Press Release

Statement: Alternative Approval Pathway for Certain Drugs

PhRMA March 1, 2013

WASHINGTON, D.C. (February 28, 2013) – Pharmaceutical Research and Manufacturers of America (PhRMA) Vice President of Scientific & Regulatory Affairs Sascha Haverfield issued the following statement today regarding the proposal to create a potential new drug approval pathway intended to expedite the development of new medicines for serious or life-threatening conditions that would address an unmet medical need, including medicines for serious or life-threatening infections caused by antibiotic-resistant bacteria:

“PhRMA is committed to helping ensure that patients have access to safe and effective new medicines, and we recognize the particular importance of developing and securing the approval of new antibiotic and anti-infective medicines that can prevent the mortality and debilitating morbidity associated with emerging or resistant pathogens.  Timely access to new medicines can be a matter of life or death for patients affected by such pathogens.  We have long supported FDA’s appropriate use of innovative approaches and regulatory flexibility when considering the type and quantity of data required to establish the safety and efficacy of these medicines—as well as other innovative medicines to address unmet medical needs. 

“PhRMA believes that existing regulatory frameworks at the FDA are sufficiently flexible to facilitate streamlined development of antibiotics and anti-infectives for serious and life-threatening infections. However, we recognize that the development of new antibiotics and anti-infectives represents an urgent and unique public health challenge that requires special attention. 

“We stand ready to continue our work with FDA and other stakeholders toward establishment of an appropriate and targeted regulatory approach that will accelerate the development and availability of sorely needed novel antibiotic and anti-infective medicines.  FDA could accomplish this goal by initiating a new program using a combination of existing regulatory flexibility granted under the Federal Food, Drug, and Cosmetic Act and voluntary stewardship programs managed by healthcare professionals to address possible resistance issues.

“As FDA continues to explore ways to enhance its use of regulatory flexibility to accelerate development of novel antibiotics and anti-infectives, it will be important to ensure that any new regulatory approach will not result in confusion of expectations or a diversion of resources from existing and proven pathways, as well as from pathways that remain to be implemented.  Such an outcome could hamper access to important therapies for patients in need. 

“Furthermore, any program implemented by FDA should seek to avoid unintended consequences that could harm patient care, such as the possibility of direct or indirect government restrictions on the practice of medicine.

“The dramatic increase in the emergence of antibiotic-resistance bacteria over the last decade is now a public health crisis.  Meanwhile, the number of antibiotics approved in the U.S. has been steadily decreasing in the past two decades and the pipeline for new antibiotics is alarmingly slim.  PhRMA stands ready to work with the agency and other stakeholders to explore effective ways to facilitate antibiotic and anti-infective development and approval through a combination of existing regulatory flexibility, and the strengthening of antibiotic and anti-infective stewardship programs designed and operated by health care providers and health care systems.”

PhRMA’s comments to FDA can be accessed at

About PhRMA

The Pharmaceutical Research and Manufacturers of America (PhRMA) represents the country’s leading innovative biopharmaceutical research and biotechnology companies, which are devoted to discovering and developing medicines that enable patients to live longer, healthier, and more productive lives. Since 2000, PhRMA member companies have invested over $500 billion in the search for new treatments and cures, including an estimated $49.5 billion in 2011 alone.

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