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3.
Am J Law Med ; 47(2-3): 249-263, 2021 07.
Article in English | MEDLINE | ID: covidwho-2062063

ABSTRACT

The posture of American regulation of medicine is negative-we assume that a new drug is unsafe and ineffective until it is proven safe and effective.1 This regulatory posture is a heuristic normative principle, a specific instance of the so-called precautionary principle in public health law.2 It is defensible, if debatable, in many ordinary circumstances.3 But like many normative heuristics, this negative posture may compel suboptimal decision-making in emergencies, where context-specific decisions must be made and a range of unique values may apply.


Subject(s)
Decision Making/ethics , Drug Approval/legislation & jurisprudence , Emergencies , Legislation, Drug/standards , Public Health , Drug Approval/organization & administration , Government , Humans , Legislation, Drug/organization & administration , Politics , United States
4.
JAMA ; 328(11): 1043-1044, 2022 09 20.
Article in English | MEDLINE | ID: covidwho-2058984
9.
Ther Innov Regul Sci ; 56(5): 698-703, 2022 09.
Article in English | MEDLINE | ID: covidwho-2003772

ABSTRACT

The accelerated approval pathway has been criticized recently for employing lower regulatory standards than traditional drug approval, undue delays in withdrawing approvals of drugs for which studies have not confirmed clinical benefit, and confirmatory trials not being pursued with due diligence. This article examines the status of confirmatory studies of drugs approved under the US Food and Drug Administration's (FDA's) accelerated approval program between December 1992 and December 2021. It includes background on the program and provides broader context about the program's performance to date over its 30-year history. Our analysis demonstrates that the accelerated approval program has been largely successful, with half of accelerated approvals converted to traditional approval in a median time of 3.2 years. Furthermore, recent FDA actions show that the agency is appropriately managing the program when a drug approved under accelerated approval fails to confirm a clinical benefit. Any proposed changes to the program should be based on cumulative experience with the program, rather than outliers.


Subject(s)
Drug Approval , Pharmaceutical Preparations , United States , United States Food and Drug Administration
11.
BioDrugs ; 36(4): 431-436, 2022 Jul.
Article in English | MEDLINE | ID: covidwho-1930606

ABSTRACT

Biologics are increasingly vital medicines that significantly reduce morbidity as well as mortality, yet access continues to be an issue even in apparently wealthy countries, such as the USA. While patient access is expected to improve with the introduction of biosimilars, misperceptions in a significant part based on terminology continue to make a sustained contribution by biosimilars difficult. Patients are and will continue to suffer needlessly if biosimilars continue to be impugned. Consequently, it is increasingly urgent that semantics are clarified, and in particular, the implication that interchangeable biologics are better biosimilars dismissed. This paper distinguishes between the real differences between biologics that matter clinically to patients and discusses the actual meaning of a US Food and Drug Administration designation of interchangeability for a biosimilar product. This will help highlight where there is need for further Food and Drug Administration education and which stakeholders likely need that education the most.


Subject(s)
Biosimilar Pharmaceuticals , Biosimilar Pharmaceuticals/therapeutic use , Drug Approval , Humans , United States , United States Food and Drug Administration
12.
Aust Health Rev ; 46(3): 309-315, 2022 Jun.
Article in English | MEDLINE | ID: covidwho-1900757

ABSTRACT

Objective Examine the use of priority and provisional approval pathways by the Australian Therapeutic Goods Administration (TGA) for evaluating new medicines. Methods This observational study assessed all new medicines approved by the TGA between 1 January 2018 and 18 October 2021. It examined how frequently priority and provisional approval pathways are being used, the conditions which the medicines are being approved to treat, how long medicines are spending in the approval pathways, the additional therapeutic value of the medicines being approved through these pathways and how the use of the pathways compares with similar regulatory pathways used by Health Canada. Results The TGA approved 138 new medicines in the time period under study, of which 33 were approved through either the priority or provisional approval pathways. Sixteen were approved to treat cancer. It took the TGA a mean of 130 (95% CI 118, 143) and 144 (95% 101, 188) working days for priority and provisional pathways, respectively. Therapeutic evaluations were available for 16 of these medicines and 11 offered little to no therapeutic gain over existing medicines. There was moderate agreement between the TGA and Health Canada in their use of these pathways (Kappa = 0.5458, 95% CI 0.3900, 0.7016). Conclusions The priority and provisional approval pathways are now being used by the TGA for about one-third of all new medicine approvals. Although the medicines approved in these ways are moving through the review process more quickly than those approved through the standard approval pathway, the majority of these medicines, for which an evaluation of therapeutic value is available, do not offer any substantial additional therapeutic value over existing medicines.


Subject(s)
Drug Approval , Australia , Canada , Cross-Sectional Studies , Humans , Pharmaceutical Preparations
13.
Sci Rep ; 12(1): 7444, 2022 05 06.
Article in English | MEDLINE | ID: covidwho-1890254

ABSTRACT

Emergency vaccine use requires weighing a large number of uncertain risks and possible benefits. In the COVID-19 pandemic, decisions about what evidence is necessary to authorize emergency use have proven controversial, and vary between countries. We construct a simple mathematical model of the risks and benefits of emergency vaccination to an individual, and apply this to the hypothetical scenario of individual decision-making between emergency use of a COVID-19 vaccine without safety and efficacy data, versus waiting for efficacy and safety to be established. Even with conservative modelling assumptions and uncertainty distributions for vaccine efficacy (mean expectation = 17%) and serious adverse event risk (mean expectation = 0.3%), high risk individuals (e.g. those who are elderly and have a household contact with COVID-19) are better off using the 'emergency vaccine' rather than waiting for more information (absolute risk reduction for mortality up to 2%). Very early emergency authorization of vaccines despite very limited data may be the better public health strategy when confronted with a dangerous emerging infectious disease.


Subject(s)
COVID-19 Vaccines , COVID-19 , Drug Approval , COVID-19/prevention & control , COVID-19 Vaccines/adverse effects , Humans , Pandemics/prevention & control , Vaccination/adverse effects
14.
BMJ ; 377: o1282, 2022 05 31.
Article in English | MEDLINE | ID: covidwho-1874536
15.
Naunyn Schmiedebergs Arch Pharmacol ; 395(8): 867-885, 2022 08.
Article in English | MEDLINE | ID: covidwho-1838298

ABSTRACT

The second year of the COVID-19 pandemic had no adverse effect on the number of new drug approvals by the US Food and Drug Administration (FDA). Quite the contrary, with a total of 50 new drugs, 2021 belongs to the most successful FDA years. We assign these new drugs to one of three levels of innovation: (1) first drug against a condition ("first-in-indication"), (2) first drug using a novel molecular mechanism ("first-in-class"), and (3) "next-in-class", i.e., a drug using an already exploited molecular mechanism. We identify 21 first-in-class, 28 next-in-class, and only one first-in-indication drugs. By treatment area, the largest group is once again cancer drugs, many of which target specific genetic alterations. Every second drug approved in 2021 targets an orphan disease, half of them being cancers. Small molecules continue to dominate new drug approvals, followed by antibodies and non-antibody biopharmaceuticals. In 2021, the FDA continued to approve drugs without strong evidence of clinical effects, best exemplified by the aducanumab controversy.


Subject(s)
COVID-19 , Drug-Related Side Effects and Adverse Reactions , COVID-19/drug therapy , Drug Approval , Humans , Pandemics , United States , United States Food and Drug Administration
17.
Lancet ; 399(10331): 1223-1224, 2022 03 26.
Article in English | MEDLINE | ID: covidwho-1815312
19.
20.
Healthc Policy ; 17(3): 81-90, 2022 02.
Article in English | MEDLINE | ID: covidwho-1761263

ABSTRACT

Regulatory and reimbursement decisions for drugs and vaccines are increasingly based on limited safety and efficacy evidence. In this environment, life-cycle approaches to evaluation are needed. A life-cycle approach grants market approval and/or positive reimbursement decisions based on an undertaking to conduct post-market clinical trials that address evidentiary uncertainties, relying on the collection and analysis of post-market data. In practice, however, both conditional regulatory and reimbursement decisions have proven problematic. Here we discuss some of the regulatory implications and unsettled ethical and pragmatic issues, taking lessons from the recent experiences of Israel in rapidly approving the Pfizer-BioNTech COVID-19 vaccine.


Subject(s)
Drug Approval , Insurance, Health , COVID-19/prevention & control , Canada , Drug Approval/legislation & jurisprudence , Humans , Insurance, Health/legislation & jurisprudence
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