Tufts Center for the Study of Drug Development

About: Tufts Center for the Study of Drug Development is a based out in . It is known for research contribution in the topics: Drug development & Clinical trial. The organization has 78 authors who have published 258 publications receiving 16047 citations.

FDA's risk evaluation and mitigation strategies (REMS): effective and efficient safety tools or process poltergeist?

Abstract: Implementation of REMS began in March 2008 and by mid-2011 close to 200 New Molecular Entities (NMEs) and New Drug Applications (NDAs) (i.e., NMEs plus new doses and formulations of drugs) approved by FDA were required to have a REMS. As the REMS program expands, there has been an increasing chorus of stakeholders who have expressed a range of concerns and criticisms about the program's impact on the health care system. Yet, these impacts still remain underexplored by academic study. The authors conducted a series of qualitative interviews with individuals representing the experiences and opinions of five stakeholder groups involved in various aspects of REMS programs. The groups were comprised of representatives of biopharmaceutical companies, payers, health care providers (HCPs), pharmacists, and patient advocacy organizations. Questions were organized around the following themes: REMS implementation and administration; REMS components (i.e., medication guides, communication plans, and elements to assure safe use); effects on patient access and delivery of care; program outcomes; and, issues specific to each stakeholder group. What was most surprising was not that respondent groups with such divergent perspectives and diverse roles within the REMS program disagreed on certain points, but rather that they agreed on so many points. There was general agreement that the program is burdensome and not an improvement over previous risk management programs. Respondents also concurred almost unanimously that the patient information is weighted much too heavily on the risk end of the risk/benefit scale. Similarly, there was general concern from all the responder groups about uncompensated time and resources expended by HCPs and pharmacists. While some positive aspects were noted, these tended to be viewed as opportunities for improvement rather than actual benefits of the REMS program as currently implemented. As PDUFA V draws ever nearer, it's clear that FDA is attempting to address the program's shortcomings but it is clear that more needs to be done.

Assessing the Performance of the EMEA’s Centralized Procedure: A Comparative Analysis with the US FDA

Abstract: In 1995, the European Medicines Agency (EMEA) implemented the centralized procedure for the approval of human medicines to facilitate marketing authorizations that are valid throughout the European Union (EU). Over the last decade, the European Commission (EC) has promulgated additional regulations, such as exceptional circumstances approvals and orphan designations, to enhance the quality and speed of drug development for products that go through the EMEA’s centralized procedure. These regulations have parallel programs in the US Food and Drug Administration (FDA). The current study examines the EMEA’s centralized procedure and analyzes regulatory review times for products approved from 2000 through 2005. EMEA performance metrics are then compared with those of the FDA for the 71 products approved by both agencies during the study period. Time from application submission to approval, as well as the effect of regulatory initiatives implemented by each regulatory body on drug approval times, are analyzed and compared. The results indicate that the EMEA met its mandated performance goals for review milestones. Whereas mean approval times for products approved by both the EMEA and the FDA were similar (15.8 versus 15.7 months, respectively), there was considerably more variability in FDA approval times. Regulatory designations designed to facilitate access to new medicines were shown to be effective in both the EMEA and FDA. For products approved by both agencies, nearly three times as many were approved first in the United States (52 versus 19 were first approved in the United States and the EU, respectively).

Impact of the new US health-care-reform legislation on the pharmaceutical industry: who are the real winners?

Abstract: Over the past two years, the US pharmaceutical and biotechnology industries were preparing themselves for passage of some type of health-reform legislation with a clear appreciation—and concern— about the enormous impact any law would be likely to have on the structure and viability of the research-based industry. Now, with final passage in March 2010 of the patient Protection and Affordable Care Act and its companion “quick-fix” and budget bill, the Health Care and Education Reconciliation Act, it is a good time to take a look at how the industry fared and assess how the various provisions of the health-care reform bill might affect the industry’s long-term prosperity and growth.

Follow-on drugs and indications: the importance of incremental innovation to medical practice.

Abstract: Over the past several decades, biopharmaceutical innovation has resulted in substantial improvements in medical treatment and care. New medicines, diagnostic tools, and drug–device combinations have increased the length and improved the quality of millions of patients’ lives. Health economists, however, caution that such technologic advances are an important cause of rising healthcare expenditures. Tension has arisen between the drive to stimulate biopharmaceutical innovation on the one hand and the need to bring rising healthcare costs under control on the other. As the biopharmaceutical armamentarium expands, physicians and patients are faced with important choices regarding which innovations to use and when. Similarly, third party payers confront a major challenge deciding which drugs to reimburse, under what kinds of cost-sharing arrangements, and with what formulary restrictions. Biopharmaceutical innovation is comprised of two components. The first is research and development leading to the production of novel treatments and firstin-class medicines. The second is the much more common but equally important creation of incremental improvements over existing therapies leading to the development of follow-on medicines and new uses for existing medicines (ie, supplemental indications). Breakthrough or first-in-class biopharmaceuticals attract the public’s attention, because such drugs may address unmet medical needs or provide treatments for indications in which current therapies are inadequate. Accordingly, payers and policymakers are inclined to view breakthrough medicines favorably, which is typically reflected in the products’ comparatively swift and generous reimbursement. On the other hand, payers may question the value of incremental innovation and follow-on drugs. In some cases, this may be reflected in delays in reimbursement after marketing approval as well as in the imposition of formulary restrictions. A novel therapeutic entity or first-in-class drug can be seen as providing stimulus for the evolution of new classes of drugs. In time, other drugs with similar chemical properties (ie, follow-ons) will likely be approved for marketing for the same or similar indications. Moreover, research suggests that increasingly, follow-on drugs were already in late stages of development when the first-in-class drug was approved. Typically, there is a race among competing developers to be first to market with a new class of compounds. Obviously, only the first approved product will be considered the first-inclass drug; all subsequent approvals will be considered follow-on products. In fact, one may argue that the distinction between breakthrough and follow-on drugs is not particularly meaningful; the development of new products can best be characterized as a race among candidates rather than post hoc imitation. Nonetheless, critics of the drug industry opine that research-based companies devote too many resources to developing and marketing follow-on drugs and indications rather than creating more breakthrough drugs. As a corollary, some critics contend that follow-on research yields drugs with negligible added value. For example, the global alliance Health Action International asserts, ‘‘few medicines on the market are the product of innovation and new research. . . . The industry churns out mostly copycats . . . that offer little or no added therapeutic value over breakthrough drugs.’’ Others argue, however, that follow-on drugs and indications provide therapeutic options, which frequently offer improved safety and efficacy profiles and enhance patient compliance. Wertheimer et al, for example, suggest, ‘‘the availability of a broad range of medicines enables physicians to treat with precision the individual needs of diverse patients and provides options when the first agent used is either ineffective or not tolerated.’’ To evaluate the public health impact of follow-on research and development, one must first consider the Tufts Center for the Study of Drug Development, Tufts University, Boston, MA. *Address for correspondence: Senior Research Fellow, Tufts Center for the Study of Drug Development, Tufts University, 75 Kneeland Street, Suite 1100, Boston, MA 02111. E-mail: joshua.cohen@ tufts.edu American Journal of Therapeutics 15, 89–91 (2008)