MAINTAINING A ROBUST GLOBAL THERAPEUTICS PIPELINE1

Luria provided a worldwide forecast of drug sales in 2016. Oncology drugs are predicted to hold the biggest market share of any drug class, with anticoagulants undergoing explosive sales growth. Antirheumatics, antivirals, antidiabetics, vaccines, and dermatologicals also are expected to show robust growth in sales over the next 4 to 5 years. The number of drugs that will be seeking approval during that time will tax global regulatory mechanisms, he stated. To meet this projected need, the global drug development community will need both more regulators and enhanced regulatory science to improve the efficiency of the regulatory process.

The requirements to approve a drug for marketing can differ across countries. In the EU, pharmaceutical law says that drug approval rests on showing that the balance of benefits and risks is positive, with no mention made of effectiveness, relative effectiveness, or cost-effectiveness. EU law also states that regulators should refuse marketing authorization if quality, safety, or efficacy is not demonstrated sufficiently based on objective criteria, which is aimed at making decisions transparent. EU regulators can grant conditional approvals with a requirement for follow-up measurements, so long as there are adequate data to support such an approval. Luria noted, however, that he does not believe these efforts are succeeding at speeding up drug approvals.

The traditional approach to improving efficiency is through processes such as the EU’s harmonization program, which is knitting together the regulatory systems of 27 member nations. This same type of approach could be tried in other regions, said Luria.

Progressive Approval

EMA has developed a 2015 roadmap (EMA, 2010). One of the items in this roadmap, which according to Luria is quite similar to efforts under way in the United States, Canada, and Singapore, is a staggered or progressive approval pathway that is based on sponsors showing a progressive reduction of uncertainty. The proposed new paradigm calls for progressively authorizing increased indications for a drug as knowledge and investment increase. Through iterative phases of information gathering followed by regulatory evaluation and action, progressive authorization seeks to align regulatory decision making with emerging information on benefits and risks. It seeks to maximize the positive impact of new drugs

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1This section is based on the presentation by Xavier Luria, Head, Safety and Efficacy of Medicines, EMA.



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