Faster approvals?: Trends in the use of FDA’s expedited approval programs for oncology medications.

2017 ◽  
Vol 35 (15_suppl) ◽  
pp. e18270-e18270
Author(s):  
Lisa Ambrosini Vadola ◽  
Monique A Pond ◽  
Ann Winter-Vann ◽  
Robin Whitsell
Keyword(s):  
Health Care Providers ◽  
Fast Track ◽  
Priority Review ◽  
Pharmaceutical Companies ◽  
Care Providers ◽  
Regulatory Pathways ◽  
Oncology Drug ◽  
Accelerated Approval ◽  
Fast Track Designation ◽  
Speed To Market

e18270 Background: With the importance of speed-to-market and addressing unmet needs, pharmaceutical companies have sought accelerated approvals through the Food and Drug Administration (FDA). Introduced with the FDA Safety and Innovation Act (FDASIA) of 2012, Breakthrough Therapy Designation (BTD) has become an important mechanism for approval of serious and life-threatening conditions that do not have adequate therapies. Notably, these pathways have been ill-understood by both pharmaceutical companies and health care providers. This study assessed how BTD and other FDA designations have played a role in the approval of oncology drug marketing applications and evaluated trends in the use of these regulatory pathways. Methods: We analyzed publicly available data on novel oncology drug approvals by the FDA from 2012-2016, including the 4 expedited programs for serious conditions (BTD, Accelerated Approval, Fast Track, and Priority Review). Results: Of the 43 novel oncology drugs approved by the FDA between 2012-2016, 42 used at least 1 of the expedited approval programs, including 65% that used ≥2 programs and 35% that used ≥3 programs. The BTD has been used by 15 of the 43 (35%) approved novel oncology drugs since 2012. The use of the BTD, Accelerated Approval pathway, and Priority Review designation among approved oncology drugs has generally increased each year from 2012-2016, while the use of the Fast Track designation has decreased over the same time period. Conclusions: Companies seeking oncology approvals often use more than one expediting strategy. Alone or in combinations, the BTD, Accelerated Approval, and Priority Review have been shown to play an increasingly important role in oncology drug development. Data collected between 2012-2016 suggest that use of BTD is growing more common, while use of the Fast Track designation has decreased among approved oncology drugs. Additional expedited approval programs have remained steady or increased since the FDA introduced BTD. Based on these observations, we anticipate use of the BTD, Accelerated Approval, and Priority Review designation will grow in future oncology drug applications.

Safety-Related Postmarketing Modifications of Drugs for Hematological Malignancies

2019 ◽  
Vol 143 (1) ◽  
pp. 73-77
Author(s):  
Anat Gafter-Gvili ◽  
Ariadna Tibau ◽  
Pia Raanani ◽  
Daniel Shepshelovich
Keyword(s):  
Hematological Malignancies ◽  
New Drugs ◽  
Priority Review ◽  
Regulatory Review ◽  
Regulatory Pathways ◽  
The Us ◽  
Black Box Warnings ◽  
Accelerated Approval ◽  
Drug Approvals ◽  
Approved Drugs

The prevalence of safety-related postmarketing label modifications of medications for hematological malignancies is unknown. We identified 35 new drugs indicated for hematological malignancies approved by the US Food and Drug Administration between January 1999 and December 2014. Characteristics of supporting trials and safety-related label modifications from approval to December 2017 were collected from drug labels. Regulatory review and approval pathways were also collected. New drug approvals were supported by trials with a median of 167 patients (interquartile range 115–316). All drugs were approved based on surrogate endpoints. Twenty-seven drug approvals (77%) were not supported by randomized controlled trials. All drugs received orphan drug designation, and most were granted fast track designation, priority review, and accelerated approval (83, 74, and 60%, respectively). A total of 28 drugs (80%) had postmarketing safety-related label modifications. Additions to black box warnings, contraindications, warnings and precautions, and common adverse reactions were identified in 31, 11, 77, and 46% of drugs, respectively. Five drugs (14%) were permanently or temporarily withdrawn from the US market. Drugs for hematological malignancies are often approved based on limited evidence through expedited regulatory pathways with incomplete safety profiles. Hematologists should be vigilant for unrecognized side effects when prescribing newly approved drugs.

scholarly journals A Four-Component Framework Toward Patient-Centered, Integrated Mental Healthcare in Kenya

2021 ◽  
Vol 9 ◽  
Author(s):  
Manasi Kumar ◽  
Thomas L. Osborn ◽  
Cyrus Mugo ◽  
Hossein Akbarialiabad ◽  
Osman Warfa ◽  
...  
Keyword(s):  
Mental Health ◽  
Health Care ◽  
Health Care Providers ◽  
Professional Training ◽  
Fast Track ◽  
Mental Healthcare ◽  
Clear Understanding ◽  
Care Providers ◽  
Patient Centered ◽  
Fast Tracking

Background: How can we fast-track the global agenda of integrated mental healthcare in low- and middle-income countries (LMICs) such as Kenya? This is a question that has become increasingly important for individuals with lived experiences, policymakers, mental health advocates and health care providers at the local and international levels.Discussion: This narrative synthesis and perspective piece encompasses an overview of mental health care competencies, best practices and capacity building needed to fast track patient responsive services. In that vein we also review key policy developments like UHC to make a case for fast-tracking our four-step framework.Results: While there is an increasingly global impetus for integrated mental healthcare, there is a lack of clarity around what patient-responsive mental healthcare services should look like and how to measure and improve provider readiness appropriately. Here, our collaborative team of local and international experts proposes a simple four-step approach to integrating responsive mental healthcare in Kenya. Our recommended framework prioritizes a clear understanding and demonstration of multidimensional skills by the provider. The four steps are (1) provider sensitization, (2) continuous supervision, (3) continuous professional training, and (4) leadership empowerment.Conclusion: Our proposed framework can provide pointers to embracing patient-centered and provider empowerment focused quality of care improvements. Though elements of our proposed framework are well-known, it has not been sufficiently intertwined and therefore not been integrated. We think in the current times our integrated framework offers an opportunity to “building back better” mental health for all.

Direct-to-Docs Opioid Marketing

Pained ◽  
2020 ◽  
pp. 141-144
Author(s):  
Michael D. Stein ◽  
Sandro Galea
Keyword(s):  
Health Care Providers ◽  
Direct Marketing ◽  
Pharmaceutical Companies ◽  
Opioid Overdose ◽  
Care Providers ◽  
The Public ◽  
Opioid Prescribing ◽  
Overdose Mortality ◽  
The Relationship ◽  
Overdose Deaths

This chapter addresses the pharmaceutical industry’s direct-to-physician marketing of opioids, a factor that has fueled the current addiction epidemic. A 2019 study sought to understand the relationship between mortality from opioid overdose and the pharmaceutical industry’s direct marketing of opioids to physicians. The study analyzed the association between three factors in every US county: the amount of marketing payments pharmaceutical companies made to physicians, opioid prescribing rates, and the number of overdose deaths. Researchers found that direct marketing of opioids to physicians was associated with increased opioid prescribing rates and increased overdose mortality 1 year after marketing engagements. As such, the national response to the opioid epidemic has focused in part on reducing the number of opioids prescribed by physicians. Additionally, the Physician Payments Sunshine Act promotes financial transparency between pharmaceutical companies and health care providers. By increasing regulation around pharmaceutical direct-to-physician marketing, and by making reports of pharmaceutical company payments to physicians available to the public, states have the potential to reduce overdose mortality.

scholarly journals The Potential Benefit of Expedited Development and Approval Programs in Precision Medicine

2021 ◽  
Vol 11 (1) ◽  
pp. 45
Author(s):  
Ariel Kantor ◽  
Susanne B. Haga
Keyword(s):  
Precision Medicine ◽  
Fast Track ◽  
Unmet Needs ◽  
Drug Approval ◽  
Priority Review ◽  
The Us ◽  
Causes Of Disease ◽  
Accelerated Approval ◽  
Legislative Changes ◽  
Innovative Drugs

Background: Increased understanding of the molecular causes of disease has begun to fulfill the promise of precision medicine with the development of targeted drugs, particularly for serious diseases with unmet needs. The drug approval regulatory process is a critical component to the continued growth of precision medicine drugs and devices. To facilitate the development and approval process of drugs for serious unmet needs, four expedited approval programs have been developed in the US: priority review, accelerated approval, fast track, and breakthrough therapy programs. Methods: To determine if expedited approval programs are fulfilling the intended goals, we reviewed drug approvals by the US Food and Drug Administration (FDA) between 2011 and 2017 for new molecular entities (NMEs). Results: From 2011 through 2017, the FDA approved 250 NMEs, ranging from 27 approvals in 2013 to 46 in 2017. The NME approvals spanned 22 different disease classes; almost one-third of all NMEs were for oncology treatments. Conclusions: As these pathways are utilized more, additional legislative changes may be needed to re-align incentives to promote continued development of innovative drugs for serious unmet needs in a safe, efficacious, and affordable manner.

scholarly journals 1235. On the Edge of Tomorrow: Expedited Regulatory Pathways for Anti-Infective Therapies

2020 ◽  
Vol 7 (Supplement_1) ◽  
pp. S637-S637
Author(s):  
Cem Atillasoy ◽  
Panagiotis Gourlias
Keyword(s):  
Infectious Disease ◽  
Infectious Diseases ◽  
Drug Development ◽  
Orphan Drug ◽  
Fast Track ◽  
Orphan Drug Status ◽  
Priority Review ◽  
Drug Status ◽  
Development Programs ◽  
Accelerated Approval

Abstract Background The FDA has developed expedited review programs and pathways to increase drug development for products that have a major clinical benefit. These programs include: Fast Track, Orphan Drug Status, Accelerated Approval, Priority Review, Breakthrough Therapy (BTD) and Qualified Infectious Disease Products (QIPD). Given the heightened awareness of infectious diseases--and emerging global threats, such as resistant bacteria and Ebola—academia and industry have developed and received approval for 88 new infectious disease agents. The objective of this study was to assess the use of expedited review pathways for the 88 anti-infective agents that were approved between 2001-2020. FDA Expedited Drug Development Programs Methods We analyzed the FDA Drug Approval Database entitled, “Compilation of CDER New Molecular Entity (NME) Drug and New Biologic Approvals” for anti-infective therapies that were approved after 2000. Anti-infective therapies were defined as agents that were used to treat or prevent infectious diseases and include antibiotics, antivirals and antifungals. Our analysis focused on a comparison of the percentage of approved anti-infective agents that used each of the aforementioned designations across 2 decades (2001-2010 & 2011-2020). A drug may have one, none, or multiple of these designations. Results There were significant differences in the percentage of anti-infective agents approved with priority review, fast track and accelerated approval in 2001-2010 compared to 2011-2020 (See Results Figure 1) BTD and QIDP did not exist until 2012, thus preventing comparisons between decades. QIDP • Between 2012-2020, 16 anti-infectives have been approved with QIDP. From 2017-2020, 40% (n=10) of approved anti-infectives had QIDP. Orphan Drug Status: • Between 2017-2020, 32% of anti-infectives approved have the orphan drug designation. Comparison of FDA Expedited Drug Development Programs use between 2001-2010 and 2011-2020 Conclusion Our findings indicate Priority Review and Fast Track use has increased since 2010 among anti-infective products. Additionally, our analyses indicate that since 2017 there has been increased use of Orphan Drug Status and QIDP. However, there has been limited use of Breakthrough Therapy and Accelerated Approvals. These two pathways should be increasingly considered by academia, industry and the FDA to further expedite innovative anti-infective development. Disclosures All Authors: No reported disclosures

scholarly journals Time to inclusion in clinical guidance documents for non-oncological orphan drugs and biologics with expedited FDA designations: a retrospective survival analysis

BMJ Open ◽  
2021 ◽  
Vol 11 (12) ◽  
pp. e057744
Author(s):  
Ryan Rodriguez ◽  
Rachel Brunner ◽  
Samantha Spencer ◽  
Dima M Qato
Keyword(s):  
Survival Analysis ◽  
Fast Track ◽  
Orphan Drugs ◽  
Priority Review ◽  
Guidance Document ◽  
Entire Cohort ◽  
Fda Approval ◽  
Life Threatening ◽  
Guidance Documents ◽  
Accelerated Approval

ObjectivesDrug and biological products that treat rare, serious or life-threatening conditions can receive US Food and Drug Administration (FDA) orphan designation and expedited programme designations (accelerated approval, breakthrough therapy, fast track or priority review) meant to incentivise development. Timely recommendations from guidance documents may encourage more rapid and appropriate use and access to these medicines for serious conditions. We sought to determine time between FDA approval and inclusion in guidance documents for non-oncological orphan products overall and by number and type of expedited programme designations.Design and settingRetrospective survival analysis of non-oncological orphan products with ≥1 expedited designation approved since 1992. In June 2020, PubMed, Turning Research into Practice and Guideline Central databases were searched to identify guidance documents influencing US practice that included each product.Main outcomes and measuresThe primary outcome was time to guidance inclusion, defined as any recommendation on use provided within the recommendation framework used by the guidance document.ResultsAmong 135 included non-oncological orphan products, 97.0% (n=131) were designated with priority review, 49.6% (n=67) fast track, 16.3% (n=22) breakthrough therapy and 14.1% (n=19) accelerated approval. Sixty per cent of products (n=81) received ≥2 designations. Overall, 74.1% (n=100) were included in a guidance document. The median time to inclusion was 2.87 years (IQR 2.21–4.18) for the entire cohort. In survival analyses, guidance inclusion was more likely to occur earlier for products with ≥2 designations (HR, 1.84; 95% CI 1.21 to 2.79) and for those with fast-track designation compared with priority review (HR 1.40; 95% CI 1.02 to 2.0). Of 35 products not included in a guidance document, 54.3% (n=19) were approved in 2018 or later.ConclusionsAmong non-oncological orphan products with priority designations, nearly 3 years had passed between FDA approval and inclusion in any guidance document. These findings suggest that despite efforts to expedite availability, appropriate access to these treatments may be delayed because of the lack of timely guidance on their use in clinical practice.

Expedited development and review pathways for novel oncology drugs.

2021 ◽  
Vol 39 (15_suppl) ◽  
pp. e13578-e13578
Author(s):  
Lisa Ambrosini Vadola ◽  
Ann Winter-Vann ◽  
Miriam Knoll ◽  
Robin Whitsell
Keyword(s):  
Drug Development ◽  
Drug Evaluation ◽  
Stakeholder Involvement ◽  
Priority Review ◽  
Time Interval ◽  
Oncology Drug ◽  
Data Limitations ◽  
Accelerated Approval ◽  
Drug Approvals ◽  
Drug Supply Chain

e13578 Background: Following approval of the Food and Drug Administration (FDA) Safety and Innovation Act in 2012, the FDA launched a 3-year plan to promote innovation, increase stakeholder involvement, secure the drug supply chain, and fund these endeavors by collecting user fees. The FDA created its fourth expedited pathway, Breakthrough Therapy Designation (BTD) to focus on the development and approval of therapies to treat serious and life-threatening conditions that lack adequate therapies. Our 2017 study (ASCO abstract #e18270) identified a trend of increased use of these approaches with oncology drugs, particularly the BTD, which was a nascent pathway at the time. We aimed to reexamine FDA pathway and approval trends in oncology interventions. Methods: We analyzed publicly available data on novel drug approvals by the FDA’s Center for Drug Evaluation and Research (CDER) from 2012-2020 and the 4 expedited pathways (BTD, Accelerated Approval, Fast Track Designation [FTD], and Priority Review). Results: Between 2012 and 2020, CDER approved 380 new chemical entities, including 101 oncology drugs. Due to data limitations, 3 novel oncology biologics (approved by the Center for Biologics Evaluation and Research) and 4 diagnostics were excluded from the analyses. Oncology drugs comprised a mean 27% (range 14-34%) of all approvals from 2012-2020, including 25% from 2012–2016 and 28% from 2017-2020. Of all approved oncology drugs from 2012-2020, 94% (range 82-100%) utilized at least one expedited pathway. Oncology approvals were more likely than non-oncology approvals to have used one or more expedited pathways. Use of these pathways for oncology approvals increased from 2012-2016 to 2017-2020: ≥2 (65% vs 78%) and ≥3 (35% vs 50%) pathways. BTD usage for oncology drugs increased from 35% in 2012-2016 to 57% in 2017-2020, though for 2012-2016, the time interval between awarding the (new) designation and the remainder of development activities must be considered. Presumably because BTD grants additional benefits over FTD, generally the use of FTD for oncology drugs has declined over time. The use of Priority Review and Accelerated Approval has remained the same. Despite the pandemic, for oncology and non-oncology drugs alike, approvals using pathways remained consistent between 2019 and 2020. Conclusions: Efficient FDA review plays an important role in oncology drug development. Since its inception in 2012, BTD has been adopted and expeditiously used, comprising more than half of all novel oncology drug approvals from 2017-2020. Our data show a gradual increase in approvals of drugs granted BTD, which, given the duration of drug development, likely reflects the time between the acceptance of the BTD request and remainder of the pre-submission development activities. As BTD was expressly developed to increase stakeholder interaction and prioritize innovation, the speed-to-adoption and use of this pathway among oncology interventions is promising.

The Effect of Information Disclosure on Industry Payments to Physicians

2020 ◽  
pp. 002224372097210
Author(s):  
Tong Guo ◽  
S. Sriram ◽  
Puneet Manchanda
Keyword(s):  
Health Care Providers ◽  
Information Disclosure ◽  
Unintended Consequences ◽  
Pharmaceutical Companies ◽  
Care Providers ◽  
Online Data ◽  
Heterogeneous Treatment Effects ◽  
State Laws ◽  
Disclosure Laws ◽  
Causal Impact

In 2019, U.S. pharmaceutical companies paid $3.6 billion to physicians in the form of gifts to promote their drugs. To curb inappropriate financial relationships between health care providers and firms, several state laws require firms to publicly declare the payments they make to physicians. In 2013, this disclosure law was rolled out to all 50 states. The authors investigate the causal impact of this increased transparency on subsequent payments between firms and physicians. While firms and physicians were informed of the disclosure regulation at data collection, complete transparency did not occur until the data were published online. The authors estimate the heterogeneous treatment effects of the online data disclosure exploiting the phased rollout of the disclosure laws across states, facilitated by recent advances in machine learning methods. Using a 29-month national panel covering $100 million in payments between 16 antidiabetic brands and 50,000 physicians, the authors find that the monthly payments changed insignificantly, on average, due to disclosure. However, the average null effect masks some unintended consequences of disclosure, wherein payments may have increased for more expensive drugs and among physicians who prescribed more heavily. The authors further explore potential mechanisms that can parsimoniously describe the data pattern.

Malpractice & Negligence: Cal. Supreme Court Clarifies Negligence Provisions under State’s Elder Abuse Act

1999 ◽  
Vol 27 (2) ◽  
pp. 203-203
Author(s):  
Kendra Carlson
Keyword(s):  
Health Care ◽  
Supreme Court ◽  
Health Care Providers ◽  
Elder Abuse ◽  
Skilled Nursing Facility ◽  
Pressure Sores ◽  
Care Providers ◽  
Nursing Facility ◽  
Skilled Nursing ◽  
The Supreme Court

The Supreme Court of California held, in Delaney v. Baker, 82 Cal. Rptr. 2d 610 (1999), that the heightened remedies available under the Elder Abuse Act (Act), Cal. Welf. & Inst. Code, §§ 15657,15657.2 (West 1998), apply to health care providers who engage in reckless neglect of an elder adult. The court interpreted two sections of the Act: (1) section 15657, which provides for enhanced remedies for reckless neglect; and (2) section 15657.2, which limits recovery for actions based on “professional negligence.” The court held that reckless neglect is distinct from professional negligence and therefore the restrictions on remedies against health care providers for professional negligence are inapplicable.Kay Delaney sued Meadowood, a skilled nursing facility (SNF), after a resident, her mother, died. Evidence at trial indicated that Rose Wallien, the decedent, was left lying in her own urine and feces for extended periods of time and had stage I11 and IV pressure sores on her ankles, feet, and buttocks at the time of her death.

Using Technology to Improve Access to Health Care for Culturally and Linguistically Diverse Populations

2012 ◽  
Vol 19 (3) ◽  
pp. 71-76 ◽  
Author(s):  
Pauline A. Mashima
Keyword(s):  
Health Care ◽  
Health Care Providers ◽  
Access To Health Care ◽  
Underserved Populations ◽  
Linguistically Diverse ◽  
Care Providers ◽  
Department Of Health ◽  
Use Of Technology ◽  
Access To Health ◽  
Improve Access

Important initiatives in health care include (a) improving access to services for disadvantaged populations, (b) providing equal access for individuals with limited or non-English proficiency, and (c) ensuring cultural competence of health-care providers to facilitate effective services for individuals from diverse racial and ethnic backgrounds (U.S. Department of Health and Human Services, Office of Minority Health, 2001). This article provides a brief overview of the use of technology by speech-language pathologists and audiologists to extend their services to underserved populations who live in remote geographic areas, or when cultural and linguistic differences impact service delivery.

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