Feb 6, 2020: Amarin Corporation plc announced that BioNJ awarded Amarin with an Innovator Award in recognition of the approval of a new indication for VASCEPA® (icosapent ethyl) by the U.S.FDA in December 2019.
This approval places VASCEPA as the first and only approved drug for minimizing cardiovascular events in millions of select patients at high risk.
The approval follows a decade of research and development in a clinically complex field of the disease marked by significant unmet medical needs.
“BioNJ is happy to present Amarin with the Innovator Award for its dedication to expanding this affordable and innovative therapy, VASCEPA, for use to reduce cardiovascular risk,” said Debbie Hart, president and chief executive officer of BioNJ.
“Amarin has clearly demonstrated that it is a committed corporate citizen and is dedicated to the continued expansion of the biotech industry in the State of New Jersey.”
The success of VASCEPA as a recognized new, cost-effective therapy in order to reduce cardiovascular risk has propelled Amarin’s growth from a dozen employees in 2011 to its current size of more than 1000 people in the United States and Ireland, more than 100 of whom are in New Jersey.
In its important new cardiovascular risk reduction designation, Amarin’s successful completion of the groundbreaking REDUCE-IT ® clinical trial was the basis for VASCEPA’s FDA-approval.
In this review of statin therapy patients who had specific cardiovascular risk factors amid wellcontrolled cholesterol, VASCEPA showed a significant 25 per cent reduction in major adverse cardiovascular events beyond the advantages of the existing medical norm.
In recognition of these extraordinary results, VASCEPA received a 16-0 vote in favor of the approval by the FDA Endocrinologic and Metabolic Drugs Advisory Committee, with subsequent approval granted by the FDA.
Feb 5, 2020: Nestlé Health Science and Valbiotis, a research and development company committed to scientific innovation for preventing and fighting metabolic diseases, have entered into a deliberate partnership for the development and commercialization of an innovative and patented product to reduce the risk of developing Type 2 Diabetes.
TOTUM-63 is the combination of 5 plant extracts specially designed in order to reduce the risk of developing Type 2 Diabetes on prediabetic subjects.
Sébastien Peltier, CEO of Valbiotis commented, “Nestlé Health Science is an ideal strategic partner for Valbiotis.
Its global reach, strategic intent to develop science-based nutritional health solutions and focus on fighting metabolic disorders like diabetes will be instrumental to TOTUM-63’s worldwide commercialization success.
We are excited about the opportunity that this deal brings to Valbiotis and to the many millions of people around the world at risk of becoming Type 2 diabetics.
This transformational deal arrives just five years after the creation of Valbiotis and is a recognition of the hard work, commitment and vision of our team.”
Under the terms of the agreement, Valbiotis grants exclusive and global commercial rights to Nestlé Health Science for the use of TOTUM-63 in the prediabetes and type 2 diabetes market.
This blog has been written after discussion with many NIPER qualifiers. Even the Writer of this blog is Ex NIPERian. He got 56 Rank in GPAT and 90 Rank in NIPER. To answer” How to Score AIR Under 100 in NIPER”, this blog is divided into the following categories.
Introduction
Exam Pattern
Eligibility
Import topics and syllabus-Must read
Key Advice for every NIPER aspirants
Recommended Books
Importance of being a NIPERian
Table of Contents
Introduction
The National Institute of Pharmaceutical Education and Research (NIPER) are the national level-Pharmaceutical Sciences Institutes.
The primary goal of NIPER is to become the centre of excellence for advanced studies and research in Pharmaceutical Science. NIPER Mohali was the first established NIPER through a Parliament act dated 26-Jun-1998.
During the years 2007- 2008, the Indian Government established six more NIPERs and list of all NIPERs is as given below:
Total number of questions will be 200 (One mark each)
Questions will be an objective-Multiple choice
Questions will come from B.Pharm. And M. Sc. (relevant field) syllabus
Few additional questions from General Aptitude, General English, Pharma News, Recent drug approvals, and Recent Big Events
Negative marking is done for each wrong answer (25% Mark will be deducted)
Duration of exam: 2 Hours.
Exam Date: June 10, 2020 (tentative)
Start date of application: Last week of April-2020 (tentative)
The end date of filling application/registration: May Second week-2020 (tentative)Actual Dates may vary but above tentative dates can help you to schedule your study time table.
NIPER JEE 2020 Result declaration: 10 days after the examination (Second week of June-2020
Group Discussion and Interview for MBA (Pharm): Qualified NIPER test-students will be called for interview (based on RANK)-Second week of July–2020
Counselling: Second Week of July.(Including for Pharma MBA)
Total Seats: 600-650 approx
Course offered at NIPERs: M. Pharm,M.S., M. Tech (Pharm), MBA (Pharm), and Ph. D
Eligibility
“The candidate should have a Bachelor’s degree in Pharmacy or equivalent degree (Depends on Courses, you are applying for). See the table below.
Candidates should have secured 60 percents or 6.75 GPA for the General category.
55 percents or 6.25 CGPA for SC /ST candidates and 50 percents or 5.75 GPA for PH candidates’ in aggregate in the qualifying exam.
Candidates should also have a valid GATE or NET or GPAT score.”
B.Pharm.; B.Tech (Chemical Engg. or equivalent); M.Sc. (Chemical /Life Sciences)
“Important to note that for students awaiting Final year result: Registrar of the respective university has to send confidential results from official email ID before final counselling to the following E mail ID: [email protected].”
As per the 2019 NIPER brochure: “It is mandatory to bring GPAT/GATE/NET scorecard at the time of counselling/Group Discussion and Interview. Further GPAT/GATE/NET is an essential qualification for all programmes [including M.B.A.(Pharm.) except for the following categories of candidates.” “Candidates holding B.V.Sc./M.B.B.S./B.A.M.S .degree Foreign nationals Sponsored candidate from public/private sector undertaking, Govt. departments and research and development organizations Candidates applying as NRI or their wards [in case of M.B.A.(Pharm.)]”
Import topics and syllabus-Must read
An official statement from 2019 NIPER brochure: The level of questions will be of B.Pharm. And M. Sc. (relevant field). They do not provide topic wise syllabus but it covers the following topics
GPAT syllabus + “General Aptitude questions, General English, Pharma News, Recent drug approvals, Recent Big News and General Chemistry
Important topics: These topics are part of GPAT syallabus as well. Chemistry: Basic organic, physical and Inorganic Chemistry, Reactions name and type, intermediate and final product name, Stereochemistry- Good number of questions will come from chemistry. Important structures such as diazepam, Sulfonamides etc and SAR.
Basic Statistics: mean, median, mode, ANOVA, paired test etc
Pharmacology: Pharmacology classification and important side effects, Receptor classification and function such as Adrenergic receptor, Opioid Receptors etc., Mechanism of action and Target.
Pharmacognosy: Pharmacognosy classification, testing methods eg alkaloids, active constituents and use, Basic Pharmacognosy
Pharmaceutics: Role and names of various ingredients in the tablet, capsule, injection, aerosol etc and their evaluation method and apparatus used, Different limits (all tables), Novel drug delivery system such as dendrimers.
Pharmaceutical analysis: Various stability testing, HPLC, Chromatography, various formula, pH, PKA, surfactant HLB values, complete Spectroscopy
Biotechnology: Gene expression, transcription, replication translation, recombination and mutation and Gene therapy
Microbiology: Everything about antibody, various microbiological test, sterilization methods
Forensic Pharmacy All Schedules and forms numbers
Biochemistry: Protein, Peptide and Enzyme and basic biochemistry
Frequently asked question in Previous papers `(Taken From Pharmatutor)
“Polarity of solvents for chromatography, Rf values for better separation, Absorbent in reverse phase chromatography, Partition coefficient between liquid-liquid phase, Naming of peptides (i.e. pentapeptide, hexapeptide) Hetero and homo diens, Setro of biphenyl, cyclohexane(conformations)
Haemllet equation and saturation kinetics, IR frequency of carbonyl compounds, Solvents for IR and NMR, Mechanism of action of barbiturates, pyrimethamine, co-trimoxazole
The technique for separation of isomers and racemic mixture, Applications of spectroscopic technique, Gas laws (boyle charles etc), Polymers in pharmaceutics, Calculation for J value (NMR), Isomers of important phytopharmaceuticals and its use, Alpha carotene contains how many rings, Squalene contains how many isoprene units,
Use of each 5 HT receptors, Measurement of flow properties and its importance, Role of excipients and stage of the addition of each Solvents for oral use, iv use and reconstitution, All IP standards for tablets and capsules, Accelerated stability testing,
Applications of various mixers and dryers, Identification of peaks in mass spectroscopy, Surfactants its classification and HLB values for different surfactants, Antimalarial drugs their structure, IUPAC, mechanism of action Enzymes as biomarkers in a various disease state, Protein binding of important drugs, Vitamins: name, deficiency“
Additional Important topics which are not in GPAT syllabus
Recently approved drugs by the FDA and EMA
Recently given Nobel Prize winners- name and work
Basics of Clinical Research like Phase of clinical trials, type of studies, Important labs in India and their locations.
The drug approval process in India and the USA
Patent and exclusivity
Recent Big Merger/Acquisition in Pharma Industry.
Brand name of Highest Selling (Blockbuster) drug in India and their Manufacturer name
Any recent big news Like Coronavirus outbreak, International games
General aptitude questions: Coding and Decoding, Chain Rule, Alligation or MixtureProblems on NumbersTime and WorkRatio and Proportion, Average, Percentage, diagram-based questions
Basic English Grammer Question: Active and Passive voices, Synonyms Idioms and Phrases Word, Correction Questions, Common Errors in Tense Grammar
Key Advice for every NIPER aspirants:
Who scored less in GPAT (Just qualified) or could not get a good rank, should not be disappointed. You can score quite well in NIPER as both exams are quite different. There are many individuals who got AIR 2300 or >2200 in GPAT but got AIR under 100 in NIPER. You need to work hard with a positive attitude.
Revise the key topics
You must give mock tests as much as possible. This is the best thing you can do now. You have to finish 200 questions in 120 minutes, so accuracy and time management is the key. Both these things can be learnt through the mock test.
In the first attempt, you should solve those questions, where you are 100 percent sure. Do not waste much time on any one question. Any question should not be unseen when the timeout.
In Second attempt, you can start answering, where you are not 100 percent sure. Attempt those question as well, where you are reasonably sure (not necessarily 100 percent)
Leave those questions, where you do not have any Idea.
Avoid Social Media for three months. Take care of your health.
Recommended Books
Subject
Author Name/Book Name/website
Organic Chemistry
Morrison Boyd
Advanced Organic Chemistry
Bahl and Bahl
Pharmacology
K D Tripathi (Essentials of Medical Pharmacology), Rang and Dale
This is quite an important question and We are sure that the answer to this question can motivate you. There are six unavoidable reasons to choose NIPER and they are:
First of all, it is a National Institute for excellence- It means you will carry a brand value with you always.
Highly talented teaching faculty- Even Scientists come for teaching. You will get the opportunity to attend many workshops and National seminars.
All NIPERs are associated with Research Institutes like CSIR and have collaborations with many Pharma companies: You will get the chance to do project work in Big Pharma companies and CSIR or ICMR labs.
Awesome Hostel Life- All NIPER hostels are well maintained. You will remember hostel life throughout your life. Good facility for sports as well.
Reasonable Fees- Needless to mention this. you can easily compare with Private Institutes.
Placements are quite well. You can get jobs in Many MNCs
Feb 7, 2020: U.S. FDA authorized marketing of software to assist medical professionals in the acquisition of cardiac ultrasound, or echocardiography, images.
This software, called Caption Guidance and is an accessory to compatible diagnostic ultrasound systems and uses artificial intelligence in order to help the user capture images of a patient’s heart that are of acceptable diagnostic quality.
The Caption Guidance software is indicated for use in ultrasound examination of the heart, known as two-dimensional transthoracic echocardiography (2D-TTE), for the adult patients, especially in the acquisition of standard views of the heart from different angles. These views are normally used in the diagnosis of various cardiac conditions.
“Echocardiograms are one of the most widely used diagnostic tools in the diagnosis and treatment of heart disease,” said Robert Ochs, Ph.D., deputy director of the Office of In Vitro Diagnostics and Radiological Health in the FDA’s Center for Devices and Radiological Health.
“Today’s marketing authorization enables medical professionals who may not be experts in ultrasonography, such as a registered nurse in a family care clinic or others, to use this tool.
This is especially important because it demonstrates the potential for artificial intelligence and machine learning technologies to increase access to safe and effective cardiac diagnostics that can be life-saving for patients.”
According to the Centers for Disease Control and Prevention, heart disease is the leading cause of death in the US, killing one out of every four people, or around 647,000 Americans each year.
The term heart disease refers to several types of heart conditions. The most common type is coronary artery disease, which may cause a heart attack. Other kinds of heart disease may involve the valves in the heart, or the heart may not pump well and cause heart failure.
Cardiac diagnostic tests also essential
to identify heart conditions. These can be electrocardiograms (EKG or ECG),
Holter monitors and cardiac ultrasound examinations.
The software authorized is the first software authorized to guide users through cardiac ultrasound image achievements.
The Caption Guidance software was developed by using machine learning to train the software to differentiate between the acceptable and unacceptable image quality. This information formed the basis for an interactive AI user interface which provides users with prescriptive guidance on how to manoeuvre the ultrasound probe to acquire standard diagnostic quality echocardiographic images and video clips.
AI interface provides real-time feedback on potential image quality, is capable of auto capturing video clips, and automatically saves the best video clip from a given view. Significantly, the cardiologist still reviews the images for the final assessment of the images and videos for patient evaluation.
The FDA analyzed data from two independent reports. For one test, 50 qualified sonographers scanned patients, using the Caption Guidance program and without it. In both cases, the sonographers were able to capture photographs of equal diagnostic quality.
The other study involved teaching eight registered nurses who are not sonography experts to use the Caption Guidance program and ask them to capture regular echocardiography pictures, followed by five cardiologists reviewing the accuracy of the pictures taken. The data showed that the Caption Guidance software enabled the registered nurses to acquire echocardiography images and also videos of diagnostic quality.
Feb 6, 2020: Its primary endpoint was reached by the Sanofi Phase 2b trial evaluating its investigational BTK (Bruton’s tyrosine kinase) inhibitor (SAR442168), an oral brain penetrating, selective small molecule.
In the trial, as measured by the magnetic resonance imaging (MRI), SAR442168 significantly reduced disease activity associated despite multiple sclerosis (MS). With no new safety results, SAR442168 had been well accepted.
The BTK inhibitor is deliberate to modulate both adaptive (B-cell activation) and innate (CNS microglial cells) immune cells linked to neuroinflammation in the brain and spinal cord.
Four Phase 3 studies will investigate the effects of SAR442168 on rates of MS relapse, the progression of the disability and underlying damage to the central nervous system.
Phase 3 trials in both recurrent and progressive forms of MS are scheduled to be launched in mid-year.
In the US and Europe, there are about 1.2 million people diagnosed with MS, an impulsive,
chronic disease that attacks the central nervous system.
regardless of the current treatments, many MS patients continue to accumulate disability, and one in four MS patients suffer from progressive forms of the disease with the limited or no treatments available. The global market for MS therapies exceeds €20 billion annually. Comprehensive results from the Phase 2b trial with advanced imaging endpoints will be presented at an upcoming medical meeting.
About the Phase 2b Trial: The Phase 2b study was a 12 week ranging, randomized, double-blind, week dose placebo-controlled trial assessing SAR442168 in patients with chronic MS.
About SAR442168: SAR442168 is an investigational, oral, brain-penetrator, selective small-molecule inhibitor of BTK. SAR442168 works by BTK binding as well as cerebrospinal fluid exposure in the Phase 1 studies.
The effectiveness and safety of SA442168 have not been reviewed by any regulatory authority.
Feb. 06, 2020: Aquestive Therapeutics, Inc, announced today that it had a constructive face-to-face pre-IND Application meeting with the U.S.FDA for its drug candidate, AQST-108, a “first of its kind”
Oral sublingual film formulation delivering systemic epinephrine under development for anaphylaxis treatment using Aquestive’s proprietary PharmFilm ® technology.A pre-IND meeting provides an opportunity for an open communication between a drug sponsor and the FDA in order to discuss the sponsor’s IND development plan and to obtain the agency’s guidance for the clinical studies for the sponsor’s new drug candidate.
The FDA has confirmed that the clinical development for AQST108 will be reviewed under the 505(b(2) pathway as proposed by Aquestive and that no further studies would be required before the proposed IND application is opened.
The FDA indicated that patients who resist the standard use of intramuscular injection in the treatment of anaphylaxis appear to have an unmet medical need, and that AQST-108 may potentially address some of those unmet needs.
“Anaphylaxis is a severe condition affecting a large population of patients for whom a major unmet need exists. The only options available to patients currently require an injection and AQST-108 would potentially bring major progress and positive change to patients.”
Feb. 05, 2020: The U.S. FDA moved closer on Tuesday to formulating standardized testing of talc-based products for the presence of toxic asbestos fibers.
Experts from eight different federal agencies explained about earlier recommendations at a day-long hearing. The session also featured input from 25 members of the public, which included industry legislative bodies, consumer advocates and testing experts.
“The next step will be producing a white paper after reviewing all the recommendations as an authoritative guide on the issue.”
Feb. 05, 2020: VIVUS, a biopharmaceutical company announced that the U.S.FDA has approved the sNDA for an improved formulation of PANCREAZE® (pancrelipase) Delayed Release Capsules that extends the shelf life to 36 months across all PANCREAZE dosages.
VIVUS worked closely with the Nordmark Arzneimittel GmbH & Co. KG, its manufacturing partner for the PANCREAZE, on the FDA approval pursuant to the terms of the amended contract manufacturing agreement announced in June 2019.
PANCREAZE is indicated for the treatment of the exocrine pancreatic insufficiency (EPI) due to cystic fibrosis or other conditions.
Approved in 2010, PANCREAZE is a pancreatic enzyme preparation consisting of the pancrelipase, an extract derived from the porcine pancreatic glands, as well as other enzyme classes, including porcine-derived lipases, proteases and the amylases. The pancreatic enzymes in the PANCREAZE act like digestive enzymes physiologically secreted by the pancreas.
About Pancreaze:PANCREAZE is a prescription medicine used in order to treat people who cannot digest food normally because their pancreas does not make enough enzymes due to cystic fibrosis or other conditions.
PANCREAZE help your body by using fats, proteins, and sugars from food. PANCREAZE contains a mixture of the digestive enzymes including lipases, proteases, and the amylases from pig pancreas. PANCREAZE is safe and effective in the children when taken as prescribed by the doctor.
Feb. 05, 2020: Alector, Inc., a clinical-stage biotechnology company pioneering immuno-neurology announced that the U.S.FDA has granted Fast Track designation to AL101 for the treatment of patients with progranulin gene mutations causative of frontotemporal dementia (FTD-GRN).
AL101, the company’s second product candidate designed in order to restore progranulin levels in the brain, is currently being evaluated in a Phase 1 trial in healthy volunteers.
“There is a clear and validated connection between progranulin expression in the brain and certain neurodegenerative disorders like FTD, Alzheimer’s disease and Parkinson’s disease.
At Alector, we are pioneering the development of the therapies targeting the progranulin pathway, with two product candidates being tested in the clinical trials,”
“AL101 is the second product candidate from our progranulin program in order to receive Fast Track designation from the FDA, underscoring the need for the new treatment options for these patients.
We look forward to working with the agency to advance AL101 as quickly as possible.”Fast Track designation is designed to facilitate the development and expedite the review of therapies for the serious conditions and fill an unmet medical need.
Programs with Fast Track designation may benefit from the early and frequent communications with the FDA, potential priority review, and additionally a rolling submission of the marketing application.
About AL101: AL101 is Alector’s wholly-owned human monoclonal antibody and second product designed in order to restore levels of progranulin in the central nervous system. Progranulin is a regulator of the immune activity in brain with genetic links to the multiple neurodegenerative disorders.
Feb. 05, 2020: BioXcel Therapeutics announced that its IND
application for BXCL501, the Company’s proprietary sublingual thin-film formulation
of dexmedetomidine, has received clearance from the U.S.FDA for the treatment
of opioid withdrawal symptoms.
“Opioid overdose is reported as the number one cause of death in the United States for those under the age of 50 years, and the distressing and challenging symptoms of opioid withdrawal are a primary reason for relapse.
Better treatment options are urgently needed to help manage the debilitating withdrawal symptoms and help this underserved population from continued opioid abuse. BXCL501, our investigational non-opioid therapy, may present key advantages to treating symptoms due to its intrinsic effectiveness and favorable delivery method.
Opioid withdrawal is an emotional and physiological medical condition which may be caused by the intense drive of the noradrenergic neurons that arise in the brainstem from the locus coeruleus.
The RELEASE trial is a Phase 1b/2 multicenter, randomized, double-blind, placebo-controlled, ascending dose study designed to determine the health, pharmacokinetics, tolerability and effectiveness of BXCL501 in patients with opioid withdrawal symptoms.
Patients are administered twice daily for five days to be randomized into several dose cohorts of BXCL501, or equivalent placebo.
BXCL501 BXCL501 is a potential first-in-class, proprietary sublingual thin film made of dexmedetomidine, a selective alpha-2a receptor agonist for the treatment of acute agitation.
This blog talks about Protocol parts and key information needed for any Data Manager to design CRF. Protocols are quite specific to studies but share some common information and that is included in this blog to develop a general understanding about protocol. As per ICH, followings are the parts of any protocol.
Protocol components:
Study title
Introduction/Background
Objectives and End Points
Study design and its rationale
Population (Inclusion/exclusion criteria)
Treatment
Informed Consent Procedure
Visit Schedule and Assessment
Efficacy assessment
Safety Assessment
Discontinuation of the Study
Data collection and its management
Statistical methods
Ethics
Quality Control and Assurance
Publication Policy
Reference
1. Study title: Protocol first page always has the title and Sponsor name
For example: “An Open label Phase III study to evaluate effectiveness of ATC-XX in treatment of Triple Negative Breast Cancer”
The data Manager can easy interpret about Phase, Design and
Target indication by just reading the study title.
2. Table of content: It is same as any book’s table of
content. Protocol is a big document so it is easiest way to navigate in the
protocol.
3. Introduction: It gives the overview of the disease, epidemiology and current treatments. It tells about investigational drug candidates and purpose of the study. Not important for designing CRF but quite useful while preparing study related presentations.
4. Objectives and End Points: Primary objective and secondary objectives and their associated end points are described in this segment. All study specific assessments are done considering primary and secondary points. All studies do not have secondary objectives.
5. Study design and its rationale: This segment provides the key information for the data manager. In case of blinded study, Clinical Data manager has to provide restriction based data base access. The site generally has Clinical research co-coordinator and Investigator access to add data into database. If it is adoptive design, then one has to be ready for many post production data base changes. This part gives the insight about rationale for the study design. If any particular combination of drugs is used or risk/benefit associated with any combination then this segment provide the rationale behind that.
For example, it is needed to blind prolactin for many antipsychotic interventional studies. It is well known that Antipsychotic drugs increase the level of prolactin and by knowing its level one can learn about subject’s randomized status. This segment gives information about number of participants, cohorts and randomization method. It describes the study arms and One CRF is dedicated for study arms in eDC. Many CRF pages can be dependent on particular study arm. Just note down the study arms and read all the protocol with respect to each arm because many data point including inclusion/exclusion criteria may differ in each study arm. It is one of the most important segments of a protocol from a data manager point of view.
6. Population (Inclusion/exclusion criteria): It is key information which determines which subjects meet the all set criteria to participate in a study. Inclusion criteria/exclusion criteria may be as simple as age/sex/Demography/Race and it may be as complicated as looking for a particular mutation in a tumor. Any subject, which does not meet all inclusion criteria but continue in study, may be the case of protocol deviations. Rescreening criteria should be checked in this section carefully. Sometimes Protocol allows rescreening after a particular time window eg one month. Rescreened subject may have a different subject number. Inclusion criteria/exclusion criteria numbers need to be captured in CRF. It is confusing sometime for <= or >= signs commonly used for laboratory values. Some protocol allows performing retest if initial laboratory results do not meet the inclusion criteria. Data Manager has to note these minute details.
7. Treatment: Description of all investigational drugs with their dosage form, route of administration, dosages, duration and frequency of administration and supply type (bulk, vials etc) can be seen here. Prohibited medication, concomitant therapy, infusion reaction therapy and prophylaxis regimens are described in this section. Prior and Concomitant therapy CRF, Dose administration record CRF are the two CRFs which gets 90 percents information from here. This part can be further divided into these parts. A) Subject numbering, treatment assignment and randomization (if study has this). B) Guidelines for dose reductions/modification, determination of Maximum tolerated dose (If it is Phase I study). C) Determination of dose limiting toxicities. D) Handling of study treatment and additional treatment E) Instruction on how to administer the drugs. F) Additional guidance if any applicable
8. Informed Consent Procedure: It suggests about things which
are needed to convey to subjects such as study related risk, any pregnancy
related restrictions, common side effect which are known about investigational
drugs. IRB/IEC approved informed consent form need to be signed by Subject
prior participating in a study. Informed consent CRF is commonest CRF among all
study related CRFs.
9. Visit Schedule and Assessment: This segment provide a description about visits, cycle in each visit, time window between each visits and assessment to be carried out at each visit and cycle. Data Manager must read the table given in protocol in this section. Vital sign, physical examination, laboratory assessment, imagine assessment, ECG, PK evaluation; RECIST evaluation (oncology studies), study drug administration time, post treatment evaluation, baseline assessments and study specific assessments are the common points. 70 percents CRF pages can be prepared by just this table.
10. Efficacy assessment: The procedure or check which can clearly tells whether investigational drug works or not. iRECIST and RECIST assessments are the common for oncology assessments and CRF for this is included in the database design. Measuring glucose level and Hba1c is done for diabetic studies quite commonly. It depends generally on primary and secondary end points in study. Safety Assessment: It describes the definition of adverse events, serious adverse event and reporting requirements. Duration, start date, end date, Outcome of adverse event, relationship to study drug, CTCAE Grades, are the common information required to report an adverse event. Protocol can provide study specific guidance for reporting the adverse event. CTCAE version is mentioned in the protocol. Protocol says about timing for SAE reporting, In general, 24 hours it should be reported to sponsor after learning of its occurrence. Data Manager read this section carefully as there is one dedicated CRF for this crucial information. Generally, AE is noted once subject signs the informed consent form and may be recorded even till follow up visit as per study requirement
11. Discontinuation of the Study: Protocol defines the circumstances under
which study drug can be withdrawn. Generally they are; Subject or guardian
decision, adverse event including death or major protocol deviation and early
termination of study by sponsor. Data manger has to understand under which
circumstance study treatment is discontinued and under which circumstance
subject is discontinued from the study. Even after discontinuation of study
treatment, subject can be in study. Protocol may suggest assessment before or
after discontinuation of study treatment.
12. Data collection and its management: It explains the process of data recording,
format (electronic or paper), site monitoring, and review and query
management. It also mentions the names
of dictionary for coding of concomitant medications, Medical history and
adverse event.
Recording keeping is done by sponsor designated portal which
is matching regulatory stanadard.
13. Statistical methods: Protocol describes the statistical method to analyse the date. It talks about analysis of primary end point, secondary end point, handling of missing values, assessment of subject dosing, identification of recommended dose (Phase I), and PK data. It can include additional information based on study.
14. Ethics: Protocol mentions the statement about its
commitment to follow ICH GCP and local guideline of particular country and
other related information such role of IRB and Investigator. It is not
important for CRF designing (Data Manager prospective)
15. Quality Control and Assurance: It contains the information about sponsor’s internal process of audit, standard operating procedure, all activities to meet GCP compliance. This segment does not have any particular information to be added directly into CRF.
16. Publication Policy : It says where the protocol will be
registered (eg clinicaltrial.gov), website of mode of publication of study
results. Every sponsor may have different policy for publication.
17. Reference: List of all reference used in protocol to
gather information.
Feb 04, 2020: MorphoSys AG announced the initiation of an expanded access program (EAP) in the U.S. for tafasitamab, an investigational anti-CD19 antibody.
The EAP may provide a way to tafasitamab for use in the patients with relapsed or refractory diffuse large B cell lymphoma (r/r DLBCL) in combination with lenalidomide.
According to the FDA, expanded access programs – sometimes called “compassionate use” – provide a pathway for the patient to receive an investigational medicine for a serious disease or condition.
They are frequently made available when there are no comparable or satisfactory alternative therapies to treat the disease or conditions; patient enrollment in the clinical trials is not possible; potential patient assistance justifies the potential risk of the treatment and providing the investigational medicine will not interfere with investigational trials that could hold up the medicine’s marketing approval for the treatment indication.
To qualify for the tafasitamab EAP, patients with r/r DLBCL need to assemble the EAP inclusion/exclusion criteria that are associated with the MorphoSys’ L-MIND study.
Treatment of the DLBCL patients in EAP is suggested with tafasitamab in the combination with lenalidomide according to the treatment schedule in L-MIND. The EAP will be available for the limited time while the FDA reviews MorphoSys’ Biologics License Application (BLA) for the tafasitamab.
