Asian representation in clinical trials of new drugs for the treatment of cancer.

2017 ◽  
Vol 35 (15_suppl) ◽  
pp. 6564-6564 ◽  
Author(s):  
Lola A. Fashoyin-Aje ◽  
Laura L. Fernandes ◽  
Steven Lemery ◽  
Patricia Keegan ◽  
Rajeshwari Sridhara ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Far East ◽  
Mainland China ◽  
Asian Population ◽  
The Philippines ◽  
New Drugs ◽  
Asian Patients ◽  
Number Of Patients ◽  
The Us ◽  
Demographic Subgroup

6564 Background: In the US, statistics for Asians are often aggregated with other racial groups. This poses challenges in estimating the cancer burden and in defining cancer clinical trial enrollment targets in this demographic subgroup. ‘Asian‘ refers to persons with origins in the Far East, Southeast Asia, or the Indian sub-continent. Asians comprise 6% of the US population and the largest Asian subgroups in the US are of Chinese (22%), Filipino (19%), Asian Indian (19%), Vietnamese (10%), Korean (9%), and Japanese (7%) descent. The representation of Asian patients in global clinical trials may not be reflective of the Asian subgroups in the US. FDA conducted an analysis to describe patients categorized as ‘Asian’ in clinical trials supporting the approval of new drugs. Methods: We reviewed the marketing applications of 33 new molecular entities approved for the treatment of solid tumor malignancies between 2011- 2016 to identify trials that provided the primary evidence of safety and efficacy. Results: A total of 29,941 patients were enrolled; 17 % were Asian. Most Asian patients were enrolled in Korea (20%), Taiwan (20%), mainland China (20%), Japan (16%), and US (5%). Few patients were enrolled in India (3%); the Philippines (1%); Vietnam (0). In the US, Asian patients comprised 3% of the total number of patients enrolled. Conclusions: Asian patients represented a heterogeneous mix. A large proportion was enrolled in Taiwan (20%) and Korea (20%), whereas the largest proportion of US Asians have origins in mainland China (22%), the Philippines (19%), India (19%), and Vietnam (10%). Nevertheless, although Asians share a common ancestry, it is not clear whether data from global clinical trials are generalizable to Asian patients in the US. Therefore, strategies to improve the enrollment of US Asian patients in clinical trials are needed. Among patients enrolled in the US, 3% were Asians, a proportion that is below US Asian population estimates (6%). While most site-specific cancer incidence and death rates are lower in US Asians compared to Whites, the rates of some cancers (e. g., stomach and liver) are higher in this group. Therefore, studies are needed to determine adequate enrollment targets in this demographic subgroup.

Major lupus organ involvement: severe lupus nephritis

Lupus ◽  
2010 ◽  
Vol 19 (12) ◽  
pp. 1391-1398 ◽  
Author(s):  
Y. Avihingsanon ◽  
N. Hirankarn
Keyword(s):  
Lupus Nephritis ◽  
Lupus Erythematosus ◽  
Opportunistic Infections ◽  
Early Recognition ◽  
Asian Population ◽  
Immunosuppressive Drugs ◽  
First Episode ◽  
Asian Patients ◽  
Renal Flare ◽  
Number Of Patients

Lupus nephritis is a common and severe complication of systemic lupus erythematosus. A number of patients have nephritis as a presenting feature that, in its severe form, can shortly lead to end-stage renal disease and/or death. Renal flare usually occurs a few years after the first episode and is remarkably predominant in the Asian population. Frequent monitoring for renal flare enhances early recognition and timely treatment. The mainstay therapy continues to be the prolonged use of cytotoxic/immunosuppressive drugs that have a number of undesirable effects, particularly ovarian failure and development of opportunistic infections. This review will focus on the pathogenesis and the unique genetic factors found in Asian patients with lupus nephritis. Here, we propose an appropriate management scheme for the treatment of lupus nephritis in Asian patients.

Enrollment of Elderly Patients in Clinical Trials for Cancer Drug Registration: A 7-Year Experience by the US Food and Drug Administration

2004 ◽  
Vol 22 (22) ◽  
pp. 4626-4631 ◽  
Author(s):  
Lilia Talarico ◽  
Gang Chen ◽  
Richard Pazdur
Keyword(s):  
Clinical Trials ◽  
Cancer Patients ◽  
Drug Administration ◽  
Food And Drug Administration ◽  
The Elderly ◽  
New Drugs ◽  
Cancer Drug ◽  
Cancer Therapies ◽  
Age Related ◽  
The Us

Purpose To analyze the age-related enrollment of cancer patients onto registration trials of new drugs or new indications approved by the US Food and Drug Administration from 1995 to 2002. Patients and Methods This study involved retrospective analyses of demographic data of cancer patients enrolled onto registration trials. The data on 28,766 cancer patients from 55 registration trials were analyzed according to age distributions of ≥ 65, ≥ 70, and ≥ 75 years. The rates of enrollment in each age group for each cancer were compared with the corresponding rates in the US cancer population. The age distributions of the US cancer population were derived from the Surveillance, Epidemiology, and End Results Program of the National Cancer Institute for the period 1995 to 1999 based on the 2000 US Census. Results The proportions of the overall patient populations aged ≥ 65, ≥ 70, and ≥ 75 years were 36%, 20%, and 9% compared with 60%, 46%, and 31%, respectively, in the US cancer population. Statistically significant under-representation of the elderly (P < .001) was noted in registration trials for all cancer treatment except for breast cancer hormonal therapies. Patients aged ≥ 70 years accounted for most of the under-representation. Conclusion Elderly were under-represented in the registration trials of new cancer therapies. Various strategies may be needed to evaluate cancer therapies for the elderly in prospective clinical trials and to improve cancer care in the elderly population.

Regulatory Aspects of Vascular Dementia in the United States

2003 ◽  
Vol 15 (S1) ◽  
pp. 293-295 ◽  
Author(s):  
Armando Oliva ◽  
Ranjit Mani ◽  
Russell Katz
Keyword(s):  
Clinical Trials ◽  
Vascular Dementia ◽  
Diagnostic Criteria ◽  
The United States ◽  
Clinical Syndrome ◽  
New Drugs ◽  
The Us ◽  
Significant Interest ◽  
Modifying Effect ◽  
Special Meeting

There is significant interest in the development of new drugs to treat vascular dementia. However, before US approval of new drugs for this entity is possible, certain issues with regulatory implications need to be addressed. Is vascular dementia a distinct clinical syndrome with valid diagnostic criteria? Can this entity be distinguished from Alzheimer's disease (AD) and other causes of dementia? What design features are important for clinical trials in this disorder? The US Food and Drug Administration (FDA) convened a special meeting of the Peripheral and Central Nervous System Advisory Committee in an attempt to answer these questions. The conclusions from this meeting indicate that vascular dementia (VaD) is a pathologically heterogeneous disorder but appears to be reasonably distinguishable from AD dementia. The NINDS-AIREN diagnostic criteria are suitable as entry criteria for vascular dementia trials. Trials should be similar in duration to AD dementia trials and should employ a dual outcome strategy (cognitive + global/functional measures). For drugs that are believed to have a disease-modifying effect, clinical trials should study specific vascular dementia subtypes and would need to employ substantially different designs from those used currently. The term “vascular dementia” may not be entirely appropriate to describe this population.

Exclusion of Patients Living with Chronic Viral Infections and Underrepresentation of Ethnic Minorities in Diffuse Large B-Cell Lymphoma Clinical Trials

Blood ◽  
2020 ◽  
Vol 136 (Supplement 1) ◽  
pp. 36-37
Author(s):  
Stephanie Boisclair ◽  
Richie Uba ◽  
Amanda Brahim ◽  
Fernando Vargas Madueno ◽  
Julio C. Chavez ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Viral Hepatitis ◽  
Race And Ethnicity ◽  
Cell Lymphoma ◽  
B Cell Lymphoma ◽  
Chronic Viral Hepatitis ◽  
Asian Patients ◽  
Large B Cell Lymphoma ◽  
The Us ◽  
Phase Ii And Iii

Introduction There is a substantial underrepresentation of ethnic minorities within oncology studies. Specifically, Black, Asian and Hispanic patients have a low participation rate in US based randomized clinical trials (RCT). Furthermore, patients living with HIV (PLWH) or chronic viral hepatitis are often systematically excluded from these studies. Diffuse large B-cell lymphoma (DLBCL) is one of the most common malignancies associated with HIV and viral hepatitis. Given these characteristics, it is imperative that studies reporting on safety and efficacy of new treatments mirror real-world populations. Objective: The primary objective of our study was to describe the demographic characteristics of patients who have participated in DLBCL phase II and III randomized US clinical trials; evaluating each group's enrollment fraction compared to US DLBCL prevalence. The secondary objective was analyzing the inclusion of PLWH and chronic viral hepatitis in US based DLBCL RCT. Methods and Measures: We queried PubMed, ClinicalTrials.gov, as well as ASCO and ASH meeting abstracts for phase II and III RCTs on DLBCL from 2000 to 2020, including chimeric antigen receptor T cells (CAR-T) therapy studies. Trials that did not report race and ethnicity or recruited outside of the US were excluded. We analyzed enrollment data and compared to DLBCL prevalence demographics obtained from the SEER 18 Registries. Enrollment fraction (EF) is defined as the number of trial enrollees divided by the estimated US prevalence of DLBCL. Results: We identified 51 trials between 2000 and 2020 with only 25 (49%) studies reporting race and ethnicity. Only 9 trials enrolled solely in the US which comprised of 763 patients. Compared with an EF of 1.88% among Non-Hispanic whites, lower EF was noted in Blacks (1.08%; P=0.0009), Hispanics (0.71; P=&lt;0.0001) and Asian patients (0.71%; P=&lt;0.0001). The EF of patients &lt; 65 years old was significantly higher than those aged 65 or more (1.2 vs 0.79%, P=&lt;0.0001). The EF of males and females was 1.8 vs 1.4%, respectively (P=0.001). PLWH and/or chronic viral hepatitis were excluded in 89% and 67% of the clinical trials analyzed (Table 1). Conclusions: US based clinical trials for DLBCL have significant underrepresentation of Hispanic, Black and Asian patients. Moreover, despite being commonly associated with HIV and chronic viral hepatitis, patients with these comorbidities are typically excluded from clinical trials. Pharmacoethnicity, ethnic differences in susceptibility to the effects of therapy, remains a challenge in oncology because clinical trials have significant underrepresentation of minorities. Efforts are needed to address the ascertainment bias and improve external validity of RCTs in DLCBL. Reasons for clinical trial underrepresentation are complex and multifactorial including lack of access, cultural differences and healthcare system mistrust. Healthcare professionals must address the inequalities through awareness and education with the objective of increasing enrollment of minorities in clinical trials. Although there have been efforts to increase enrollment of patients with HIV and chronic viral hepatitis in clinical trials, it remains inadequate. In accordance to recent recommendations by the FDA, investigators should consider having a predefined plan for inclusion of such patients. Disclosures Sandoval-Sus: Massive Bio: Consultancy; Janssen: Consultancy; MorphoSys US: Consultancy; Celgene: Speakers Bureau.

IS ALZHEIMER’S DISEASE DRUG DEVELOPMENT BROKEN? WHAT MUST BE IMPROVED

2014 ◽  
pp. 1-6
Author(s):  
P.-J. Ousset ◽  
J. Cummings ◽  
J. Delrieu ◽  
V. Legrand ◽  
N. Prins ◽  
...  
Keyword(s):  
Alzheimer’S Disease ◽  
Alzheimer's Disease ◽  
Clinical Trials ◽  
Drug Development ◽  
Drop Out ◽  
New Drugs ◽  
Trial Duration ◽  
Surrogate Outcomes ◽  
Technical Requirements ◽  
Number Of Patients

During the decade from 2002 to 2012, 99.6% of the 244 agents tested for efficacy in slowing the progression of Alzheimer’s’ disease (AD) failed to achieve their primary endpoints. At a CTAD symposium on November 14, 2013, in San Diego, USA, an international group of AD researchers met to discuss the evolution of trials over the past 10 years and proposed a number of changes intended to streamline and enhance the efficiency of clinical trials. Approximately 1,031 AD trials were conducted between 2000 and 2012. The number of patients per trial site tended to decrease over time necessitating a larger number of sites. The use of biomarkers for enrichment purposes, or as measures of target engagement or surrogate outcomes, results in higher screen failure and drop-out rates, adding to trial duration and/or costs. Present disease modifying AD trials ask for increasing logistical and technical requirements, necessitating the creation of highly specialized trial facilities and limiting the participation of smaller sites. Due to heavy administrative and regulatory task, only about 13% of the team's time is used for the essential recruitment. Proposals and perspectives: Strategies suggested to improve the efficiency of recruitment include establishing “ready to go cohorts” in advance of trials using biomarkers and clinical measures. Simplification and harmonization of administrative procedures, including harmonization of certification procedures, are urgently needed. Alternative approaches, such as using the Internet to screen volunteers for possible inclusion needs to be evaluated. The AD drug development enterprise from discovery through clinical trials requires re-examination and re-organization if new drugs are to be delivered to patients in a timely way.

scholarly journals Pharmacotherapy for Patients with Obesity

2018 ◽  
Vol 64 (1) ◽  
pp. 118-129 ◽  
Author(s):  
Kishore M Gadde ◽  
John W Apolzan ◽  
Hans-Rudolf Berthoud
Keyword(s):  
Weight Loss ◽  
Clinical Trials ◽  
Weight Management ◽  
Neural Control ◽  
Safety Data ◽  
New Drugs ◽  
Future Research ◽  
The Us ◽  
Antiobesity Drugs

Abstract BACKGROUND Although pharmacotherapy is not the cornerstone of obesity treatment, it is a valuable tool that could be considered for patients who have not had adequate benefit from lifestyle interventions or who have difficulty maintaining initial weight loss over longer periods. CONTENT This review focuses on the role of antiobesity drugs, the mechanisms by which the drugs work, potential pharmacological targets in the neural control of food intake and regulation of body weight, the history of antiobesity drugs, a summary of efficacy and safety data from clinical trials, and the clinical application of pharmacotherapy. Currently, 5 approved drug therapies are available in the US for long-term weight management, with only 2 of these meeting the stronger Food and Drug Administration (FDA) criteria of 5% weight loss relative to a placebo after 1 year and others receiving approval based on the categorical criterion of the proportions of patients achieving 5% weight loss. Interpretation of the results of clinical trials conducted before regulatory agency approval is limited by high dropout rates; thus, the results might not be replicable in clinical practice settings. Many patients who are suitable candidates for pharmacotherapy are not using the new drugs due to lack of insurance coverage and high out-of-pocket costs. SUMMARY With the availability of 4 new drugs since 2012, clinicians in the US now have more tools for long-term weight management. The quality of pharmacotherapy clinical investigations needs considerable improvement. Future research should focus on examining the mediators and moderators of response.

Comparison of the Basis for Approval and Pivotal Studies on Hematological Malignancies between the US and Japan.

Blood ◽  
2006 ◽  
Vol 108 (11) ◽  
pp. 5495-5495
Author(s):  
Fumitaka Nagamura ◽  
Tokiko Nagamura-Inoue ◽  
Arinobu Tojo
Keyword(s):  
Clinical Trials ◽  
Hematological Malignancies ◽  
Response Rate ◽  
Phase 1 ◽  
Regulatory Agencies ◽  
Remission Induction ◽  
Package Inserts ◽  
Number Of Patients ◽  
The Us ◽  
Efficacy Parameters

Abstract Introduction: Clinical trials are designed based on certain hypotheses and to meet the requirements for approval from regulatory agencies. We previously reported on differences in the trends of drug approvals for hematological malignancies between the US and Japan (Proc ASH #3120, 2005). This time, we report on differences in the basis for approval and designs of pivotal studies between the two countries. Methods: Drugs approved for hematological malignancies from January 1985 to December 2005 in both the US and Japan were selected. Of these candidates, only indications common to both countries were considered. Supportive care drugs, immunomodulators, biochemical modulators, and off-label use were excluded. Package inserts, reviews and analyses by regulatory agencies, and publications on clinical trials were examined. Results: Ten drugs (mitoxantrone, idarubicin, pentostatin, fludarabine, cladribine, tretinoin, rituximab, arsenic trioxide, imatinib mesylate, and gemtuzumab ozogamicin) were met the criteria. “Line or type of therapy (e.g. 1st line, or for remission induction)” was specified for all drugs in the US and three in Japan. Limitations on age, e.g., adults, were indicated in the package inserts of 5 drugs in the US and 1 in Japan. The phase of pivotal studies are summarized in Table 1. In Japan, no description on phase was seen in 2 drugs, and the result of 1 comparative study was applied. The number of patients enrolled into pivotal studies are summarized in Table 2. The number of efficacy parameters examined in each pivotal study (range, mean, and median) were 1–11, 4.7, and 3 in the US and 1–5, 2.3, and 2 in Japan, respectively. Survival was used as an efficacy parameter in 7 drugs in the US, but none in Japan (PFS: 1 drug). Differences in efficacy parameters between the two countries have decreased, and inappropriate uses of efficacy parameters, e.g., overall response rate for mitoxantrone and idarubicin, have disappeared recently in Japan. Conclusion: The high ratio of RCT and the large number of patients enrolled into clinical trials are characteristics of the US. The approved indications in Japan are broad-based, however, the description of package inserts, number of patients, and the use of efficacy parameters were inferior. The basic principle for approval of regulatory agencies in Japan had been response rate, however, the policy has become changing. So, the criticisms of Japanese clinical trials, such as poor design, insufficient information, and less utilization for approval by other countries, could be resolved. Table 1: Number of pivotal studies classified by phase # of drugs Randomized comparative trial (RCT) Single arm stdy (SA) Phase 1 study Combined data Others US 10 9 15 0 5 1 (data after cross-over) Japan 8 1 14 2 4 (foreign data) 0 Table 2: Number of patients enrolled into pivotal studies SA study (range, mean, range) RCT* (range, mean, median) *Number of patients on study arm US 31–532, 130.1, 89 40–553, 149.1, 111 Japan with foreign data 10–532, 88.9, 47.0 31: 1 study Japan without foreign data 10–74, 37.4, 38.5 31: 1 study

scholarly journals Variation in the estimated costs of pivotal clinical benefit trials supporting the US approval of new therapeutic agents, 2015–2017: a cross-sectional study

BMJ Open ◽  
2020 ◽  
Vol 10 (6) ◽  
pp. e038863 ◽  
Author(s):  
Thomas J Moore ◽  
James Heyward ◽  
Gerard Anderson ◽  
G Caleb Alexander
Keyword(s):  
Clinical Trials ◽  
Software Tool ◽  
Cross Sectional Study ◽  
Therapeutic Agents ◽  
Secondary Outcome ◽  
Sectional Study ◽  
Cross Sectional ◽  
Number Of Patients ◽  
The Us ◽  
Clinic Visits

ObjectivesLittle is routinely disclosed about the costs of the pivotal clinical trials that provide the key scientific evidence of the treatment benefits of new therapeutic agents. We expand our earlier research to examine why the estimated costs may vary 100-fold.DesignA cross-sectional study of the estimated costs of the pivotal clinical trials supporting the approval of 101 new therapeutic agents approved by the US Food and Drug Administration from 2015 to 2017.MethodsWe licensed a software tool used by the pharmaceutical industry to estimate the likely costs of clinical trials to be conducted by contract research organisations. For each trial we collected 52 study characteristics. Linear regression was used to assess the most important factors affecting costs.Primary and secondary outcome measuresThe mean and 95% CI of 225 pivotal clinical trials using varying assumptions. We also assessed median estimated costs per patient, per clinic visit and per drug.ResultsMeasured as pivotal trials cost per approved drug, the 101 new molecular entities had an estimated median cost of US$48 million (IQR US$20 million–US$102 million). The 225 individual clinical trials had a median estimate of US$19 million (IQR US$12 million–US$33 million) per trial and US$41 413 (IQR, US$29 894–US$75 047) per patient. The largest single factor driving cost was the number of patients required to establish the treatment effects and varied from 4 patients to 8442. Next was the number of trial clinic visits, which ranged from 2 to 166. Our statistical model showed trial costs rose exponentially with these two variables (R2=0.696, F=257.9, p<0.01).ConclusionsThe estimated costs are modest for measuring the benefits of new therapeutic agents but rise exponentially as more patients and clinic visits are required to establish a drug effect.

scholarly journals Proportion of Patients with FLT3 Positive AML Required to Enroll on Clinical Trials to Satisfy the Recruitment Needs of FLT3 Inhibitor Trials: Are We Running out of Patients?

Blood ◽  
2018 ◽  
Vol 132 (Supplement 1) ◽  
pp. 713-713
Author(s):  
Taylor Bucy ◽  
John Zoscak ◽  
Motomi Mori ◽  
Vinayak Prasad ◽  
Uma Borate
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Cooperative Group ◽  
Participation Rates ◽  
Therapeutic Trials ◽  
Fda Approval ◽  
Number Of Patients ◽  
The Us ◽  
Flt3 Mutations ◽  
Flt3 Inhibitors

Abstract Introduction. The push for personalized medicine in oncology has generated an influx of therapies targeting similar pathways, despite only 5% of clinically tested agents receiving FDA approval (Mattina 2017). Acute myeloid leukemia (AML) is a malignancy where identification of prognostically significant mutations suggests growing potential for therapeutic inhibition. FMS-like tyrosine kinase 3 (FLT3) mutations are deemed one of few "actionable" mutations and include 30% of de novo AML cases (25% ITD, 5% TKD) (Garcia 2017; Fathi 2016). In 2017 there will be approximately 21000 new AML cases, roughly 6000 of these patients will be FLT3+ (ACS 2017). In line with prior research in melanoma elucidating the growing number of trials aimed at personalized medicine, we sought to identify the total number of therapeutic trials studying FLT3 inhibitors (Tang 2018). We analyzed the total number of FLT3+ patients required for recruitment to these trials to ensure successful completion compared to incidence of this molecular abnormality within the AML patient population. Methods.A systematic review of therapeutic clinical trials focusing on adult FLT3+ AML from 2000-2017 was conducted using the PRISMA 2009 guidelines (Figure 1). We identified 78 therapeutic trials of FLT3 targeted therapies by cross-referencing ClinicalTrials.gov (66) and PubMed publications (12). Assuming constant accrual rate over the duration of each trial and constant rate of FLT3 mutations at 30% since 2000, statistical analysis was performed using the study start dates and primary completion dates of all trials from ClinicalTrials.gov. Incidence data was collected from the CDC, SEER database, and literature review (De Kouchkovsky 2016; ASCO 2018). Projections of discrepancies between anticipated clinical trial enrollment were provided using consistently cited rates of adult participation of 1%, 3% and 5% versus participant enrollment needed to satisfy current projected trends (Rinde 2018; Unger 2016). Results. Twenty-four therapeutic FLT3 inhibitors being investigated were identified in 78 distinct clinical trials. Pharmaceutical versus cooperative group support was 2.23:1, with 29 different pharmaceutical sponsors and 13 cooperative group/non-profit/academic sponsors(Table 1). Thirty-eight (48.7%) trials/publications accrued in the US only, 21 (26.9%) at international sites only, 15 (19.2%) accrued in both the US and internationally, and 4 (5.1%) had no location listed at time of search. Only one study (NCT03258931) proposed a head-to-head comparison of midostaurin versus crenolanib. The number of patients needed to satisfy enrollment began to surpass the upper bound of estimated participation in 2010, dropping slightly from 2013-2014 and noticeably surpassing projected participation rates from 2015-2016 (Figures 2 & 3). In 2017, approximately 21380 patients were diagnosed with AML, roughly 6414 were FLT3 positive. Assuming 5% participation rate, 320.7 FLT3 positive patients would be expected to enroll in clinical trials. However, the total number of patients needed to satisfy enrollment in 2017 was 1235; after excluding international and completed trials, the total number needed is 844.49 patients. Based on statistical analyses, we estimate that 13.2% of all US patients with FLT3 pos AML would have to enroll to satisfy eligibility in 2017; roughly 3 times the upper level of historical clinical trial participation rates in the US. Conclusions. The current clinical trial process investigating targeted therapies in AML requires an unusually high and unsustainable enrollment, given the discrepancy between the incidence of AML patients with targetable mutations and the number of pharmaceutical agents being studied for these small patient populations. This discrepancy becomes even more startling when considering barriers to enrollment, including insurance market restrictions, geographical, socioeconomic, and demographic factors. Most of these trials do not compare available agents to identify the drug that may provide the most patient benefit. Whether this method of finding new therapies eventually leading to FDA approval continues to be sustainable or whether such duplicative trials dilute the valuable resource of AML patients with rare, targetable mutations, thus impeding development of the most effective therapeutic agents, must considered by the research and regulatory community. Disclosures Borate: Novartis: Consultancy; Agios: Consultancy.

scholarly journals Current requirements for studies of drugs for the pathogenetic treatment of multiple sclerosis

2019 ◽  
Vol 11 (4) ◽  
pp. 166-171
Author(s):  
A. N. Boyko ◽  
N. N. Spirin ◽  
Ya. V. Vlasov ◽  
M. N. Zakharova
Keyword(s):  
Multiple Sclerosis ◽  
Clinical Trials ◽  
High Efficiency ◽  
New Drugs ◽  
Extensive Experience ◽  
Number Of Patients ◽  
Long Term Follow Up ◽  
Based Medicine

More than 10 multiple sclerosis-modifying drugs (MSMDs) are widely used now. Novel MSMDs should be investigated in strict accordance with the evidence-based medicine principles governing clinical trials (of both original drugs and their analogues) that prove the high efficiency, safety, and tolerability of new drugs versus the already existing ones. Russia has gained extensive experience in conducting such studies using the well-known drugs as a comparison group. The efficiency and safety of new therapy should be evaluated according to the international criteria on the basis of a sufficient number of patients during a long-term follow-up. When combining the drugs, their efficiency and the risk of adverse effects can vary. The published results of a small study of the combined drug Leucovir (Belarus) do not meet these requirements, and the possibility of using this drug to treat multiple sclerosis can be discussed only after adequate phases II and III clinical trials.

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