An Economic and Regulatory Analysis of Breast Cancer Drugs Approved by the US Food and Drug Administration

Introduction Breast cancer is the most common cancer among women and the leading cause of cancer death among women worldwide. The pharmacological options for breast cancer include chemotherapy, hormone therapy, targeted therapy, and immunotherapy, which are used for the prevention or treatment of breast cancer. This study assessed trends in FDA approvals and prices at the market entry of new drugs indicated for breast cancer in the period 1980-2022. The study also evaluated the factors associated with the price of the new breast cancer drugs at market entry. Material and Methods Regulatory data were collected from the FDA website, and the wholesale acquisition cost (WAC) at market entry from the IBM Micromedex Redbook. We estimated the cost per year or per treatment as defined on the FDA-approved drug label. The WAC was adjusted to 2022 dollars using the consumer price index. Descriptive statistics and generalized linear model regression analysis were conducted. Results As of December 31, 2022, the FDA approved 30 drugs including 23 new molecular entities and 7 new biologics, with 60 indications for different stages of breast cancer and 71 indications for other diseases. The FDA approved 22 (75.9%) drugs using a priority review designation and 5 (17.2%) were granted orphan designation. The median of the inflation-adjusted WAC treatment cost at market entry was higher for drugs approved for advanced and metastatic stages of breast cancer (n=42, median=$88,019, interquartile range (IQR)=$148,969) than those approved for early stages of breast cancer (n=18, median=$51,150, IQR=$141,203). The price of breast cancer drugs at market entry was positively associated with the stage of cancer (specifically, stage 4), approval date, priority review designation, and HER2-positive or TNBC breast cancer subtypes. Conclusions The FDA approved a large number of drugs indicated for the treatment of different types of breast cancer in the period 1980-2022. The CPI-adjusted WAC treatment cost at market entry significantly increased in the period of analysis. Drugs for advanced cancer states, priority review designation, and for specific cancer subtypes were associated with higher treatment costs

Role of Interventional Radiology in Management of Gastrointestinal Bleeding

Gastrointestinal bleeding is a common and potentially life-threatening condition that requires prompt and effective management. Interventional radiology has emerged as a valuable tool in the management of gastrointestinal bleeding, offering minimally invasive techniques that can rapidly control bleeding and improve patient outcomes. This review aims to provide an overview of the role of interventional radiology in the management of gastrointestinal bleeding, including its various techniques and their efficacy. The review discusses the different interventional radiology procedures that can be used to diagnose and treat gastrointestinal bleeding. It also highlights the advantages of techniques used in evaluation and management, including their ability to localize and control bleeding, as well as their low complication rates and shorter recovery times compared to traditional surgical approaches. Furthermore, the review addresses the specific indications for interventional radiology in the management of gastrointestinal bleeding, as well as the role of interventional radiology in the setting of underlying conditions. Overall, this review provides a comprehensive overview of the role of interventional radiology in the management of gastrointestinal bleeding, highlighting its effectiveness and potential benefits for patients. It also emphasizes the need for further research and collaboration between interventional radiologists and gastroenterologists to optimize the use of these techniques in clinical practice

Parental knowledge of RVS infection and attitude to infant immunization with monoclonal antibodies in western region, Saudi Arabia

Background: Human Respiratory Syncytial Virus (RSV) is a single-stranded (RNA) virus belongs to genus orthopneumovirus. It accounted as one of the main causes of lower respiratory tract infection in the pediatric age group and associated with their hospitalization and morbidity. Prophylactic monoclonal antibodies help in improvement and reduction of the serious complications resulting from the virus. Objective : The aim of this paper is to assess parental knowledge of RSV infection and attitudes to infant immunization with monoclonal antibodies. Method: A cross-sectional study conducted using online questionnaire from February 2023 to June 2023 in western region in the Kingdom of Saudi Arabia. A total of 606 of participants, which include any parents or caregiver who believe in childhood vaccination in western region of Saudi Arabia with exclusion of any parents who hesitate towards or refuse childhood vaccinations and outside western region. The statistical analysis done using IBM SPSS. Results: The study included 606 participants from the western province of Saudi Arabia, 218 (36%) were in the age group of 20-30 years, 383 (63.2%) had bachelor degrees And 77(12.7) works in healthcare, RSV was the least known childhood infectious agent (48.7%) of the participants never heard of it. 542 (89.4%) had a positive attitude toward childhood vaccinations in general and (51.2%) toward RSV vaccination with (75.2%) concerned about its safety.Conclusion: Despite the fact that practically all children were exposed to RSV, a sizable percentage of parents never heard of it. We propose that dependable healthcare professionals provide evidence-based information regarding RSV and its safety, effectiveness, and duration of protection against RSV for parents

Evolving trends in the management of acute appendicitis during COVID-19 waves. The ACIE appy II study

Background: In 2020, ACIE Appy study showed that COVID-19 pandemic heavily affected the management of patients with acute appendicitis (AA) worldwide, with an increased rate of non-operative management (NOM) strategies and a trend toward open surgery due to concern of virus transmission by laparoscopy and controversial recommendations on this issue. The aim of this study was to survey again the same group of surgeons to assess if any difference in management attitudes of AA had occurred in the later stages of the outbreak. Methods: From August 15 to September 30, 2021, an online questionnaire was sent to all 709 participants of the ACIE Appy study. The questionnaire included questions on personal protective equipment (PPE), local policies and screening for SARS-CoV-2 infection, NOM, surgical approach and disease presentations in 2021. The results were compared with the results from the previous study. Results: A total of 476 answers were collected (response rate 67.1%). Screening policies were significatively improved with most patients screened regardless of symptoms (89.5% vs. 37.4%) with PCR and antigenic test as the preferred test (74.1% vs. 26.3%). More patients tested positive before surgery and commercial systems were the preferred ones to filter smoke plumes during laparoscopy. Laparoscopic appendicectomy was the first option in the treatment of AA, with a declined use of NOM. Conclusion: Management of AA has improved in the last waves of pandemic. Increased evidence regarding SARS-COV-2 infection along with a timely healthcare systems response has been translated into tailored attitudes and a better care for patients with AA worldwide

Awareness of Parents about the Side Effects of Phenobarbital and the Management of Epileptic Children, Taif City, Saudi Arabia: A Cross-Sectional Study

Many children were suffering from neurological problems. One of these neurological problems is epilepsy, which causes seizures. Children with seizures were managed with different drugs. A drug like phenobarbital is used mainly for epilepsy. This study aimed to assess the awareness of parents about the side effects of phenobarbital and the management of epileptic children, describe the clinical presentation of children with epilepsy and assess the knowledge of parents about the management of epileptic children. A descriptive cross-sectional study was conducted among parents of children diagnosed with epilepsy. Online questionnaire survey distributed to parents of children with epilepsy. A total of 200 parents were included. The age group is 31-40 years old, 72(36%). Parents' knowledge about epilepsy was 98(49%). There were 104(52%) parents who reported seizures caused by low doses of Phenobarbital led to a loss of consciousness. Non-compliance with the required dose occurred majorly due to forgetting the dose 102(51%). The number of children with epilepsy was significantly associated with the awareness level of parents (P=0.0001). In addition, the awareness of parents was significant at the time of epilepsy diagnosis of children (P=0.01). An inadequate awareness among parents regarding the side effects of Phenobarbital, and there were gaps in their knowledge regarding other aspects of the condition and its management

Association of Polymorphism of the Methyl Tetrahydrofolate Reductase (MTHFR) Gene with Anti-Seizure Medication Response in Pediatric Patients in Jeddah, Saudi Arabia

Background and Objectives: Epilepsy is a chronic brain disease, with inherent and noninherent factors. Although over 20 anti-seizure medications (ASMs) are commercially available, nearly one-third of patients develop drug-resistant epilepsy. We evaluated the association between the clinical features and the methyl tetrahydrofolate (MTHFR) rs1801133 polymorphism and ASMs response among pediatric patients with epilepsy. Materials and Methods: This was a multicenter, retrospective, case–control study of 101 children with epilepsy and 59 healthy children in Jeddah. The MTHFR rs1801133 polymorphism was genotyped using the real-time polymerase chain reaction TaqMan Genotyping Assay. Results: Among the patients with epilepsy, 56 and 45 showed good and poor responses to ASMs, respectively. No significant genetic association was noted between the single-nucleotide polymorphism (SNP) rs1801133 within the MTHFR gene and the response to ASMs. However, a significant association was noted between reports of drug-induced toxicity and an increase in allele A frequencies. The MTHFR rs1801133 genotype was significantly associated with the development of electrolyte disturbance among good and poor responders to ASMs. Conclusions: This is the first pharmacogenetic study of MTHFR in patients with epilepsy in Saudi Arabia that found no significant association between the MTHFR SNP rs1801133 and gene susceptibility and drug responsiveness. A larger sample size is needed for testing gene polymorphisms in the future

Assessment of the Medication Administration Errors in the Tertiary Hospital in Saudi Arabia: A prospective Observational Study

Medication errors can occur at any of the three steps of the medication use process: prescribing, dispensing and administration. Drug administration errors were the second most frequent type of medication error, after prescribing errors, but the latter were often intercepted; hence, administration errors were more probably to reach the patients. Therefore, this study was conducted to determine the frequency and types of drug administration errors in a Taif hospital ward. Prospective study based on a disguised observation technique in nine wards in a general hospital in Taif, Saudi Arabia (800 beds). A pharmacist accompanied nurses and witnessed the preparation and administration of drugs to all patients during the three drug rounds on each of six days per ward. The main outcomes were the number, type and clinical importance of errors and associated risk factors. The drug administration error rate was calculated. Relationships between the drug dose frequency, dosage form and types of medication administration error were measured. A total of 7105 medications administered by 250 nursing staff members to 700 patients were observed. Observers intervened in seven administrations. There are 1769 medication administration errors confirmed. The most common medication administration errors were drug preparation error (40.56%, n =727) then, improper dose error (18.58%, n=333); the most common drug class error was Antibiotic (38.9%, n =399) then Analgesic and anti-inflammatory drugs (17%, n =176). The most drug dose frequency had Drug preparation error was seen in a drug used three times a day by 484. MAEs were more likely to occur in the evening shift compared to the morning and afternoon shifts. The study indicates that the frequency of drug administration errors in developing countries such as Malaysia is similar to that in developed countries

MmpL3 Inhibition as a Promising Approach to Develop Novel Therapies against Tuberculosis: A Spotlight on SQ109, Clinical Studies, and Patents Literature

Tuberculosis (TB) is accountable for considerable global morbidity and mortality. Effective TB therapy with multiple drugs completes in about six months. The longer duration of TB therapy challenges patient compliance and contributes to treatment collapse and drug resistance (DR) progress. Therefore, new medications with an innovative mechanism of action are desperately required to shorten the TB therapy’s duration and effective TB control. The mycobacterial membrane protein Large 3 (MmpL3) is a novel, mycobacteria-conserved and recognized promiscuous drug target used in the development of better treatments for multi-drug resistance TB (MDR-TB) and extensively drug-resistant TB (XDR-TB). This article spotlights MmpL3, the clinical studies of its inhibitor (SQ109), and the patent literature. The literature on MmpL3 inhibitors was searched on PubMed and freely available patent databases (Espacenet, USPTO, and PatentScope). SQ109, an analog of ethambutol (EMB), is an established MmpL3 inhibitor and has completed Phase 2b-3 clinical trials. Infectex and Sequella are developing orally active SQ109 in partnership to treat MDR pulmonary TB. SQ109 has demonstrated activity against drug-sensitive (DS) and drug-resistant (DR) Mycobacterium tuberculosis (Mtb) and a synergistic effect with isoniazid (INH), rifampicin (RIF), clofazimine (CFZ), and bedaquiline (BNQ). The combination of SQ109, clofazimine, bedaquiline, and pyrazinamide (PZA) has been patented due to its excellent anti-TB activity against MDR-TB, XDR-TB, and latent-TB. The combinations of SQ109 with other anti-TB drugs (chloroquine, hydroxychloroquine, and sutezolid) have also been claimed in the patent literature. SQ109 is more potent than EMB and could substitute EMB in the intensive stage of TB treatment with the three- or four-drug combination. Developing MmpL3 inhibitors is a promising approach to fighting the challenges associated with DS-TB and DR-TB. The authors foresee MmpL3 inhibitors such as SQ109 as future drugs for TB treatment

PREVALENCE OF SICKLE-CELL DISEASE IN SAUDI ARABIA: A SYSTEMATIC REVIEW

Background: A series of hemoglobinopathies known collectively as sickle cell disease (SCD) contain abnormalities in the gene encoding the beta component of haemoglobin. There are other subcategories that fall under the SCD umbrella, including sickle cell disease (SCD), haemoglobin SC disease (HbSC), and haemoglobin sickle-beta thalassemia (beta-thalassemia positive or beta-thalassemia negative). The prevalence of SCD varies greatly across Saudi Arabia, with the Eastern province having the greatest frequency and the southern regions having the second-highest prevalence. The reported sickle-cell prevalence ranges from 2% to 27%, and in some regions, up to 2.6% of people will have SCD. Objectives: The study aims to summarize current evidences regarding Prevalence of Sickle-Cell Disease in Saudi Arabia. Methods: For article selection, the PubMed database and EBSCO Information Services were used. All relevant articles relevant with our topic and other articles were used in our review. Other articles that were not related to this field were excluded. The data was extracted in a specific format that was reviewed by the group members. Conclusion: Although the prevenance of sickle cell anemia is relatively high due to multiple reasons such as consanguinity, the prevalence of genetic diseases in Saudi Arabia may be significantly lowered during the following decades as a result of premarital screening there. Also, acute chest syndrome in SCD patients is relatively infrequent in Saudi Arabia's Eastern Province, it nonetheless has a major impact on morbidity and death. If patients with African haplotypes are compared, it has a low prevalence and recurrence