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United States Medical Device Regulatory Framework
Published in Jack Wong, Raymond K. Y. Tong, Handbook of Medical Device Regulatory Affairs in Asia, 2018
In accordance with section 513(f )(1) of the FD&C Act, devices that were not in commercial distribution before May 28, 1976 (the date of enactment of the Medical Device Amendments of 1976), generally referred to as “postamendment” devices, are classified automatically by statute into class III without any FDA rulemaking process. These devices remain in class III and require a PMA unless the device is classified or reclassified into class I or II. However, in 1997, the Food and Drug Administration Modernization Act (FDAMA) added the De Novo classification pathway under section 513(f )(2) of the FD&C Act, establishing an alternate pathway to classify new devices into Class I or II that had automatically been placed in Class III after receiving a Not Substantially Equivalent (NSE) determination in response to a 510(k) submission. Subsequently, in 2012, the FD&C Act was amended once again by the Food and Drug Administration Safety and Innovation Act (FDASIA), to provide a second option for De Novo classification. In this second pathway, a sponsor who determines that there is no legally marketed predicate may request a risk-based classification of their device and submit a De Novo request without first receiving an NSE determination via a submitted 510(k).
Regulatory and Legislative Policy and Science Considerations in the Era of Patient-Centeredness, Big Data, and Value
Published in Qi Jiang, Weili He, Benefit-Risk Assessment Methods in Medical Product Development, 2017
Tarek A. Hammad, George A. Neyarapally
The Food and Drug Administration Safety and Innovation Act (FDASIA) of 2012, including the PDUFA V reauthorization, included several provisions relevant to drug development and evaluation in the context of B–R assessment.80 The law created a new breakthrough therapy pathway that enables drugs with preliminary evidence of a substantial benefit to be approved81—this is analogous to previous authorities designed to achieve the same purpose, including accelerated approval, but enhances FDA’s ability to communicate with and support the industry in obtaining approval for drugs with early evidence of substantial benefit. This also introduces challenges in the evaluation of the benefits and risks of these drugs in light of the limited evidence available at the time of approval.82 Since its inception, many applications for such drugs have been received and some drugs have been approved as breakthrough therapies. In fact, FDA already received 92 requests for breakthrough therapy status by September 2013 and granted the designation for approximately 29% of these requests.83 This dovetails with the increasing number of orphan drugs and medical products approved in recent years as both of these types of drugs tend to have limited evidence on benefits and risks at the time of approval in light of the small target patient populations in many cases.
Towards precision medicine
Published in Yann Joly, Bartha Maria Knoppers, Routledge Handbook of Medical Law and Ethics, 2014
The Food and Drug Administration Safety and Innovation Act was signed into US law on 9 July 2012. It allows drugs to enter an accelerated development and approval pathway if ‘preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints’ (Reynolds 2013). As such, the development of new drugs targeting serious and life-threatening diseases has seen their development accelerated by approaches based on PGx.
Patent landscape of pediatric-friendly oral dosage forms and administration devices
Published in Expert Opinion on Therapeutic Patents, 2021
Christina Karavasili, Angelos Gkaragkounis, Dimitrios G. Fatouros
Age stratification within the pediatric patient population is crucial during the design of age-appropriate formulations and administration devices for optimal drug delivery. EMA encourages the commercialization of such products via a reward system for extended patent protection. However, the effectiveness of the rewards is highly dependent on factors, such as the limited number of patients, the difficulty in recruiting pediatric subjects and the continued off-label use of comparable and lower cost medicinal products, as indicated by an in-depth cost-benefit assessment report analyzing the economic impact of these regulations in Europe [174,175]. The current legislation related to pediatric product development in the United States is the Best Pharmaceuticals for Children Act (BPCA) that was first enacted in 2002 and permanently reauthorized in 2012 by the Title V of the Food and Drug Administration Safety and Innovation Act (FDASIA) [176]. BPCA provides financial incentives to sponsors who voluntarily complete pediatric clinical studies, outlined in an FDA-issued Written Request, in the form of additional marketing exclusivity. This exclusivity provision is a 6-month extension of an existing patent or an additional 6-month marketing exclusivity for the entire moiety and has proven effective in encouraging pediatric clinical testing of drugs in order to improve their safety and dosing information [177]. On the other hand, in Japan, pharmaceutical companies are granted premiums as rewards for pediatric-drug development, the effect of which translates to the drug’s price not being reduced every 2 years, as normally occurs within the Japanese Health System [178,179].
Drug evaluation studies in neonates: how to overcome the current limitations
Published in Expert Review of Clinical Pharmacology, 2018
Karel Allegaert, Anne Smits, John N van den Anker
Stepwise and over the last decades, the United States, European Union, and World Health Organization developed legal initiatives to improve children’s health through improving access to safe and effective medicines for children [6]. These initiatives have resulted in a relevant and significant increase in pediatric studies. Because of the poor performance of these initiatives in neonates, additional initiatives like the Food and Drug Administration Safety and Innovation Act (FDASIA) were initiated to generate more neonatal drug research initiatives [7]. At present, studies in infants and in newborns cover 3 and 2% of registered studies, respectively [8]. Unfortunately, Stiers and Ward concluded that only a very small number of label changes in children (1997–2010, FDA) were related to neonates (24/406, 6%). In addition to the small number of studies, the authors also had to conclude that the labels for neonates were not changed despite the high a priori likelihood that drugs like caspofungin, intravenous acetaminophen, or valganciclovir are relevant to neonates. In their assessment, this was due to insufficient reflection on neonatal pathophysiology, including inaccurate dose selection and failure to document efficacy or safety. These reflect different types of limitations of the approaches used to document drug safety and efficacy in neonates [9]. This setting is not unique in pediatric product development, but most prevalent in neonates: 42% of studies in children (submitted to the FDA 2007–2014) failed to document safety (16%) or efficacy (86%) due to failure to sufficiently consider the differences between adult and pediatric disease entities (n = 8) or using inaccurate doses (n = 10) [10]. Benjamin et al. reported that only 31/253 (12%) of studies conducted within the framework of the pediatric exclusivity (1998–2004) in the US included newborns, limited to sedation/pain (4 studies) infectious diseases (10 studies) or gastro-intestinal diseases (8 studies) as therapeutic areas [11]. These analyses clearly illustrate that there is more to do than just increasing the number of studies or research volume [7].