The President’s Council of Advisors on Science and Technology held a public meeting on April 4 to discuss the issue of antimicrobial resistance.
Drug safety and innovation director Elizabeth Jungman testified on the need to spur the development of new antibacterial drugs.
Oral Comments before the President’s Council of Advisors on Science and Technology
April 4, 2014
Elizabeth Jungman, Director, Drug Safety and Innovation
The Pew Charitable Trusts
Thank you for the opportunity to comment on the Council's important work to address the urgent threat of antibiotic resistance. My name is Elizabeth Jungman, and I direct drug safety and innovation work at The Pew Charitable Trusts, an independent, nonpartisan research and policy organization. Pew works to advance policies to reduce unnecessary use of antibiotics in food animal production, and to spur the development of new antibacterial drugs for people. My focus today is drug development.
As you already know, we have a pressing need for new antibiotics. Doctors already face patients with untreatable infections and threats such as NDM-1 and CRE hint at the potential of worse to come. Pew's analysis of the antibiotics pipeline1 (which is available on our website and will be updated regularly) suggests some reason for optimism: about 45 new antibiotics were in clinical testing as of March 2014.2 However, many drugs in early phase testing don’t pan out. Perhaps only 1 out of 5 – even 1 in 10 – will reach market.3
To keep up in the race against resistance, newer and better antibiotics will be needed, but scientific, economic, and regulatory barriers all slow the pace of antibiotic drug development. The scientific questions -- like how to get drugs into Gram-negative bacteria -- are hard, and greater investment and coordination are needed to help solve them. We urge the council to consider this.
The GAIN Act, passed in 2012, is a step toward addressing economic barriers, by providing additional exclusivity for some new antibiotics.
A remaining challenge is the regulatory. PCAST, in its 2012 report, recommended an approval pathway for drugs for use in a limited population of patients. This approach has been called Special Medical Use or, applied to antibiotics, LPAD – limited population antibacterial drugs.4 LPAD is intended to aid antibiotic development by making clinical testing more feasible and less expensive. Pew has extensively vetted this concept with drug companies, FDA, providers and payors. Along with the Infectious Disease Society of America, we issued a core set of principles to guide the establishment of such a pathway, including the need for effective labeling to foster judicious use of LPAD products.5 In addition to creating a regulatory pathway for drugs that might not otherwise have one, the narrow indications contemplated by LPAD could allow for premium pricing for high-need antibiotics, which would address economic incentives as well.6 Renewed PCAST support for this type of pathway could help create an option for getting antibiotics to patients who have no other option.
1 “Tracking the Pipeline of Antibiotics in Development,” The Pew Charitable Trusts, March 12, 2014, http://www.pewhealth.org/other-resource/tracking-the-pipeline-of-antibiotics-in-development-85899541556.
2 “Antibiotics Currently in Clinical Development,” The Pew Charitable Trusts, March 12, 2014, http://www.pewhealth.org/other-resource/antibiotics-currently-in-clinical-development-85899541594.
3 M. Hay et al., "Clinical Development Success Rates for Investigational Drugs," Nature Biotechnology 32, no. 1 (2014): 40-51. - See more at: http://www.pewhealth.org/other-resource/antibiotics-currently-in-clinical-development-85899541594#sthash.A3jOuqVa.dpuf.
4 “Report to the President on Propelling Innovation in Drug Discovery, Development, and Evaluation,” Executive Office of the President, President’s Council of Advisors on Science and Technology, September 2012, http://www.whitehouse.gov/sites/default/files/microsites/ostp/pcast-fda-final.pdf.
5 “Core Principles for a Limited Population Antibacterial Drug (LPAD) Pathway,” The Pew Charitable Trusts, September 12, 2013, http://www.pewhealth.org/other-resource/core-principles-for-a-limited-population-antibacterial-drug-lpad-pathway-85899504572.
6 Panelists at a Pew-hosted conference generally agreed that the narrow market established by a limited population pathway sets the stage for premium pricing for antibiotics, possibly as much as $15,000 to $30,000 per course. Although considerably higher than those associated with traditional antibiotics, some argued that this range properly represents the lifesaving value of high-need antibiotics and aligns better with judicious use goals. “A New Pathway for Antibiotic Innovation,” The Pew Charitable Trusts, June 2013, http://www.pewhealth.org/uploadedFiles/PHG/Content_Level_Pages/Issue_Briefs/AntibioticsReport.pdf.