Rare diseases remain a challenge for many of the countries in the world. The millions of people collectively suffering from rare diseases, in the context of raging COVID-19 pandemics globally, require an innovative and recent solution from different stakeholders. Regulatory bodies such as the Food and Drug Administration and the European Medicines Agency have come up with many different approaches including financial assistance to prompt drug development and approval. Novel approaches pertinent to clinical trials of such drugs such as patient centricity, early interaction with regulatory bodies, and establishing clinical outcome of interest have been experimented. Various international organizations including cross-country collaborators have initiated various projects or consortiums to bridge the gap between knowledge and practice. The challenges remain more pivotal in developing countries such as India, which has adopted few noteworthy initiatives by involving relevant stakeholders in the presence of limited resources, infrastructures, and a nascent regulatory framework. Therefore, it is imperative to revisit the key aspects of orphan drug development to fulfill the unmet needs of such patients suffering from various rare diseases.

The New Drugs and Clinical Trial Rules (NDCTR) came in force in 2019, these brought in some changes, but certain earlier parts, such as Rule 122 DAB, were incorporated unchanged. A few sections of the NDCTR are problematic and need revision for the smooth conduct of clinical trials in India. Training of Ethics Committee (EC) members and other stakeholders is a very important driver for clinical research but has not been defined clearly. In addition, some processes of EC review need a relook. Compensation formulae were finalized in 2013; now, the altered economic situation has eroded the value of money. Hence, for the protection of research participants, an urgent review of the compensation formula is suggested.

Aim: World Health Organization (WHO) core prescribing indicators are highly standardized tools in reliably assessing the essential aspects of drug utilization pattern. It is critical that the rational use of drug prescribing is scrutinized for the utmost benefit of patient welfare. In this study, we aim to assess the prescription pattern and prescribing behavior of physicians using the WHO-recommended core prescribing indicators at a teaching hospital in South India. Materials and Methods: A prospective, descriptive cross-sectional study was conducted in the general medicine outpatient department of a tertiary care hospital for a period of 1 month in June 2019. A total of 600 prescriptions were sampled based on the WHO “How to investigate drug use in health facilities” document recommendation. The WHO guidelines and methods were observed to ensure data reliability. Descriptive statistical analyses such as frequencies, percentages, mean, and standard deviation were used to present the data. Results: The WHO core prescribing indicators analysis revealed that the average number of drugs per encounter was 2.38 ± 1.1 and only 796 (55.4%) of the drugs were prescribed by generic name. Whereas, the percentage of encounters prescribed with an antibiotic 44 (7.3%) and an injection 63 (10.5%) was less than the ideal recommendations as per WHO and 1265 (88%) of the drugs were prescribed from the National List of Essential Medicines. Conclusion: This study on prescription pattern audit done using the WHO core prescribing indicators highlights that prescriptions encountered with antibiotic and injection use were in accordance with the WHO recommendations.

Background: Diabetes mellitus is a chronic noncommunicable disease, and hypertension (HT) is the most common comorbidity which affects their quality of life (QoL). Aim: The aim of the study was to assess the effects of antihypertensive agents (viz., amlodipine, ramipril, telmisartan, and ramipril with telmisartan) on the blood pressure (BP) and QoL. Methodology: It was an open-labeled prospective intention-to-treat study done in diabetic hypertensive patients (CTRI/2016/10/007340). Patients were randomly assigned antihypertensive agents, namely, amlodipine, ramipril, telmisartan, and a combination of ramipril with telmisartan (RT) in four groups. They were evaluated for BP, blood sugar level, and QoL at baseline and 24^th week. Results: After 24 weeks of therapy, systolic BP (SBP) and diastolic BP (DBP) were significantly reduced in all groups. In amlodipine, there was a mean percentage fall of SBP by 15.85% (confidence interval [CI]: 21.38–28.13) and DBP by 11.22% (CI: 8.41–12.70); in ramipril – 14.4% (CI: 18.61–25.15) and 12.4% (CI 8.88–13.99); telmisartan – 18.4% (CI: 24.89–10.79) and 14.6% (CI 10.79–16.24); and in RT group, SBP 17.7% (CI: 23.38–29.18) and DBP 12.4% (CI: 9.05–13.02). QoL score increased by 30.56% (CI: 14.30–10.90), 30.94% (CI: 14.21–10.68), 28.07% (CI: 14.89–11.20), and 28.84% (CI: 15.49–11.77), in respective groups (P < 0.0001, each). However, they were nonsignificant between the study groups (P > 0.05). Conclusion: Amlodipine, ramipril, telmisartan, and a combination of RT are equally effective to improve BP and QoL among diabetic hypertensive patients. However, amlodipine and telmisartan lacked in dry cough and more tolerable than the ramipril and RT therapy. Henceforth, amlodipine and telmisartan are better choice to control HT among DM patients.

Context: A failure to obtain continued Institutional Ethics Committee (IEC) approval for the study before the expiry date assigned by the IEC is considered as “lapse of the IEC approval” to continue the study at the site by the Investigator. Considering this, we had conducted an audit of principal investigators (PI's) compliance for Continuing Review Application (CRA) submission timelines and decision taken on the lapses in the validity of IEC approval continuation. Aim: The aim of this study is to assess the pre- and post-policy trends of non-compliance management of delayed CRA submission and compare the PI's compliance for submission of CRA between Investigator Initiated trial (IIT) and Pharma studies. Setting and Design: The present study was a retrospective audit of CRAs of ongoing projects submitted by PIs to IEC, ACTREC. Materials and Methods: The data from total 199 CRAs submitted for review to the IEC between the year January 2016 and December-2017 were collected and maintained in Microsoft Excel sheet, and later, the data were exported into the SPSS software version 21 for the analysis. Statistical Analysis: All categorical data were presented in numbers and percentage. The first primary objective was assessed by calculating the duration between the dates of approval for any study to the date of next CRA submission. The CRAs submitted after the project expiry date were considered as a lapse in following the IEC SOP. Results: This retrospective audit revealed that CRA reminder sent by the IEC to the PI played an important role in compliance w. r. t timely in following the IEC SOPof the CRA by the PI. As a result, overall, 90% of CRAs showed compliance in submitting CRAs to IEC in both IIT and Pharma study. The number of lapses were reduced to 7 in the postpolicy period as compared to 15 lapses in the prepolicy period. Conclusion: This retrospective audit reveals that CRA reminder sent by the IEC to the PI played an important role in improving the compliance of PIs in submitting CRA to IEC. Each IEC should develop the policy to minimize the delays in CRA submission by the PI and prevent lapses in following the IEC SOP.

Purpose: Very few studies conducted in India have analyzed insurance policies and clinical trial agreement (CTA) submitted to ethics committee (EC). This study was conducted to review and find out deficiencies in it. Materials and Methods: This was a retrospective observational study. All the protocols for regulatory clinical trials and academic research sponsored by the Indian Council of Medical Research or other funding agency were included. Insurance documents and CTA submitted with the study protocols were analyzed. Results: A total of seventy CTA and insurance policies were analyzed. CTA mentioned that parties involved in 60 (86%) forms, scope of the agreement in 15 (21%) forms, responsibilities of the party in 68 (97%) forms, and payment details in 58 (83%) forms. Nearly 88.5% of the insurance policies mentioned whether the policy covers the participants for injury due to all clauses and 91% of the policies mentioned the validity period of insurance. Conclusion: It was found that both the documents contained almost all the required elements. This was probably because this institutional EC insisted on and thoroughly reviewed the documents to ensure that adequate compensation of research-related injuries has been provided for and this fact is informed to the trial subject. As very few studies are available in the literature, we could not compare majority of the findings of this study with others.

Background: Nowadays, brand-name drugs are becoming an out-of-pocket expense which comprises 80% of total health-care expenditures. However, generic drugs are less expensive than brand-name drugs with the same therapeutic effect, but many doctors hold negative views of generics and resist prescribing. This study was designed to assess the knowledge, attitude, and practice of doctors toward generic medicines. Methods: This was a questionnaire-based cross-sectional study conducted in a multispecialty private hospital. The study participants were doctors who were practising in a hospital during the study period (January 2017 to July 2017). The questionnaire comprises 35 questions related to demographics, knowledge, attitude, and practice evaluation of generic medicines. Descriptive statistics was applied to represent participant characteristics and response rates. Results: A total of 86 questionnaires were distributed to the doctors and the response rate was 37%. The majority of doctors who participated in this survey perceived that generic medicine is effective, safe, and has same active component, dose, and bioequivalent as the brand medicines. Majority of the doctors (72%) believe that generic drugs were manufactured by poor techniques. However, more than three-quarter of doctors (78%) routinely prescribed generic drugs. Conclusion: Most of the doctors had an honest angle about the efficacy and safety of generic medicine. However, a high proportion of physicians believe that the generic drugs are of poorer quality. To have a better understanding of the generic drug, the doctor must be well informed about the generics products during their academic career that will significantly impact health-care budgets.

Context: The rational prescription leads to a healthy and good-quality life of a patient. Irrational, inappropriate, and unnecessary prescriptions are major therapeutic issues in Bangladesh, which can cause severe consequences. Aim: This cross-sectional study was conducted to evaluate the prescription patterns and errors as well as to review the most frequently prescribed drug classes among outpatients at a secondary hospital in Pabna, a district of Bangladesh. Methods: A total of 400 prescriptions were reviewed from March 2019 to May 2019. In this study, statistical data analysis was implemented by IBM SPSS Statistics V22 and data revealed in frequencies, mean, and percentage. Spearman's rank correlation coefficient was calculated to show the correlation between bivariate coded variables. Results: The results revealed that majority of the prescription were prescribed for females (73.5%) where proton-pump inhibitors (PPIs), analgesics, vitamins, and single antibiotics were most frequently prescribed medicine for the female patients compared to male patients. Almost half of the collected prescription contained four medicines (47%). Maximum number of prescriptions contained two (30.5%) essential drugs and among 1402 medicines of 400 prescriptions, antiulcerants were most frequently prescribed medicine (23.32%) where esomeprazole was highly prescribed generic drug (44.75%). Moreover, Spearman's rank correlation coefficient suggested that PPIs and analgesics were frequently prescribed medicines at a time for the patients (0.182). According to the age group, the study also got some significant variations in prescribing pattern. However, most common prescription errors were prescriber's name not mentioned (100%), diagnosis not mentioned (96.75%), dose not indicated (15.41%), and wrong drug name (0.36%). Conclusion: Findings of the current study represent the existing prescribing trends of different therapeutic classes of drugs and common prescription errors in a secondary health facility of Bangladesh. From this study, it is observed that physicians prescribed rationally in some cases but need to ensure rationality in all prescriptions. Continuous monitoring of drug use, development of prescribing guidelines, and training are recommended to ensure and implement good-quality prescribing practices for promoting the rational and cost-effective use of drugs.

Screening tests are done to diagnose asymptomatic disease in apparently healthy people with the aim to reduce mortality and morbidity from the disease. Certain criteria need to be fulfilled before we adopt population-level screening for any disease. Several biases exist in evaluating screening studies, and the ideal study design would be a randomized trial with hard endpoints such as mortality and morbidity.