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FDA approves less invasive surgical approach for Abbott’s heart pump in order to help patients avoid Open Heart Surgery

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FDA approves less invasive surgical approach for Abbott’s heart pump in order to help patients avoid Open Heart Surgery

 Jan. 07, 2020: U.S. FDA approved Abbott’s HeartMate 3 heart pump that will allow more advanced heart failure patients to avoid open heart surgery.

The new, less persistent approach is designed in order to provide surgeons a choice in surgical method for patients receiving the HeartMate 3™ Left Ventricular Assist Device (LVAD), the industry’s leading heart pump.

Around 615,000 people in the U.S. are living with heart failure and almost 40% are considered to have reached an advanced stage where traditional therapies no longer work. Heart pumps are small, implantable mechanical devices that pump blood through the body in the people whose heart is too weak to do so on its own.

People living with a heart pump may be waiting for a heart transplant or may not be candidates for the transplant and will live with the device for the rest of their life.

Abbott’s HeartMate 3 heart pump can now be implanted using lateral thoracotomy – a surgical approach where an incision is made between a patient’s ribs to access the heart.

The approval is based on two studies – the ELEVATE study: a multi-center, controlled, observational registry collecting post-marketing data, and the LAT Feasibility study: a single arm, prospective, multicenter study.

Results of two trials found that the bleeding (requiring surgery), infection and arrhythmias were lower in the group implanted via the less-invasive surgical approach than those who underwent open heart surgery.

The HeartMate 3 received approval from the FDA in 2017 for the patients with advanced heart failure whose hearts are unable to circulate blood through the body, and are waiting for a transplant, known as bridge to transplant.

In 2018, HeartMate 3 was approved as a destination therapy for those individuals who require a new heart but are not eligible for a transplant.https://abbott.mediaroom.com/2020-01-07-FDA-Approves-Less-Invasive-Surgical-Approach-for-Abbotts-Heart-Pump-to-Help-Patients-Avoid-Open-Heart-Surgery

Dietary supplements containing zinc and folic acid marketed as a treatment for male infertility does not improve pregnancy rates, sperm counts or sperm function

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Dietary supplements containing zinc and folic acid marketed as a treatment for male infertility does not improve pregnancy rates, sperm counts or sperm function

Jan 07, 2020: According to a study conducted by the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD),Dietary supplements containing zinc and folic acid — marketed as a treatment for male infertility — do not appear to improve pregnancy rates, sperm counts or sperm function.

Zinc is an essential mineral for the sperm formation, and folate, natural form of folic acid, depends on zinc to help form DNA in the sperm. Previous studies of these nutrients for the treatment of male infertility have produced conflicting results.

In the current trial, researchers enrolled 2,370 couples scheduling infertility treatments in four U.S. cities and their surrounding areas.

The men were assigned at random in order to receive either a placebo or a daily supplement containing  5mg of folic acid and 30mg of zinc.

Live births does not vary considerably among the two groups: 404 (34%) in the supplement group and 416 (35%) in the placebo group. Likewise, the groups did not differ among various measures for sperm health, such as sperm movement, shape and total count.

However, the quantity of sperm DNA fragmentation—broken DNA in the sperm—was higher in the supplement group (29.7%), compared to the placebo group (27.2%). Studies related a high rate of sperm DNA fragmentation to the infertility. https://www.nih.gov/news-events/news-releases/zinc-folic-acid-supplement-does-not-improve-male-fertility-nih-study-suggests

BMS Completes Divestment of its oral solid, biologics, and sterile product Manufacturing and packaging Facility in Anagni, Italy

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BMS Completes Divestment of its oral solid, biologics, and sterile product Manufacturing and packaging Facility in Anagni, Italy

JAN 07, 2020: Bristol-Myers Squibb Company announced that it has completed its previously announced divestment of its oral solid, biologics, and sterile product manufacturing and packaging facility in Anagni, Italy, to Catalent Inc.

The divestiture is a part of Bristol-Myers Squibb’s strategy to make simpler and to realign its business portfolio to address changes in its business and the future desires of its evolving pipeline.

The Anagni facility manufactures and the packages cardiovascular, anticancer, metabolic and anti-inflammatory medicines as well as non-penicillin-based antibiotics, antivirals, analgesics as the injectables and biologics.

The Company is now focusing resources on its highest priorities of discovering, developing and delivering transformational medicines for the patients facing serious diseases. https://news.bms.com/press-release/corporatefinancial-news/bristol-myers-squibb-completes-divestment-manufacturing-facili

FDA Ok’s Teleflex for Expanded Indication of the UroLift® System for Treatment of Larger Prostates, Up to 100cc

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FDA Ok’s Teleflex for Expanded Indication of the UroLift® System for Treatment of Larger Prostates, Up to 100cc

Jan. 07, 2020: Teleflex announced that the U.S. Food and Drug Administration (FDA) has granted the company an expanded indication for the use of its UroLift® System to treat larger prostates, between 80cc and100cc.

This minimally invasive, in-office treatment provides rapid relief and recovery from the symptoms of benign prostatic hyperplasia (BPH), is non-cancerous enlargement of the prostate that occurs as men age. The situation affects over 40 million men in the United States alone. 

More than 40% of men in their 50s have BPH and over 80% of men in their 70s have BPH. The symptoms of BPH can include frequent urination and can cause loss of productivity, depression and decreased quality of life. If left untreated, the condition can worsen over time and cause permanent bladder damage.

Data presented to the FDA demonstrates that the UroLift System treatment is safe and effective in men with prostate sizes between 80cc and 100cc, with outcomes similar to the L.I.F.T. randomized controlled trial. Further, there are no discernable differences in reported adverse events, indicating a comparable safety profile.

There is also a strong and growing body of clinical evidence supporting the safe, effective use of the UroLift System, including a large retrospective real-world study which highlights the results of 1,413 patients who inward the UroLift System treatment across 14 sites in North America and Australia.

Results were consistent with those seen in previous clinical studies of the UroLift System treatment, and included patient subgroups—such as those in retention, with large prostates and comorbidities such as diabetes and prostate cancer—not commonly seen in clinical trials. https://fda.einnews.com/pr_news/506556989/teleflex-announces-fda-clearance-for-expanded-indication-of-the-urolift-system-for-treatment-of-larger-prostates-up-to-100cc

Valneva Announces End of Phase 2 Meeting with the FDA for its Chikungunya Vaccine Candidate

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Valneva Announces End of Phase 2 Meeting with the FDA for its Chikungunya Vaccine Candidate

Jan 07, 2020: Valneva SE announced that the End Of Phase 2 (EOP2) meeting has been scheduled with the U.S. FDA on February 24, 2020 for its single-shot chikungunya vaccine candidate, VLA1553.

This plans will be presented for Phase 3 clinical studies and licensure. VLA1553 has been awarded Fast Track designation by the FDA and may be eligible for a Priority Review Voucher.In November 2019, Valneva reported final Phase 1 results confirming VLA1553’s outstanding immunogenicity and safety profile,  also completed all required non-clinical studies requested by the FDA.

Phase 1 Clinical Study VLA1553-101 was a randomized, observer-blinded, multicenter, dose-escalation Phase 1 clinical study investigating three dose levels of VLA1553, administered as a single immunization.

It enrolled 120 healthy volunteers, 18 to 45 years of age, in the United States. Subjects were randomized into three different study groups to accept one of three dose levels (30 subjects in the low and medium and 60 subjects in the high dose group).

The protocol includes a re-vaccination with the live-attenuated vaccine candidate VLA1553 at Month 6 (for 30 subjects in the high dose group) or Month 12 (for all others) to confirm that a single vaccination will be sufficient to induce high titer neutralizing antibodies and protect subjects from vaccine-induced viremia (intrinsic viral challenge).

Study participants were followed until 13 months after initial vaccination. An independent Drug Safety Monitoring Board (DSMB) continuously oversaw the study and reviewed safety data.

Additional information, including a detailed description of the study design, eligibility criteria and investigator sites, is available at ClinicalTrials.gov (NCT03382964).Chikungunya is a mosquito-borne viral disease caused by the chikungunya virus (CHIKV), a Togaviridae virus, transmitted by the Aedes mosquitoes.

Clinical symptoms include acute onset of fever, debilitating joint and muscle pain, headache, nausea and rash, potentially developing into long-term, serious health impairments https://fda.einnews.com/pr_news/506572181/valneva-announces-end-of-phase-2-meeting-with-the-fda-for-its-chikungunya-vaccine-candidate

BAVENCIO (avelumab) extensively Improved Overall Survival in Patients with Locally Advanced or Metastatic Urothelial Carcinoma

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BAVENCIO (avelumab) extensively Improved Overall Survival in Patients with Locally Advanced or Metastatic Urothelial Carcinoma

Jan 06, 2020: Merck and Pfizer Inc, announced the Phase III JAVELIN Bladder 100 study met its primary endpoint on the whole survival (OS) at the planned interim analysis.

In this study, patients deals with previously untreated locally advanced or metastatic urothelial carcinoma (UC) whose disease does not progress on induction chemotherapy and who were randomized to accept first-line maintenance therapy with BAVENCIO® (avelumab) and best supportive care (BSC) lived considerably longer than those who received BSC only.

A statistically noteworthy improvement in OS was demonstrated in the BAVENCIO arm in each of the co-primary populations: all randomized patients and patients with PD-L1–positive tumors.

 The safety profile for BAVENCIO in the trial was reliable with that in the JAVELIN monotherapy clinical development program.

The results of the study will be submitted for the presentation at an upcoming medical congress and shared with the U.S. FDA and other health authorities.

UC accounts with 90% of all bladder cancer. When the bladder cancer is metastatic, the five-year survival rate is 5%, then combination chemotherapy is currently the first-line standard of care for the patients with advanced disease, but despite high initial response rates, strong and complete responses following first-line chemotherapy are uncommon, and most patients will eventually experience disease progression within nine months after initiation of treatment.

FDA approved BAVENCIO in 2017 for the treatment of patients with locally advanced or metastatic urothelial carcinoma who have disease progression during or following platinum-containing chemotherapy, or who have disease progression within 12 months of neo-adjuvant or adjuvant treatment with the platinum-containing chemotherapy.

This indication is approved below accelerated approval based on tumor response and duration of response. JAVELIN Bladder 100 is the confirmatory study for the conversion towards full approval.
https://www.merckgroup.com/en/news/bavencio-bladder-100-06-01-2020.html

FDA approved AstraZeneca’s Farxiga(dapagliflozin) for Priority Review for the patients with heart failure with reduced ejection fraction

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FDA approved AstraZeneca’s Farxiga(dapagliflozin) for Priority Review for the patients with heart failure with reduced ejection fraction

Jan 06, 2019: AstraZeneca announced the U.S. FDA has accepted a supplemental New Drug Application (sNDA) and granted Priority Review for Farxiga (dapagliflozin) in order to reduce the risk of cardiovascular (CV) death or the worsening of heart failure (HF) in adults with heart failure and reduced ejection fraction (HFrEF) with and without type-2 diabetes (T2D). 

Farxiga is the first-in-class, oral once-daily selective inhibitor of human sodium-glucose co-transporter 2 (SGLT2).

The sNDA was based on the results from the landmark Phase III DAPA-HF trial published in Sep 2019 in The New England Journal of Medicine, which showed Farxiga on top of standard of care reduced the incidence of the complex result of CV death or the worsening of HF versus placebo.

Farxiga is indicated as a monotherapy and as part of combination therapies in order to improve glycaemic control in adults with T2D. In October 2019, the FDA also approved Farxiga to reduce the risk of hospitalisation for the heart failure in patients with T2D and established cardiovascular disease or multiple CV risk factors. https://www.astrazeneca.com/media-centre/press-releases/2020/farxiga-granted-fda-priority-review-for-patients-with-heart-failure-with-reduced-ejection-fraction-06012020.html

Want to become a Pharmacist in U.S.? Then follow the journey to registration

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Want to become a Pharmacist in U.S.? Then follow the journey to registration

Becoming a U.S. citizen is difficult, but it is doubly difficult to become a Pharmacist in the U.S. It’s a highly competitive industry, possibly because the U.S. is one of the dynamic forces that is pushing the global pharmacy profession forward.

And, pharmacists in the U.S. are among the highest-paid pharmacists in the world, earning an average salary of around $120,950, according to the U.S. Department of Labor Bureau of Labor Statistics. Also, there are tons of benefits of becoming a pharmacist in the U.S., including:many opportunities for continuing education, the ability to practice in a variety of clinical settings, a strong community of the Pharmacy professionals and a salary that can provide a good standard of living.

If you are making an allowance for becoming a pharmacist in the U.S., here’s some information that will definitely help you achieve your goal. The journey to registration is as follows:

1. Apply to Education Credential Evaluators (ECE) in order to gain recognition of equivalence of your foreign qualifications. The spirit of the application is to demonstrate that your foreign undergraduate training is equal to that of someone educated in the US.The ECE requires two copies of the following documents: official university transcript proof of degree https://www.ece.org/

2. Apply to the National Board of Pharmacy (NABP) to start the Foreign Pharmacy Graduates Equivalency Committee certification process The NABP requires a single copy of your pharmacy registration document. Records sent to the ECE and NABP from the issuing education institution (either directly or via you) must be in a sealed envelope with the issuing body’s stamp over the seal. An application with demographic information also needs to be certified by a US-based notary. Once the NABP receives a satisfactory general evaluation report from the ECE,  it will then send you an authorisation to test letter (ATT) allowing you to sit in the Foreign Pharmacy Graduate Equivalency Examination (FPGEE). https://nabp.pharmacy/

3. Register online for the FPGEE and the test of English as a foreign language internet-based test (TOEFL ibt) The FPGEE is held bi-annually at a Pearson VUE Test Centre. The TOEFL ibt is available frequently at an Educational Testing Service Centre. These examinations can be done in any order, If English is not your first language, you might want to study for the FPGEE first.

4. Register as a pharmacy intern with the state board of pharmacy and start recording working  hours This can be done as soon as you are approved to sit in the FPGEE. You will need to find a pharmacist who will be your instructor at your internship facility and register as a pharmacy intern with the State Board of Pharmacy. Once got registered, start “recording” 500 to 1,500 intern hours (depending on the state). https://www.pharmacyexam.com/?gclid=Cj0KCQiA04XxBRD5ARIsAGFygj_XgIbvP4Vluhz6emU0weXlDCz6nQPTBUrS6O7RIYEAEAfdyMCIexsaAqazEALw_wcB

5. Take the FPGEE and the TOEFL ibt Results for the FPGEE can take around eight weeks to arrive. Results for the TOEFL ibt are available online within 10 days. Once you have set both of these tests, your entire assortment undergoes final evaluation by the FPGEC (which can take up to 10 weeks) before you are issued with a certificate. https://www.ets.org/toefl

6. Submit records of internship hours to the State Board of Parmacy/PCS once completed This has to be done before you apply for the North American Pharmacy Licensure Examination (NAPLEX) and Multistate Jurisprudence Pharmacy Examination (MJPE). Use the proper form and be prepared to submit proof of worked hours.

7. Once approved, apply for and take the NAPLEX and the MJPE and apply for pharmacy licensure Eligibility to take these examinations also varies according to state and your local board needs to issue another ATT first. In Massachusetts there is a combined form for the NAPLEX, MJPE and pharmacy licensure (again there is a demographic component and it needs notarising). An MJPE must be passed in every state a candidate wishes to practise in, since pharmacy law varies from state to state. Other things to consider can be some additional costs, such as: Charges for issuing documentation, notary fees, textbooks/online resources, postage and packaging and any examination repeat fees. Pharmacist’s salaries are higher in the US that can  provide Financial support while applying for the job. In hospital pharmacy, working patterns incorporate variable shift-work. https://nabp.pharmacy/programs/naplex/

  FPGEE TOEFL ibt NAPLEX MJPE
When it can be taken Bi-annually at dates set by NABP (usually April,September)  Any time  Any time  Any time 
Style of examination 250 multiple choice questions on anything from  undergraduate degree. Some familiarity is needed with US-specific pharmacy (eg,law, drug names, terminology, the healthcare model)  Reading, writing, listening and speaking sections.Computerised test with a series of questions.  185 MCQs to test ability to measure pharmacotherapy and therapeutic outcomes, prepare and dispense medicines, and implement and evaluate information. Often in a scenario-based format. Similar in content to theUK registration assessment  90 MCQs. Combines federal- and state-specificquestions to test pharmacy jurisprudence knowledge 
Test centre Any Pearson VUE Test site Any ETS Test site Any Pearson VUE Test site Any Pearson VUE Test site  
Length of examination 5.5 hours (30 minutes’ break in middle)  Four hours (10 minute break in the middle)  4.25 hours (optional 10 minute break)  Two hours (nobreak) 
Cost ($1?£0.65) $800 for examination and $85 for ECE report. Approximately $400 for copies of reports to be sent to ECE and NABP, notary fees, postage and packaging, registration as intern and registration as pharmacist $160 $421 (combined total for NAPLEX, MJPE, licensure application). May vary depending on state. $421 (combined total for NAPLEX, MJPE, licensure application). May vary depending on state.
Pass mark  Scaled score of 75 (range 0–150) Speaking 26 Writing 24 Listening 18 Reading 21    Scaled score of 75  Scaled score of 75 

How to Become a Pharmacist in the U.S. – For Non-U.S. Citizens

https://www.pharmaceutical-journal.com/test-tomorrows-pharmacist/tomorrows-pharmacist/how-to-gain-your-licence-to-practise-as-a-pharmacist-in-the-us/11128041.article?firstPass=false

Drug Approval Process in India

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Drug Approval Process in India

Regulatory Bodies for Drug approval in India

The Drug and Cosmetic Act 1940 and Rules 1945 was enacted to regulate the import, manufacture, distribution and sale of drugs and cosmetics in India. The Central Drugs Standard Control Organization (CDSCO) is the regulatory body (as FDA in the USA) in India and drug controller general of India heads the CDSCO.

In 1988, the Indian parliament added Schedule Y to the Drug and Cosmetics Rules 1945. It provides the guidelines and requirements for clinical trials. It was further amended in 2005 to make it more aligned with international standard.

Definition of New drug :

Rule 122E under The Drug and Cosmetic Act 1940 and Rules 1945 defines the ‘new drug:

“drug, including bulk drugs substance (or phytopharmaceutical drug) which has not been used in the country to any significant extent under the conditions prescribed, recommended or suggested in its labelling and has not been recognised as effective and safe by the licensing authority for the proposed claims, drugs already approved for certain claims, which are now proposed to be marketed with modified or new claims (viz., new indications, dosage, dosage forms or routes of administration), or fixed-dose combinations of two or more drugs, individually approved earlier for certain claims, which are now proposed to be combined for the first time in a fixed ratio, or a new fixed ratio, with certain claims, (viz., indications, dosage, dosage forms or routes of administration)”

It is important to point out that “new drug shall continue to be considered as a new drug for a period of four years from the date of its first approval or its inclusion in the Indian Pharmacopoeia whichever is earlier”

Information and data required for approval of clinical trial and/or to import or manufacture of a new drug for marketing in India are clearly described in 122A, 122B and 122D, 122DA, 122DAA, 122E rules of Drugs and Cosmetics Rules and Appendix I, IA and VI of Schedule Y.

Form 29: Applicant/Sponsor has to take NOC from CDSCO. Based on this, the applicant can take license in form 29 from the state Licensing authority to manufacture test batches of new drugs (for development and generation of data).

New Drug Approval Process in India:

  • New drug candidate being developed in India and not Marketed anywhere else.
  • New Drug candidate being developed outside India and not marketed anywhere in the world.
  • If the drug is already approved/marketed in other countries but not approved in India.
  • Drug candidate which are approved in other countries and being used from several years:

New drug Discovered in India and not Marketed anywhere else:

If a new substance is discovered in India and not approved in any part of the world, then all Phases of the trial (Phase I to Phase III)  needs to be conducted to get the Market authorization.

New Drug candidate being developed outside India and not marketed anywhere in the world.

An applicant has to submit Phase I data along with the application. Licensing Authority reviews the Phase I data and Based on this, they may grant permission to conduct Phase II and subsequent Phase III trial in India concurrently with another global trial of a drug candidate. It is also possible that regulatory authority may ask to repeat the Phase I trial in India.

If the drug is already approved/marketed in other countries but not approved in India.

  • In general, if a drug candidate is approved in one or more countries like USA, UK, Canada, European Union, Japan, and Australia, then it will be considered for approval of manufacture/import and marketing in India.
  • If the sponsor is seeking approval of the drug candidate in India which is already have been approved/Marketed in other countries,  then sponsor or applicant has to conduct Phase 3 data on at least 100 subjects across 3 to 4 centre to confirm the safety and efficacy of drug candidate in  Indian population.
  • The regulatory authority may ask any additional non-clinical and clinical data based on the nature of drug candidate and information available
    It is important to note that if a drug candidate is discovered in India and it is not marketed in any other countries,  then the applicant has to conduct Phase 3 studies in 500 subjects across 10 to 15 centre.

Scenarios when clinical trials are not required to be conducted for drug approval in India

If the drug is approved in other countries and applicant is seeking approval to market/manufacture/import this drug in India then it is not necessary to conduct clinical trials in India if drug candidate meets any of the following requirements.

The requirement of submitting results of local clinical trials may not be necessary As per the provisions given in the Drugs & Cosmetics Rules.

  • serious/life-threatening condition,
  • Diseases which carries special relevance in Indian Population
  • No satisfactory  therapeutic option available 
  • Unmet medical need exist
  • Rare Disease
  • If a drug candidate offers added a significant advantage over the existing treatment options for a specific disease

Drug candidate which are approved in other countries and being used from several years:

In this case, a clinical trial may be waived off in India as per rule 122 A of drug and cosmetic Act if the sufficient published safety data is available, however, how many years will be considered as “ several years” is not clear.

Important facts:

Category A: Clinical trial   (approved in the U.S., Britain, Switzerland, Australia, Canada, Germany, South Africa, Japan and the European Union) are eligible for fast-tracking.  Because these clinical trials have been approved already by a competent and robust process by a well developed regulatory framework, so the India regulatory authorities can rely on data to some extent for dug approval in India.

Category B: Apart from above, All trial falls under B. India Regulatory authority reviews the data and scrutiny process takes 16-18 weeks.

Investigational New Drug Application (IND): Data of chemistry, manufacturing, control and animal studies needs to be submitted to regulatory authority along with other documents such as protocol, investigator’s brochures (IB), and informed consent documents. A copy of this application needs to be submitted to the ethical committee as well. DCGI reviews this application and applicant can conduct a clinical trial in India once it gets approval for this.

New Drug Application: New Drug Registration using form 44 along-with preclinical, clinical and other relevant data such as marketing status in other countries, testing protocol, label, product monograph, needs to be submitted to the regulatory agency. It may take 12-18 months to review the application.

Approval of Drugs in India, with some modification over existing approved drug

Pharmaceutical companies may change an already approved  product in many ways namely:

  • New proposed Indications
  • Change in dosage 
  • dosage form (including sustained release dosage form)/formulation change
  • Route of administration (ie oral from IV)

This type of application differs from the New drug applications as in this case, drug regulatory agency relies on safety and efficacy data of already approved drug at least in part.

Please note this pathway or application is like an sNDA in The regulatory authority may ask some additional clinical or non-clinical data which is decided by case by case basis.

In the below scenario, the regulatory authority may require less or no additional data of  Animal Pharmacological, Animal Toxicological and clinical studies.

  •  If the drug is already approved by other countries for the proposed indication
  • Availability of evidence showing no difference in the metabolism of the drug due to ethnic differences
  •  Clinical data is available supporting benefit-risk ratio in favour of drug  candidate for the proposed new claim 
  • Package insert from other countries shows that there is no new safety concern if the drug is given to the Indian patient for the new claim.
  •  if new claim covers serious or life-threatening diseases or any disease which are quite relevant to India

Note: Comparative clinical trials and bio-equivalence study comparing the modified release formulation with the immediate-release formulation is required to be conducted if the applicant seeks approval for Modified release product. In case if the proposed formulation is already approved then comparative bio-equivalence study is recommended. Further guidance can be sought from CDSCO.

You can follow the blog of Fixed-Dose Combination Approval Process to have a better understanding of how India is approving fixed-dose combination.

Ref: https://journals.sagepub.com/doi/full/10.1177/0971721818762931
http://www.globalresearchonline.net/journalcontents/v13-2/004.pdf
https://cdsco.gov.in/opencms/opencms/en/Home/

ECOG Performance Status

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ECOG Performance Status

ECOG Performance Status-scale was developed by Eastern Cooperative Oncology Group. Now this group is a part of ECOG-ACRIN Cancer Research Group.
This scale was published in 1982.

Importance of ECOG performance status:

  • It provides standard criteria to measure the impact of disease on patient’s daily living abilities/performances for example, ability to walk, Office work etc.
  • It helps sponsor to recruit Desired population in oncology studies.
  • It also provides considerable information about treatment effect on patient’s level of functioning.
  • Data Manager can get the information about this while reviewing the protocol in oncology trials as all protocol clearly mentions which performance Grading system needs to be used. (other performance Grading System: Karnofsky Performance Status)
  • Please refer the below table which is taken from ECOG-ACRIN Cancer Research Group website
GradeECOG Performance Status
0Fully active, able to carry on all pre-disease performance without any restriction
1Restricted in the physically strenuous activity but ambulatory and able to carry out work of the light or sedentary nature, e.g., light house work, office work
2Ambulatory and capable of all selfcare but not able to carry out any work activities; up and about more than 50% of waking hours
3Capable of only limited selfcare; confined to bed or chair more than 50% of the waking hours
4Completely disabled; cannot carry on any selfcare; totally confined to the bed or chair
5Dead

Source: https://ecog-acrin.org/resources/ecog-performance-status

Orphan Drug Designation in US, EU and Japan

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Orphan Drug Designation in US, EU and Japan

Orphan Drug Designation in USA:

There are two routes (criteria) for obtaining orphan designation of a drug and biologics for a rare disease or condition.
Orphan drug designation and approval may be granted if application meets one of the following criteria.

(1)If a  product is intended for  treatment, prevention or diagnosis a  disease or condition that has a prevalence of less than 200,000 Individuals in USA.(FDC Act § 526(a)(2)(A))

Or

(2) even if a disease or condition affects 200,000 or more individuals, then also sponsor can seek orphan drug designation for its product.

It can only be possible if a sponsor can show that there is no reasonable expectation that the costs of drug development and making available to market can be recovered by its sale in USA. (FDC Act § 526(a)(2)(B) cost recovery provisions of the act)

Orphan Drug Designation in European Union

In order to get qualified for orphan designation, a drug candidate must meet a number of criteria:

  • Drug candidate must be intended for the treatment, prevention or diagnosis of a life-threatening or chronically debilitating diseases.
  • the prevalence of the condition in the European Union must not be more than 5 in 10,000 or there must be no reasonable expectation that the costs of drug development and making available to market can be recovered by its sale in the EU. 
  • “no satisfactory method of diagnosis, prevention or treatment of the condition concerned can be authorized, or, if such a method exists, the medicine must be of significant benefit to those affected by the condition” as per EMA

Related: The Drug approval process in EU:
Drug Approval process-FDA

Orphan Drug Designation by JAPAN:

The drug candidate (and Medical Device) Should meet the following criteria to get orphan drug Designation:
The number of patients who may use the drug or medical device should not exceed 50 000 in Japan.

Intended indication for  the treatment of serious diseases, including difficult-to-treat diseases.

“Additionally, they must be drugs or medical devices for which there are strong medical needs that meet one of the following requirements.

1. No appropriate alternative drug/medical device or treatment available
Or

2. High efficacy or safety is expected compared with existing products”
There should be a theoretical rationale for the use of the product for the target disease, and the development plan should be appropriate.” as per MHLW

References: http://www.fdalawblog.net/2009/02/the-rarely-used-cost-recovery-path-to-orphan-drug-designation-and-approval/
https://www.mhlw.go.jp/english/policy/health-medical/pharmaceuticals/orphan_drug.html
https://www.fda.gov/industry/developing-products-rare-diseases-conditions

AVITA medical receives U.S. FDA investigational device exemption approval of clinical feasibility study in order to evaluate Recell system for Vitiligo

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AVITA medical receives U.S. FDA investigational device exemption approval of clinical feasibility study in order to evaluate Recell system for Vitiligo

Dec 30, 2019: AVITA Medical Limited announced that the U.S. FDA has approved the company’s Investigational Device Exemption (IDE) application to conduct a feasibility study evaluating the safety and effectiveness of the RECELL® Autologous Cell Harvesting Device (RECELL® System) for repigmentation of depigmented lesions associated with stable vitiligo.

Vitiligo affects approximately 6.5 million people in the United States, rivalling the predominance of psoriasis. Conversely, there are limited treatment options available to patients to permanently restore skin pigmentation.

Vitiligo is a disease that causes loss of color, or pigmentation, in patches of skin that impacts the quality of life for those living with the condition.

Currently, there is no cure for vitiligo, nor a universally accepted method for limiting the spread of the disease.AVITA Medical will collaborate with a leading medical centre in order to conduct a pilot study with 10 patients, have vitiligo lesions that have been stable for at least one year.

Areas of the vitiligo lesion will be randomly treated with slightly altering cell suspensions prepared using RECELL to confirm response rates and best possible suspension parameters. https://www.avitamedical.com/uploads/pdf/AVH-Press-Release-Vitiligo-IDE-Approval-30DEC2019-FINAL.pdf

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