Hurry Up and Wait: Is Accelerated Approval of New Cancer Drugs in the Best Interests of Cancer Patients?

2003 ◽  
Vol 21 (20) ◽  
pp. 3718-3720 ◽  
Author(s):  
Richard L. Schilsky
Keyword(s):  
Cancer Patients ◽  
Best Interests ◽  
Cancer Drugs ◽  
Accelerated Approval

scholarly journals Internet Information on Oral Cancer Drugs: a Critical Comparison between Website Providers

2020 ◽  
Author(s):  
Berit Bartmann ◽  
Henriette Schallock ◽  
Clara Dubois ◽  
Christian Keinki ◽  
Bijan Zomorodbakhsch ◽  
...  
Keyword(s):  
Health Literacy ◽  
Oral Cancer ◽  
Cancer Patients ◽  
Cancer Drugs ◽  
Web Based ◽  
Non Profit ◽  
Online Newspapers ◽  
For Profit ◽  
Internet Information ◽  
Source Of Information

Abstract Cancer patients need access to high-quality information, when making decisions about oral cancer drugs. The internet is often used as a source of information published by highly heterogeneous providers. The objective was to evaluate the quality of website providers supplying online information about oral cancer drugs. One hundred websites were analyzed using content-related and formal criteria, selected from three existing evaluation methods used for cancer websites, for medical information (defined by the German Agency for Quality in Medicine), and for the “fact box” tool. A web search by a patient was simulated to identify websites to evaluate. ANOVA was used to assess information provided by non-profit organizations (governmental and non-governmental), online newspapers, for-profit organizations, and private/unknown providers. Content-related quality differences were found between online newspapers and all other categories, with online newspapers ranking significantly lower than for-profit and non-profit websites. As for formal criteria, for-profit providers scored significantly lower than non-profit providers and online newspapers for the aspect of transparency. Internet information on oral cancer drugs published by non-profit organizations constitutes the best available web-based source of information for cancer patients. Health literacy and e-health literacy should be promoted in the public domain to allow patients to reliably apply web-based information. Certification should be required by law to ensure fulfillment of requirements for data reliability and transparency (authorship and funding) before health professionals recommend websites to cancer patients.

Pharmaceutical assistance programs for cancer patients in the era of orally administered chemotherapeutics

2017 ◽  
Vol 24 (6) ◽  
pp. 424-432 ◽  
Author(s):  
Aaron Mitchell ◽  
Benyam Muluneh ◽  
Rachana Patel ◽  
Ethan Basch
Keyword(s):  
North Carolina ◽  
Cancer Patients ◽  
Care Delivery ◽  
Private Insurance ◽  
Standard Of Care ◽  
Drug Manufacturer ◽  
Cancer Drugs ◽  
Oral Agent ◽  
Assistance Programs ◽  
Oral Agents

Introduction The rising cost of cancer drugs may make treatment unaffordable for some patients. Patients often rely on drug manufacturer-administered Pharmaceutical Assistance Programs (PAPs) to obtain drugs and reduced or no cost. The overall usage of PAPs within cancer care delivery is unknown. Methods We included all cancer patients across an academically affiliated, integrated health system in North Carolina during 2014 ( N = 8591). We identified the subset of patients receiving PAP assistance to afford one or more cancer drugs, in order to calculate the proportion of patients receiving PAP assistance, and the retail value of the assistance. Results Among 8591 cancer patients, 215 unique patients submitted a total of 478 successful PAP requests for cancer drugs. 40% of PAP-utilizing patients were uninsured, 23% had Medicaid coverage, 20% had Medicare coverage, 2% were dual Medicare/Medicaid eligible, and 14% were commercially insured. Among all cancer patients who received medical treatment, 6.0% required PAP assistance, whereas 10.6% receiving an oral agent required PAP assistance. The proportion receiving PAP assistance varied substantially by drug, ranging from <1% of patients (e.g. carboplatin, methotrexate) to 50% of patients (e.g. ponatinib, temsirolimus). The majority of the retail value obtained was for oral agents, including $1,556,575 of imatinib and $1,449,633 of dasatinib, which were the two drugs with the highest aggregate retail value. Conclusions A substantial proportion of cancer patients receive private charitable assistance to obtain standard-of-care treatments. This includes patients with federal and private insurance, suggesting an inability of patients to meet cost-sharing requirements.

Costs of administration of intravenous (IV) therapies in early versus late stage breast cancer in a US population

2007 ◽  
Vol 25 (18_suppl) ◽  
pp. 6588-6588
Author(s):  
G. B. Kruse ◽  
M. M. Amonkar ◽  
D. Skonieczny ◽  
G. L. Smith
Keyword(s):  
Breast Cancer ◽  
Cancer Patients ◽  
Late Stage ◽  
Early Stage ◽  
Administrative Claims ◽  
Cancer Drugs ◽  
Breast Cancer Patients ◽  
Related Services ◽  
Specialized Equipment ◽  
Cost Components

6588 Background: There are significant costs associated with IV administration of cancer drugs ranging from adverse events associated with the administration to the need for specialized equipment, supplies and personnel time. This study utilized a novel provider-payer contract database to compare the cost components of providing IV therapy to women with early and late stage breast cancer. Methods: Women diagnosed with breast cancer (ICD-9 code 174) between 01/01/2003 and 05/31/2006 and receiving IV monotherapy were identified from an administrative claims database of >60 multi-specialty medical practices/clinics (additional ICD-9 codes 196–198 used to identify late stage breast cancer). Costs were estimated on a per IV administration visit basis using the allowable amount for a claim which closely represents the actual amount paid to providers. Published literature was used to categorize the various billable cost components. Results: 1,393 early and 828 late stage breast cancer patients receiving any of 11 IV breast cancer drugs were identified. The costs breakdown by category for all drugs and for the 2 most commonly used drugs, per IV administration visit, is presented in the table . Conclusions: Costs associated with administration of IV therapies and other visit-related services constituted more than 36% of total costs for early stage and 41% of total costs for late stage breast cancer patients. These costs represent a significant cost burden to payers. Maximization of valuable resources could be effected by increased use of effective oral therapies for treatment of breast cancer. [Table: see text] [Table: see text]

Timing of US Food and Drug Administration (FDA) cancer drug approvals relative to publication of clinical trial results.

2020 ◽  
Vol 38 (15_suppl) ◽  
pp. 2071-2071 ◽  
Author(s):  
Ali Raza Khaki ◽  
Aakash Desai ◽  
Martin W. Schoen ◽  
Bishal Gyawali ◽  
Eddy J. Chen ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Cancer Drug ◽  
Cancer Drugs ◽  
Cancer Treatments ◽  
Fda Approval ◽  
Anti Cancer ◽  
Accelerated Approval ◽  
Drug Approvals ◽  
Clinical Trial Results ◽  
Careful Assessment

2071 Background: Publication of clinical trial results in peer reviewed literature is essential to inform clinicians regarding the use of new anti-cancer treatments, which often have a low therapeutic ratio and require careful assessment of risks and benefits. Publication of registration trials should precede FDA approval to facilitate evaluation and implementation of new therapies. The timing of trial publication relative to FDA drug approvals has not been systematically investigated. Methods: We collected all FDA drug approvals for a cancer indication between 2000-19. Trials were identified using FDA labels as well as drugs and publications indexed on HemOnc.org. Approvals for generics/biosimilars, non-oncology indications and label revisions without supportive evidence were excluded. Dates of approval, the approval pathway, approval type (new vs expansion), and the first full publication related to the registration were recorded. Trials and approvals were matched using available metadata. We calculated the proportion of drugs approved prior to publication overall and for those receiving accelerated approval (AA). We used logistic regression to compare rates of pre-publication approval by approval pathway and by new vs expanded approval. Results: Among a total of 378 drug approvals, 139 (37%) had pre-publication approval. Of these, the median overall time from approval to publication was 140 days (IQR 64-281 days). For those with approval after publication, median time from publication to approval was 157 days (IQR 72-359 days). The number of drugs approved pre-publication rose by 27% between the first and last quarters of the study period, though, the proportion decreased as more anti-cancer drugs have been approved in recent years (Table). More drugs were approved pre-publication through AA than regular approval (46% vs 34%, OR 1.66 [95% CI 1.03-2.70], p=0.04) and as new approvals vs. expanded approvals (45% vs 32%, OR 1.76 [95% CI 1.15-2.70], p=0.01). Conclusions: A substantial minority of FDA approvals occur before trial results are published, with the odds being higher for drugs receiving AA and for new approvals. Since clinicians rely upon published results to inform risk/benefit decisions, efforts are needed to ensure trial results are published by the time of FDA approval of new cancer drugs and indications. [Table: see text]

scholarly journals Regulatory and clinical consequences of negative confirmatory trials of accelerated approval cancer drugs: retrospective observational study

BMJ ◽  
2021 ◽  
pp. n1959 ◽  
Author(s):  
Bishal Gyawali ◽  
Benjamin N Rome ◽  
Aaron S Kesselheim
Keyword(s):  
Observational Study ◽  
Primary Endpoint ◽  
Clinical Guidelines ◽  
Treatment Guidelines ◽  
Retrospective Observational Study ◽  
Cancer Drug ◽  
Cancer Drugs ◽  
Nccn Guidelines ◽  
Clinical Consequences ◽  
Accelerated Approval

Abstract Objectives To investigate the regulatory handling of cancer drugs that were granted accelerated approval by the US Food and Drug Administration (FDA) but failed to improve the primary endpoint in post-approval trials and to evaluate the extent to which negative post-approval trials changed the recommendations in treatment guidelines. Design Retrospective observational study. Setting FDA and National Comprehensive Cancer Network (NCCN) reports. Included drugs Cancer drugs that received accelerated approval from the FDA and had negative post-approval trials. Main outcome measures Regulatory outcomes, including withdrawal, conversion to regular approval, and no action. Results 18 indications for 10 cancer drugs that received accelerated approval but failed to improve the primary endpoint in post-approval trials were identified. Of these, 11 (61%) were voluntarily withdrawn by the manufacturer and one (bevacizumab for breast cancer) was revoked by the FDA. Of the 11 withdrawals, six occurred in 2021 alone. The remaining six (33%) indications remain on the label. The NCCN guidelines provide a high level of endorsement (category 1 endorsement for one and category 2A endorsement for seven) for accelerated approval drugs that have failed post-approval trials, sometimes even after the approval has been withdrawn or revoked. Conclusion Cancer drug indications that received accelerated approval often remained on formal FDA approved drug labelling and continued to be recommended in clinical guidelines several years after statutorily required post-approval trials showed no improvement in the primary efficacy endpoint. Clinical guidelines should better align with the results of post-approval trials of cancer drugs that received accelerated approval.

scholarly journals Accelerated Approval of Cancer Drugs: Improved Access to Therapeutic Breakthroughs or Early Release of Unsafe and Ineffective Drugs?

2009 ◽  
Vol 27 (26) ◽  
pp. 4398-4405 ◽  
Author(s):  
Elizabeth A. Richey ◽  
E. Alison Lyons ◽  
Jonathan R. Nebeker ◽  
Veena Shankaran ◽  
June M. McKoy ◽  
...  
Keyword(s):  
Orphan Drug ◽  
Phase Ii ◽  
Phase Iii ◽  
Drug Status ◽  
Cancer Drugs ◽  
Phase Ii Trials ◽  
Development Times ◽  
Phase Iii Trials ◽  
Accelerated Approval ◽  
Confirmatory Trials

Purpose Accelerated approval (AA) was initiated by the US Food and Drug Administration (FDA) to shorten development times of drugs for serious medical illnesses. Sponsors must confirm efficacy in postapproval trials. Confronted with several drugs that received AA on the basis of phase II trials and for which confirmatory trials were incomplete, FDA officials have encouraged sponsors to design AA applications on the basis of interim analyses of phase III trials. Methods We reviewed data on orphan drug status, development time, safety, and status of confirmatory trials of AAs and regular FDA approvals of new molecular entities (NMEs) for oncology indications since 1995. Results Median development times for AA NMEs (n = 19 drugs) and regular-approval oncology NMEs (n = 32 drugs) were 7.3 and 7.2 years, respectively. Phase III trials supported efficacy for 75% of regular-approval versus 26% of AA NMEs and for 73% of non–orphan versus 45% of orphan drug approvals. AA accounted for 78% of approvals for oncology NMEs between 2001 and 2003 but accounted for 32% in more recent years. Among AA NMEs, confirmatory trials were nine-fold less likely to be completed for orphan drug versus non–orphan drug indications. Postapproval, black box warnings were added to labels for four oncology NMEs (17%) that had received AA and for two oncology NMEs (9%) that had received regular approval. Conclusion AA oncology NMEs are safe and effective, although development times are not accelerated. A return to endorsing phase II trial designs for AA for oncology NMEs, particularly for orphan drug indications, may facilitate timely FDA approval of novel cancer drugs.

scholarly journals Effects of Herbal Medicine Use on Adherence to Conventional Anticancer Drugs

2020 ◽  
pp. 1-20
Author(s):  
Christine Karungi ◽  
Philip Asiimwe ◽  
Conrad Ssentongo ◽  
Sharon Primah Tuhaise ◽  
Joseph Oloro
Keyword(s):  
Herbal Medicine ◽  
Cancer Patients ◽  
Anticancer Drugs ◽  
Herbal Medicines ◽  
Referral Hospital ◽  
Secondary Outcome ◽  
Cancer Drugs ◽  
Medicine Use ◽  
Regional Referral Hospital ◽  
Anti Cancer

Background: Use of herbal medicines alongside conventional anticancer drugs is common among cancer patients. This may potentially cause reduced adherence to conventional anti-cancer drugs, unpredictable side effects and unknown drug-herb interactions. This in the long run could result in poor clinical outcomes. Aim: This study was conducted to investigate how use of herbal medicines affects adherence to conventional anti-cancer drugs, to determine the proportion of patients using both conventional and herbal anticancer medicines and to identify the common herbal medicines used alongside conventional anti-cancer drugs by patients at the Mbarara Regional Referral Hospital Oncology unit. Methods and Findings: A cross-sectional study was conducted in the oncology clinic of Mbarara Regional Referral Hospital found in Mbarara district, Uganda. Data was collected between 20th March and 20th April 2019 from 122 participants who met the inclusion criteria with subsequent consenting. Our primary outcome was adherence and secondary outcome was to investigate whether cancer patients use herbal medicines alongside conventional anti-cancer drugs. Our study had 122 patients most of them belonging to the Banyankole tribe, 75 (61.5%) being males. Of the 72 (59.02%) patients who used herbal medicine, 40 (55.56%) were males and 66.67% of the herbal users reported relief from herbal medicines. Aloe Vera was the most commonly used herb. Most of the patients 77 (63.1%) showed high adherence, this was greater in the non-herbal users than in the herbal users (COR=1.62) though this was not significant (p=0.399). Conclusion: It is most likely that majority of cancer patients use at least one herb during their course of life. The results did not show a significant relationship between herbal medicine use and adherence to conventional anti-cancer drugs. The high proportion of herbal medicine users calls for more research into the area to provide further information that can help optimize cancer treatment outcomes.

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