical to success." And to promote collaboration, it provides an online partner search tool for people, organizations, and enterprises interested in participating in its projects.
The evaluation processes that IMI uses for project selection have won approval from independent observers. External monitors found the latest assessment of expressions of interest was conducted "professionally and fairly," and with "dedication of all participants to ensuring an impartial and thoughtful evaluation."
Upcoming projects include the creation of a European compound library where the findings of participating pharmaceutical companies will be pooled, and the establishment of a European screening center with novel tools to improve procedures and pick up molecules that might otherwise be missed.
But perhaps IMI's biggest challenge in 2012 will be to drive forward a wider EU program in tackling resistance to antibiotics scheduled to get under way in the coming months. IMI's role in the short term will be to build partnerships that can speed discovery and development of novel antibiotics to treat the most urgent infections. Over the longer term, IMI could become the focal point for large-scale collaboration between European health authorities and the pharmaceutical industry, based on novel partnership arrangements for drug development in antibiotics and, further ahead, across a broad range of therapeutic needs.
There are plenty of other challenges ahead where IMI could prove a valuable resource for Europe. In particular, the known unknowns—and even more so, the unknown unknowns—of personalized medicine will demand a new capacity for research cooperation in Europe if the potential is to be exploited. Unprecedented collaboration between therapeutic and diagnostic approaches is just one of many obvious areas that could benefit. IMI is, in principle, well placed to provide that form of cross-sector support, and it has already set additional priorities in its scientific agenda for pharmacogenetics and taxonomy of human diseases, and for rare diseases and stratified therapies.
Time may not, however, be on IMI's side. Agreement in Europe on the need for a strong base for medicines research is unambiguous. What is somewhat ambiguous is how this is to be secured. There is, for instance, still no consensus on a further round of funding once IMI's initial $2 billion is exhausted—and the current economic climate in Europe is hardly propitious for publicly funded programs, even when industry puts in its own contribution too. IMI still has to come up with satisfactory solutions to the intellectual property questions attached to innovation, so that future business or research opportunities are not limited by uncertainty or inequity.
Meanwhile, there are signs that drug development in the U.S.—still Europe's biggest competitor in this field—may be recovering from its decade-long slowdown. The FDA approved 35 new medicines in 2011—one of the highest totals since the turn of the century. And these were not just lifestyle-inspired me-too products; around half were for cancer or orphan diseases. Strikingly, two of the new medicines were linked with a molecular test, making them the first companion drug-diagnostic approvals. IMI's aim of invigorating European drug research is admirable, and its achievements to date are substantial. But it will have to do even more, even faster, if it is to win the support it needs in order to be able to give the backing to European drug research that is also crucially needed.