RAC US Exam Question No 42

Question No 42:

Devices that are exempt from premarket notification are:

A. All Class I devices
B. Some Class I devices
C. Most Class I devices and some Class II devices
D. All Class I devices and some Class II devices

Answer: C

Regulatory Recon: Brexit Fallout: How Will MHRA and EMA Handle the Split?

Headlines: US

• Medivation Sanofi slams 'deceptive' coup says the saga continues fusion (fierce) (Medivation)
• Why US consumers are of two minds when it comes to pharmaceutical companies (Forbes)
• FDA defends response to the final data of Medtronic Infuse (MassDevice)
• FDA committee vote whether reductions in medication for diabetes cardiac death (Reuters)
• White House veto threat Zika Republicans plan funding (Reuters) (STAT)
• A new report urges the FDA to clarify the use of real-world testing (Focus)
• What is the composition of the private sector to help fight Zika? (Blog of Health Affairs)
• Vote "possible cures Senate in July, President HELP Says (Bloomberg)
• Senator renews its opinion Pharma Ties on federal commission (ABC)
• asks the defective filter leukocyte reduction device Recall (Focus)
• Why we inherit mitochondrial DNA only our mothers? (New York Times)
• They are scientists fraudsters deserve a second chance? (STAT)
• PwC medical cost trend: Behind the figures of 2017 (PwC)
• Four ways to address ethical tensions around accelerated approval of new prescription drugs (Health Affairs blog)

Headlines: International

• Brexit Conundrum: How MHRA Working with EMA Moving Forward (Focus) (Reuters) (ferocious) (Political)
• Statements Brexit (EFPIA) (ABPI) (leaders of EU states) (EP Vantage)
• full coverage of the referendum on the EU (The Telegraph) (BBC)
• EMA warns that tablets and oral suspension NOXAFIL have different doses and are not interchangeable (EMA)
• Adempas not for use in patients with pulmonary hypertension caused by idiopathic interstitial pneumonia (EMA)
• EMA recommends six new drugs, including cell therapy for approval (EMA)
• Gavi committed $ 27.5 million for the Pilot Program candidate against malaria WHO as more funds (focus) vaccine
• ISO pharmaceutical sector gets new tool for drug identification (ISO)
• Soon regulatory reform in Thailand? ($ PharmAsiaNews-)

USA .: Pharmacy and Biotechnology

• Biogen in negotiations to sell the stainless steel plant in 2,000L MA (BioPharma-Reporter)
• BIO opposes HB to reduce Biological Exclusivity From 12 to 7 years (Focus)
• Fill a prescription? You might be better to pay cash (KHN)
• FDA extends comment period hepatitis C Guidance for Development (FDA)
• FDA extends comment period for the guide special protocol assessment (FDA)
• Warning Letters FDA: Warning signs are still there (Compliance Monitoring Pharmaceutical)
• AstraZeneca expects to save FluMist in the United States with foreign data (Pink information- $)
• precious sets late stage pipelines in 2017 for some major events (EP Vantage)
• "Shop" CBER Role: Mounting In the Oncology Center of Excellence ($ RPM Report)
• Ocaliva ICER report is an important step forward, but too expensive ($ PharmaLetter-)
• Bye, Bye exenatide Teva to take GLP-1 agonist generic two years before AstraZeneca (Pink detailed $)
• FDA life of influenza vaccine Flublok (DSN) extends
• Why Amgen biotechnology Goliath is strong long-term investment (Street)
• Diabetes medications are expensive worth? (MedPage)
• FDA approves changes (vigabatrin) Sabril® REMS Program (Press)
• Vibrant and flourishing of Life Sciences Ecosystem Home North Carolina Wilmington (Forbes)
• Focus Therapy - product amyloidosis is still Alnylam answers (EP Vantage)
• Agreement to combat Zika virus stimulates NIH, CDC's efforts, but offers no FDA Review Dollars ($ Detailed Gray)
• Epizyme NABS agreement with Genentech combined tests for the candidate of the NHL (fierce)
• biosimilar rituximab in patients naive biological rheumatoid arthritis (GABI)
• Q & A with rare mom and CEO of biotechnology Karen Aiach (Boston Biz Journal)
• Pharma veteran Stephen Friend Bites health data supply Apple (Xconomy)

United States: pharmaceutical and biotechnology products: results of clinical studies, documents and Designations

• FDA Approves Expanded Label for Avycaz (Pharmafile) Allergan
• A new study shows Baxter telemedicine to increase access to care and reduce hospitalizations (Press) Perception

Medical devices in the US:

• GI Dynamics data reveals EndoBarrier final completion of the test (fierce)
• Returns the table FDA MDUFA high and low resolutions ($ detailed Gray)
• rheumatoid arthritis market approach 400m test $ 2,022 (BioSpectrum)
• A new blood test is Epic Sciences patients with PARP inhibitors (MedCityNews)
• The future of the devices devices (as we know them today) (MassDevice)
• Odors are the last frontier of intelligent sensors (MassDevice)
• The evaluation of rapid tests commercially rage reveals serious problems with the accuracy (RGT)
• The first step to navigate Regulatory combination products Road: pmoa (GxP Lifeline)
• Roche receives FDA approval for procalcitonin (PCT) analysis to help physicians to effectively assess the risk of sepsis and treat patients with sepsis (Press)

United States: Assortment and government

• Appeals court confirms the victory over Thermomedic Exergen (MassDevice)
• Apotex litigation Drops counterclaims Sham Litigation Neupogen and Neulasta biosimilar (Biological Blog)
• Barred experts, he says patients have Bayer IUD cases ($ Law360-)
• Florida Physician pleads guilty to fraud - years after complaints about their prescription (ProPublica)
• Janssen accuses media provider HyClone Celltrion patent violation (BioPharma-Reporter)
• scheduled for wrongful death in the bone cement Norian XR judgment (MassDevice)
• Pharma accuses payers use money to influence the monitoring of expenditure of the United States (fierce)

Upcoming Meetings and Events

• Calendar of the Advisory Committee of the FDA
• Webinar for final FDA guidance: Use of existing clinical data for extrapolation to pediatric uses of medical devices - August 8, 2016
• EMA: Workshop on testing of one arm (SAT) in Oncology - June 30, 2016

Europe

• France plans to invest € 670 million in genomics, personalized medicine (GenomeWeb)
• CLL drugs Genmab receives negative opinion from the EMA Commission (Pharmafile)
• Astellas UK ABPI suspended for "deception" and other cultural defects (Pharmafile)
• Belle rejects Celgene drugs to treat cancer of the blood (Pharmafile)
• EC grants marketing authorization for HIV Odefsey (EPR)
• AstraZeneca rejects Belle Tagrisso for lung cancer in the preliminary recommendation (Pharmafile)
• Most Borderline / Combo products to escape the high risk rating in the EU ($ detailed Gray)
• St. Jude wins the CE mark, launches algorithm SyncAV CRT (MassDevice)
• Trulicity gets the seal of approval for Welsh NHS (PharmaTimes)

Asia

• Takeda (TKPYY) Returns some molecules, the products of Amgen (AMGN) (Biospace)
• Zhejiang Hisun anti-influenza drug market Fujifilm China ($ PharmaLetter-)
• Turkey Price Pressure hits profits and market growth ($ PharmAsiaNews-)

India

• Glenmark FDA approval for new generic bags (BioSpectrum)
• Ranbaxy Dinesh Thakur Whistle Blower prosecution suggests strict laws to fix the pharmaceutical industry (Economic Times)
• FDI in the pharmaceutical industry: drug supply NLEM to keep for 5 years (Economic Times)
• pharmaceutical sector grow 20 percent in the relaxed FDI rules: Ananth Kumar (Economic Times)

Australia

• Centinel Spine Australia wins the game STALIF The implant (MassDevice)

Zika

• Without federal funding, counties are prepared to face alone Zika (Washington Post)

Other international organizations

• The reasons for the success of a company in the generic drug market Sudan (GABI)

RAC US Exam Question No 41

Question No 41:

A sponsor intends to submit a Special Protocol Assessment (SPA) request for a clinical trial that will form the primary basis of an efficacy claim in an NDA. Which of the following is TRUE?

A. The sponsor should submit the SPA request within 30 days following the start of the trial to expedite FDA feedback
B. An SPA provides an opportunity to focus on general drug development issues
C. The SPA request will be handled as a request for a Type B meeting
D. A sponsor can submit a revised protocol while the agency is reviewing an earlier version of the same protocol

Answer: D

EMA Tries to Assuage Professors on Assumptions Linked to Adaptive Pathways Pilot

The European Medicines Agency Director (EMA) Executive Guido Rasi and high medical Hans-Georg Eichler sent a letter Thursday to clarify some assumptions put in doubt by a group of nine teachers That criticize aspects of track program pilot adaptation of the agency.

Background

First announcement in March 2014, the EMA pilot program seeks to accelerate patient access to medicines for patients with urgent medical needs and then of approval for a strong indication if waiting That clinical data and the additional real world That is used for assess if treatment is really as effective as expected. From November 2015, the EMA has selected 19 proposals to participate in the pilot program.

As Rasi there Eichler describe the of pilot adaptation "way seeks to maximize the positive effect of new drugs balancing timely access to That patients might benefit more from a new drug with the need for adequate information sober evolution benefits there risks . "

Three companies participating include Bluebird Bio, in 2015. That said May that it intends to request the conditional approval for gene therapy to treat beta-thalassemia LentiGlobin BB305 greater; September biotechnology company said in 2015 Immunocore also plans to seek approval conditional Addition lead IMCgp100 biological treatment of patients with metastatic uveal melanoma, a rare and fatal disease with few options available treatment; there August 2015, Pluristem Therapeutics, indicate if found using the guide EMA in the pilot to develop a cellular therapy product That heads a sub-group of patients with critical ischemia of the above tips of look you expand to other sub-groups of patients.

assumptions

In May, nine professors of Oxford, Cambridge, the Medical School London Hygiene and Tropical National French Academy of Medicine, Royal College of Physicians, Toronto, York University, as well as other Barcelona and Italy Copenhagen raised questions with some serious regarding the assumptions made by the development in the EMA pilot ways of adaptation.

"The evidence if based in these cases does not seem very convincing for us and it seems that there is a lot of uncertainty in their potential operational implementation" teachers wrote, noting a list of eight General assumptions, including:

• "New" or "innovative" drugs and biologicals STI more effective and safer That That current
• Current mechanisms of regulation postmarket suffocate market and the market entry and innovation retardation and "new" drugs "Innovator"
• Enter the market on time is beneficial for society
• To that patients have run you will return in a Town medication if the new one just failing regulatory obstacles or later you have marketing and doctors That sober act such subsequent warnings based sober marketing or use restrictions damages
• EU information systems can support the adaptation process with impartial way (or minimally slanted) a day as observation data information
• His acceptable alternative outcomes
• The notion of That "something is better than nothing" is an acceptable principle

And although teachers think That said in the notion of the way of adaptation has "great potential to benefit society," That also claim scientific terms "should be used and correctly, there's possibility That is a misinterpretation. The term "real-world evidence" are a euphemism for observation tests, is That comes from observations That experimentation has always precede production of empirical evidence there. "

They also say that before the use of a means of adaptation leads to the authorization of a new drug, "any subsequent plane to generate evidence must be agreed there legally binding on all parties Following Agreed protocol. This is should you need to ensure of the surrender of accounts by substantial sums of public money they have been and will That invested invested ".
EMA response

In response to the letter, there Eichler Rasi offer point by point summary if agree with the teachers there try of provide more support for adaptation That way form of works and moving forward will work in.

In the event of the "new" or "innovative" drugs could give the impression of That these more effective or safer That existing STI treatments, EMA says that the terms (new and innovative) That said His synonyms his "neutral" respect of a product if innovative or new more or less is given effective or safe That existing treatments.

"The aim of the ways of adaptación're lead to potentially beneficial treatments for the group of patients right as soon as appropriate" officials wrote. "Therefore, evaluation probable of sober benefit the existing treatment options heading of" novelty "must precede has decision to follow paths of adaptation. Note the EMA received 60 requests from sponsors its pilot program pathways of adaptation course, but we selected only 20. Our goal is to '' choose the winners, in the interest of patients No. unmet medical needs. "

And although officials clarified That "real-world evidence" art used by EMA "to refer you evidence from records, electronic health records (EHR) and insurance data, whether in studies specific observation or monitoring continuous use, the risks benefits there, "they also say That" his conscious That observational studies have produced results not reproducible or contradictory, however, also have done other methodologies, including RCTs [randomized clinical trials therefore the concept. pathways of adaptation makes Insisting in the necessity of harvesting planned the observation data, where it may be necessary to supplement trials testing. This collection is based on the advice of experts there Methodologic input of multiple stakeholders. on the other hand, repeated cycles of generation is quickly testing to refine or emphasize decisions when necessary right past. "

And as to be able to change a patient's drug again when the new one Town could become less effective, EMA recognizes the difficulties such a scenario.

"We are conscious disappointing experiences in the past, in fact, doctors have not always acted on post warnings to damage the sober marketing and use restrictions" Rasi there Eichler write. "However, recent experience with flat designed Prospectiva risk sample That is if properly managed, restrictions / warnings can be successful."

In the event of That "something is better than nothing" officials also make clear That "would be unethical and incompatible with the regulatory role of" selling hope rather than aid "[Bianco and Sipp Nature 2014. 510, 336- 337]. This is why special way of adaptation makes Insisting in early enough relevant evidence of patient benefit there a strict control of patient benefit after launch ".

RAC US Exam Question No 40

Question No 40:

The Food and Drug Administration Modernization Act (FDAMA) established two types of formal early collaboration meetings. Which of the following is one of those meetings?

A. PDP meeting
B. Agreement Meeting
C. Pre-IDE meeting
D. Type A meeting

Answer: B

FDA Kicks Pharma, Device Rule Finalizations Down the Road, Past Obama’s Presidency

The US federal government is notoriously slow to establish new rules and apply them. However, the Food and Drug Administration (FDA) is taking this slow to a new level for a variety of potentially controversial proposed and final rules, some of which have persisted for decades.

On Friday, the Department of Health and Human Services (HHS), which operates under the FDA released its semi-annual inventory of actions for the development of regulations under development, which lists a fraction of FDA standards or work propose or End and many of which have been delayed without explanation.

Security Subsequent Marketing

One of the first examples of such a prolonged delay a final rule is facing new requirements for reporting on the post-market drug products for human use and biological security. First proposed in 2003, the FDA now says HHS will need until 2017 last March under President Barack Obama in office, to take the final action of the rule, which further harmonize definitions and reporting formats with used by the International Conference on harmonization.

The final rule also add or revise the existing reporting requirements for drugs and biologics, to review certain deadlines for reporting and suggest further changes to improve the quality of safety reports received by the FDA.

Another rule, which has been persistent since the regulation of proposed rulemaking (NPRM) comment period ended in 2009 (and, last fall, the FDA said it would be completed two months ago in April) clarify how to describe the information requirements post-marketing combination products (combinations of a drug, device and / or biological) security. The completion of this rule is not even on the list of HHS 'on Friday.

Furthermore, it is not included in the list of HHS 'is the decision of the FDA to delay the completion of its rules on the labeling of generic drugs, which also deals with the post-marketing and widely covered approach to security. It is also expected that this rule to come post-Obama in April 2017 (after it was proposed in 2013, open to comments in 2015 and should be published in February). The house was still trying to force the FDA to rewrite the rule.

Test Devices

On the device side, the FDA proposed a rule proposed in February 2013 to ensure clinical investigations conducted outside the United States in support of an application for premarket approval, the request for exemption from humanitarian system, an application for exemption investigational device or submission notification for marketing will be carried out in accordance with good clinical practice.

According to the FDA, in the fall of 2014 that the rule that the device could be completed in January 2015, but now lists HHS (in the agenda published on 9 June) that it intends to terminate the rule sometimes the last month.

Another Delay

Meanwhile, another rule FDA first proposed in 2006, reorganize, consolidate, clarify and amend the rules governing what companies must register establishments and list of medicinal products for human use, there including some biological drugs and animal drugs , it has been postponed until next month times.

It also goes on sale in July 2016: the change in FDA regulations for good current manufacturing practices to clarify and strengthen requirements for the label, color, dedication and design of medical gas containers and closures (first proposed in 2006).

"Despite regulatory requirements and industry standards of medical gas, there have been repeated incidents in which the cryogenic vessels of harmful industrial gases have been linked to the systems of medical oxygen supply in homes hospitals nursing and then administered patients. These incidents resulted in death and serious injury, "says the FDA." there have also been several incidents with medical gas bottles high pressure that led to death and injury to patients.

These changes, as well as current regulations are intended to ensure that the types of incidents that occurred in the past are produced, as well as other types of medical gases and potentially fatal accidents not predictable in the future. "
The FDA also expected next December to propose regulations to define and implement certain legal conditions under which aggravate products are eligible for exemption from certain requirements. FDA had planned to make the proposal on 31 March.

Also Unregistered

Nor on the agenda of HHS is the rule proposed by the FDA to allow inclusion in labeling of the symbols of autonomous devices established via a standard developed by an organization of national or international standards, provided that these symbols are explained in the unit label. The rule also revises the rules on labeling of the unit in order to allow the use of the statement symbol "prescription only" on the label.

The rule was proposed in 2013 and expected to be finalized (last fall) 31 Mar 2016.

The complete list of rules and possible dates, many of whom rejected without any transparency or explanation, as Senate Republicans questioned how the FDA uses the project and the final orientation regular, rather than through d regulatory Action

RAC US Exam Question No 39

Question No 39:

In the development of a revised manufacturing procedure, which of the following is a critical step in ensuring that the product manufactured would not be adversely affected by this change?

A. Procedure qualification
B. Product verification and/or process validation
C. Quality control
D. Conformance inspection

Answer: B

Advanced Therapies: Stakeholders Call for EMA Changes on Regulations, Manufacturing

The European Medicines Agency (EMEA) on Friday issued a report with concerns and regulatory: modifications described by developers, manufacturers and investors in therapy products Medicines (Drugs Advanced Therapy), including gene therapies, products tissue engineering therapies and somatic cell, a UN meeting last week.

Note interpenetration What recurring themes include the need for early interaction with regulators and policy, a larger Executives Transparency larger Managers harmonization between Member States of the EU regulation ATMP, What and a proposal for Industry Being EMA calling more flexible converter Advanced Drug Reservations therapy manufacturing.

"While no decision has been bis prize, European Commission, national authorities and the EMEA and have a conversation Start From The feasibility of these proposals," said EMA.

Context

In the EU regulatory framework Verter Advanced Drug therapy was established by Regulation (EC) No 1394/2007, which has been in force since 2008.

Allows automatic regulation of the UN access to a central line for authorization of advanced therapy products in the EU and created the Committee for Advanced Therapies, which experience gathered and established, among other processes, classification procedures are Certification converter rescuer The early development of advanced therapy.

An EMA convened a meeting covering advanced therapy 27 May, 2016, attended ONT University and researchers, incubators and NGOs Consortium, and NGO representatives Patients and health care, small and large pharmaceutical companies, investors, Technology assessment Bodies health, national competent authorities (ANC) and the European Commission

Manufacturing Proposal

"A mayor proposal at the meeting was to licensing requirements reflect Vierten Special Unique Drugs for manufacturing Advanced Therapy," Specifies rapport. "For example, participants take into account the manufacturing ATMP can take place in different sitios and to pour certain products, further ethical Production may need to take place near the head Involvement What is all the sites concerned. - it including hospitals - would be needed to maintain the manufacturing license. "

However, with the lack of harmonization of the United Nations, only the level of the National Motor Vehicle some Member States allow hospitals hold a manufacturing license, interested parties "a request to the authorities to more flexible requirements pendant principles the years phases of maturation development. specific proposals for adaptation needs under gone up tone based products cells a included (no products handled significantly) could be If you take into account as falling on the border between the grafts and Drug Advanced therapy activate below.

"Similarly, more pragmatic approach should be used converter meet requirements Process Validation converter Many advanced therapy, given the difficulty to produce the required amount of batch conversion to CHARACTERISTICS pharmaceuticals," Specifies rapport.

Other proposals Promotion manufacturing technologies include (for example for manufacturing header and closed systems) and manufacturing models (for example, manufacturing decentralization), which would require more flexibility.

"Regulatory bodies could also stimulate the development of production sites, such as service, the use of small and medium enterprises (SMEs) and other funds, so that capacity is available converter multipart develop different advanced therapy medicinal" said the relationship. "These suggestions were considered timely, given the European Commission What is revising the Directive on GMP requirements Pour advanced therapy and will soon launch the public consultation."

GMO

Another important issue in the meeting focused on a genetically modified (GMO), which had a cool reception in the EU.

Interested parties have indicated that GMO directive (directive 2001/18 / EC) "are not designed specifically for your drugs and aggravated lacunar infarcts compared AEC is by discrepancies in the application in the Member States. From May Different requirements among States members, the integration of assessment of GMOs in the authorization of clinical trials of the United Nations is a challenge, especially in the context of multicenter clinical trials. "

Developers of these gene therapies have called for greater uniformity is suggested as a step premiere, the establishment of a central repository in Spanish, a list of requirements and deadlines for the assessment of GMOs in all Member States.

"Some speakers also called for: modifications directive has the OMG itself," says A good relationship. "Another area that could benefit from further harmonization concern cells and tissues as materials manufacturing EMPLEA premieres Drug Therapy Advanced with contradictory interested Applicant rational application over tissues and cells (Directive 2004/23 / EC) and other relevant laws" .

Other issues

Weighing also called for greater harmonization A "facilitate the movement of materials between the tiles with different requirements (inside and outside the EU) starting and reduce the burden of having a re-tester cells and tissue Before starting the manufacture of drugs . Advanced therapy the test puts itself can then focus on: aspects of the nature of the Advanced therapy Medicinal Products ".

The industry also asked for excipients A SPECIFIC guidelines and a system master file fluent Marketing authorization with respect to excipients and content premieres.

In addition, stakeholders have proposed new tools for Development (organelle eg transferor of extrapolation, modeling / simulation, biomarkers, etc.) to meet the needs of clinical no.

"On the question f risk-benefit ratio product development, stakeholders noted that the current practice is mainly focused on the risks and the call of the additional UN accent is put on the expected advantages, but realistic, particularly when patients have incurable diseases or treatments when appropriate default font "added the relationship.

"If advanced therapy should fulfill its promise to provide innovative treatments for patients, regulators should feed regulatory environment that the UN Promoting Innovation protect public health and, ultimately, the bill easily accessible when patients to appropriate new treatments" says interpenetration.

RAC US Exam Question No 38

Question No 38:

Which of the following federal laws includes information about ANDA submissions?

A. Antibiotic Amendments of 1945
B. Durham-Humphrey Amendment of 1951
C. Drug Amendments of 1962
D. Drug Price Competition and Patent Term Restoration Act

Answer: D