Regulatory Recon: Kite CAR-T Therapy Nabs FDA Priority Review;

Regulatory Recon: Kite CAR-T Therapy Nabs FDA Priority Review; UnitedHealth, Merck Sign Pact to Explore Value-Based Payments (26 May 2017)

 Focus: United States

• UnitedHealth and sign agreement to explore Merck's Linking Drug Performance Payments (CNBC)
• Impulse Industry: Excess Aging Background Lines Imposed (BioPharmaDive)
• Kite Receives Priority Review of FDA for Treatment of RCA-T for Aggressive Non-Hodgkin's Lymphoma (Press)
• The mortality rate of Alzheimer's disease in the United States has increased dramatically in 15 years. Why? (Washington Post) (Reuters)
• Gottlieb: FDA hiring froze, plans to increase competition from generics come (Focus)
• Generics Copaxone: Who will win the first exclusive affiliates? (Warning)
• FDA Panel supports Epogen biosimilar Hospira 14.01 Vote (Focus)
• The reporter lodged a complaint against the FDA's approval for Sarepta (Boston Biz Journal)
• One quarter of American babies with congenital malformations related to Zika was born in New York (NYTimes)
• The proposed budget would reduce Trump's $ 2.2 billion in global health spending (NPR)
• The J & J USA game is not likely to be above the OTC (pink information-$) (Fierce)
• HHS action on drug prices: here is the price of the secretary who is listening (Rose- $)
• Biosimilar advisory committees remain smoother, even with regard to Lo Mismo (leaf Rosa- $) remain
• As the FDA seeks to speed up the review of two other political experts want to limit drug prices accelerated win an OK (Endpoints)
• Drug addiction experts questioned the suggestion to limit FDA opioid prescriptions (Modern Healthcare)
• SURVEY: Medical device manufacturers to be pirated wait, but few do anything (MassDevice)
• Big pharmaceutical and social media: how to avoid problems with the FDA (Forbes)
• Two years later, what has Apple achieved ResearchKit? (STAT)

Approach: International

• Update of the seventh session of the World Health Assembly on 25 May (WHO)
• uperbugs in the supply chain (Pharmafile)
• The German pharmaceutical market has established stable growth (PMLive)
• SMEs Leverage the EU's centralized procedure and scientific support EMA (Pink information- $)
• The plan that generic drugs Modi worries health experts, the Indian pharmaceutical sector (Reuters)
• The former Americans. Of AbbVie. Humira's sales are on the edge of the precipice. Analysts (Fierce), but no panic, say
• movement of the multinational pharmaceutical industry to stop generic registration before the patent in Russia (PharmaLetter- $) occurs
• Tip-Toes FDA in China to push data integrity, harmonization (Rosa sheet $)
• Three negative opinions of the CHMP - but who received an advantage? (SCRIP- $)
• India Cipla low investment in biopharmaceuticals, sharpens focus on breathing (Reuters) (Endpoints)
• Why biosimilars are changing the pharmaceutical industry (EPR)
• The FDA and EU supply chains have doubled inspection of Asian pharmaceutical products (pink information- $)
• Clinical studies conduct: China steps to facilitate the entry of new drugs (color Pink Leaf- $)

Pharmaceuticals and biotechnology

• Harpoon Therapeutics reaches $ 45 million in series B for work I-O (Fierce)
• the Celgene oncology head on ASCO, Revlimid, and the increase in the value of triplet combinations (PharmaLetter- $)
• Safety Profile ozanimod help to bring to the market multiple sclerosis, says Celgene (Fierce)
• FDA Drug Safety Podcast: The FDA does not identify any adverse effects to date with brain retention of gadolinium-based contrast agents for magnetic resonance imaging; Continuation Review (FDA)
• The market for ovarian cancer drugs (Nature)
• Shire quietly stops new investments leaving Baxalta Ventures (Boston Biz Journal)
• Rendezvous: FIIM, Merck, Chromocell, Kymab, GW Pharmaceuticals, Fix Fortuna and Pattern (SCRIP- $)
• AstraZeneca has launched a nationwide voluntary recall of a batch of BRILINTA 90 mg professional sample bottles due to report another drug in a bottle of this batch (press)
• Patient Partnership Group Rare Disease Options Cell Business Model / Gene Therapy (SCRIP- $)
• Constellation Pharmaceuticals Hires Jigar Raythatha as CEO (PharmaLetter- $)
• Does the FDA change its interpretation? (FDAZilla)

Pharmaceuticals and biotechnology: study results, deposits and denominations

• Chronology delayed by Raxone Santhera in DMD (BioCentury)
• Abeona Therapeutics receives orphan designation from the FDA for gene therapy (PRT)
• Anika announces the first patient enrolled in an additional phase III trial of CINGAL for the treatment of knee pain associated with osteoarthritis (press)

Medicalequipments

• New roles for medical devices in the fight against antibiotic-resistant superbugs (MDDI)
• Blood-based breast cancer screening can help prevent biopsy (Forbes)
• Most counterfeit contact lenses are contaminated, according to the FDA (Industry Securing)
• Ethicon J & J states the economic analysis of data in Dermabond Prinéo (MassDevice)

United States: Assortment and Government

• At Trump's initiative, states are trying to include Medicaid in conservative directions (Washington Post)
• Extended causal fault condemns another failure with claim (drugs and devices Act)
• n the race to develop telemedicine services, be aware of legal problems (MDDI)
• Mylan Institutional LLC v. Aurobindo Pharma Ltd. (Fed. Cir. 2017) (Patent Docs)
• The Consumer Advocates Coalition Challenges Notifications FDA's Final Rule GRAS (FDA Blog Law)
• The "quick search" of health care: foundations help consumers maintain a balance and a mirror for the health system (Health Affairs blog)
• Patients trio continues Pfizer, Bristol-Myers, claiming they suffer Eliquis soft pedal strap (fierce)
• Biogen Asking the Judge to File Negative Antitrust Drug Lawsuit ($ Law360-)
• Pfizer said the expert testimony in the game that Celebrex unreliable ($ Law360-)

Upcoming dates and events

• Schedule of the FDA Advisory Committee
• Subcommittee on Advisory Committee on Pediatric Oncology on drugs; opinion
• Meeting - 21-22 June 2017
• Advisory Committee on Vaccines and Related Biological Products; Notice - July 28, 2017

Europe

• Hepatera: PRIME eligibility scheme for Myrcludex B by the European Medicines Agency (Press)
• Patient safety in the NHS: culture change agents (Harvard patent of health)

Asia

• Chinese group buys James Bond brand condominiums (Bloomberg) ($ Law360-)

India

• Diagnosis agappe dressed Hitachi Chemical Company (Economic Times)
• Government identifies ADR 9 additional centers in district hospitals to expand PVP in the country (Pharmabiz)

Australia

• Reference: Examine 75 standard cells derived from TGO cord blood progenitor hematopoietic (TGA)
• tablets 10 mg BTC rosuvastatin - Reminder (TGA)
•  BTC Paracetamol Tablets - Reminder (TGA)
•  Valium (diazepam) tablets 5 mg tablets blister of 50 tablets - Reminder (TGA)
•  Apotex pantoprazole tablets 40 mg - Reminder (TGA)

Another international

• Brazilian doctors use fish skin to treat burn victims (Reuters)
• General health and other interesting items

Symptoms of celiac disease are more likely to be resolved in children (Reuters)
Michael Bliss, a historian who misspelled the myths of insulin discovery, died at 76 (New York Times)

WHO Officials Offer Opposition to Value-Based Drug Pricing

Officials from the World Health Organization (WHO) told reporters at an information session on Thursday that the agency has serious reservations at prices based on pharmaceutical prices.

"What is the value of life? This structure is good for luxury items, because you have a choice ... if I am sick of cancer, what is the choice? We believe value-based pricing is impossible for products that are indispensable, "said Marie-Paule Kieny, Deputy General Manager of Health Systems and Innovation, which he said.

Value-based fixing systems, such as the UK's National Institute of Health Care, decide what is price-paying and price-adjusted. In general, NICE estimates that interventions costing the National Health Service in the UK less than £ 20,000 ($ 26,000) per year of QALY are cost-effective.

Kieny spoke after a meeting of about 200 stakeholders from countries around the world in the first Just One Day Forum in Amsterdam this week sponsored by WHO and the Ministry of Health, Welfare and Sport of the Countries -Bas.

He called on countries to increase transparency in drug prices because "countries sometimes think they have the best deal and if too many countries believe, there is no movement or exchange of information ".

And although Kieny acknowledged that "innovation is not free," he also said the discussions should focus on "the real cost of R & D [research and development]" and the cost of failure, As well as set up price systems that a payer is willing to pay.


A new study published this week on the cost of anticancer drugs found worldwide that even though prices are the highest in the US, are the least affordable in India "by a large margin. "Despite falling prices, anti-cancer drugs are less affordable in middle-income countries than in high-income countries.

. Dr. Suzanne Hill, Director of Essential Medicines and Health Products WHO Kieny reiterated points about value systems, adding, "Who decides what value? He also said that the forum "is our first attempt to launch a program of action to understand the factors that determine the current structure of prices are."

He insisted on the forum of interest for collaboration between governments in "scrutinizing the horizon" or evaluating products in the industry for what is important and what is worth paying in the future , And the need for WHO to develop a list of vulnerable essential drugs. Like those of scarcity all over the world.

UK Pharmacovigilance Post-Brexit: Lots of Questions, Few Answers

No one knows what is going to look like British pharmacovigilance after the UK left the EU and uncertainty puts many residents qualified person responsible for pharmacovigilance (QPPV) in the UK in a precarious situation.

According to the independent academic research unit on drug safety in the UK, which met last week on the subject of pharmacovigilance post-Brexit "At worst, it will cause a serious blow to British pharmacovigilance" as the Pharmaceutical companies "will not be required to carry pharmacovigilance in the UK and can not be expected to pharmacovigilance providers from the UK to carry out these studies."

Currently, the European Medicines Agency (EMA) runs the Pharmacovigilance Risk Assessment Committee (PRAC), which uses two members from each Member State and has been chaired since its inception in 2012 by the Agency June Raine regulation of medical products And UK Health (MHRA).

But after Brexit, the UK will not show PRAC, although representatives may present as non-voting observers, as Norway and Iceland do now.

"Pharmacovigilance is above policy," said Saad Shakir, director of the Research Unit on drug safety, last week at the "UK Pharmacovigilance Forum post-Brexit" according to the audio files sent By email to focus.

He also noted the large number of QPPV leaders based in the UK, adding: "We want them to remain part of the EU apparatus. These are national assets." But now, he said, they were "afraid of what is going to happen as the regulations indicate that QPPV should reside in the EU."

Shakir said he could have negotiated a mutual recognition system or a means for EMA and MHRA to continue collaboration.

Ian Hudson, general manager of the MHRA since 2013, said recently, according to Shakir, there had been some discussion about outsourcing regulation work in the UK, but "We do not know what this will look like en -traitance."

Virginia Acha of the British Pharmaceutical Industry Association also noted that pharmacovigilance times at the short end of the rod, as it works so well now.

"There is an enthusiasm to maintain most of the status quo, but the extent to which it is possible to publish Brexit is unclear," the Drug Investigation Unit said in a statement. "Once the UK leaves the EU, it is essential that the current UK-Europe cooperation in pharmacovigilance has improved a lot. Mutual recognition of market research and MHRA EMA is required as part of the Brexit negotiations. "

In addition to this meeting focused on UK pharmacovigilance, last weekend, the European Council established guidelines for the Brexit negotiations between the UK and the European Union.

FDA Offers Draft Guidance on Generic Versions of Opioid Overdose Nasal Spray

While the opiate epidemic continues to spread, the US Food and Drug Administration (FDA) seeks to alleviate some of this pain by providing a new draft guidance to generic drug companies looking to develop generic versions of nasal spray that can Reverse opioid overdose.



The draft guide is intended to help companies understand what studies need to be done to get approval for generic nasal spray Narcan Adaptar Pharma, also known as nasal spray of naloxone hydrochloride.

The narcan nasal spray was originally approved in 2015, and the FDA notes that in November 2016, ADAPT has filed a citizen petition (a tool sometimes used to delay the entry of generic drugs) by asking the FDA to refrain from approving any referral (ANDA) narcan nasal spray, unless certain conditions are met, including conditions related to the demonstration of bioequivalence.

The petition comes as the cost of nasal spray and other forms of naloxone continues to rise.

However, the FDA said that the draft directive is, outside this citizen petition, will respond separately. And the project must begin the process of developing and marketing less expensive versions of naloxone nasal spray.

Project Details

The three-page proposal offers two options: o in vivo in in vitro studies, which reveals the specificities and differences in each of them.

For advice on how to develop a generic drug combination product for approval, the FDA stated its policy titled "Comparative Analysis and Comparative Studies Related to Human Factors for Combined Drug Revenue Device presented an ANDA (January 2017) "Which provides the current thinking of the agency in identifying and evaluating differences in user interface design for a generic drug combination product available from its RLD [drug listed in Reference]."

The FDA also clarifies that at the beginning of product development and / or prior to an ANDA offer, applicants must submit to the Office of Generic Drugs, controlled by correspondence and / or request for a pre-ANDA meeting, "the results of Comparative analysis (eg comparative labeling analysis, comparative analysis of tasks, physical comparison of the constituent part of the distribution device) including an overall assessment of all differences identified between the user interface by T A report to the Product as described in the guidelines referred to above.

EMA Board Adopts New Policy on Whistleblowing

In order to encourage complainants and others to feel secure with possible details of the irregularities, the European Medicines Agency's (EMA) Board of Directors adopted a new policy on Monday detailing how the agency handles allegations , Including those that could have an impact on drug evaluation and monitoring.

Since 2013, EMA says it received a total of 43 of these reports detailing various issues and allegations of drug manufacture in conducting clinical trials.

"While there is no formal policy so far, all reports have been treated in accordance with the principles of the new policy," said EMA, noting that this new policy will help EMA to evaluate all reports and coordinate any further investigation In a structured manner, while protecting the journalist's confidentiality.

politics

Any external entity aware of facts that may have an unequal impact on the authorization, supervision and maintenance of human and veterinary medicines may, in the public interest, be reported to EMA at the following address: @ ema.europa.eu reports .

The BMA says that it will ensure the confidentiality of information from outside sources and the identity of the sources and the protection of personal data, these data lead to a later review by the EMA or the Anti-Fraud Office (OLAF) or do not.

"Examples of claims that can be made to understand that the integrity of the data in the studies used to support the marketing authorization can not be invoked.That integrity is essential to confidence in authorized drugs.These problems can occur due to To the formation of poverty, inadequate application of procedures and controls or even falsification of data, "says the policy.

"If there is a fear that irregularities may affect the balance of drug benefits and risks, EMA's scientific committees may consider regulatory action," the agency said in a statement. If the allegations relate to a drug EMA throughout the authorized country, it may, on a case-by-case basis, refer the matter to the national drug agency of that Member State.

The policy, which came into force on 17 March 2017 and prepared in consultation with the European Commission and OLAF, will be reviewed in three years.

FDA Unveils Regulatory Science Progress Report

The report, covering the years 2015 and 2016, points to significant progress in the US Food and Drug Administration (FDA) in refining non-clinical and clinical tools to better evaluate medical products, advancing manufacturing and quality, and Improvement of the internal computer tools to support the applications of regulation of scientific examination.

In the clinic

As for non-clinical predictors, the report indicates that FDA researchers have developed a variety of software tools that are now compatible with the non-clinical evaluation of medical products.

"These tools include sophisticated models to predict the carcinogenic effects of some of the ingredients of drugs based on their structural attributes, mathematical representations of the human body (IT ghosts) that can be used to predict the effects of medical devices, such as Radiation exposure, and mechanically-informed pharmacokinetic models to help predict drug exposure in populations where it is difficult to obtain clinical data, "according to the report.

The FDA has also developed in vitro methods and tools to better understand the properties of drugs and biologicals, including vaccines and new tools to support the assessment and development of medical imaging technologies.

Genetic approaches and transplantation have also been used to create animal models that aim to predict the human response more deeply to medical products, and new physical methods and procedures have been developed to support generic bioequivalence assessment of generic drugs.

Clinical evaluation

To better support clinical evaluation of medical products, the FDA engages in the development and evaluation of biomarkers to better predict long-term treatment outcomes or to affect certain patients more likely to be successful treatments.

For example, the FDA notes that its statisticians helped design antibiotic trials that could assess factors related to the development of resistant microorganisms.

In addition, the agency has formed new biomarkers to guide treatment decisions and predict the progression of the disease.

"A long-term research effort to improve cardiovascular risk prediction contributed to the recommendation of the International Conference on Harmonization that the cost-intensive and resource-intensive QT study (required to assess the majority of candidates To drugs) could be replaced by an electrocardiogram - measures based on early-stage clinical studies, "the report said.

He also referred to the FDA's efforts regarding the use of real-world evidence, staff members have expressed concerns in the past.

Through initiatives such as the patient-centered drug development initiative, the Advisory Committee on Patient Commitment to Healthcare Products and the Patient Preferential Initiative, the FDA also incorporates patient views into the Development and evaluation of medical products.

Manufacturing and Quality

The FDA's medical product centers also address scientific issues related to new critical technologies for the manufacture of products to improve the reliability of drug delivery, complex product characterization, quality standards, Approval of product quality and understanding of interactions of regulated products with products of biological systems.

"We have developed the in-house laboratory and computing capabilities to study continuous production with an advanced process control system," the report added. "We have worked with the Biomedical Research and Development Authority (BARDA) to take advantage of continuous manufacturing to minimize vulnerability to domestic, biological and radiological chemistry. We have set in motion the creation of a 3-D printing to understand the factors Which contribute to quality and quality, the performance of implantable medical devices, drugs and combined products made with this new technology.

In addition, the FDA has developed automated approaches to predict critical properties of human stem cell preparations, such as their ability to contribute to bone growth.

Illinois

The agency has launched its "JumpStart" service that allows evaluators to organize, manage and verify the quality of clinical data in ap

European Regulatory Roundup: EU Trade Groups Propose

European Regulatory Roundup: EU Trade Groups Propose Criteria for Deciding EMA’s New Home (2 March 2017)

European business groups propose criteria for deciding the new EMA home

European biopharmaceutical groups have jointly defined the criteria for deciding the new home of the European Medicines Agency (EMA). The three trade groups have detailed their desire that the selection process takes into account six key criteria in a letter to a senior European health official.

The criteria proposed by the European Federation of Pharmaceutical Industries and Associations (EFPIA), EuropaBio and Medicines for Europe aim to minimize the risk of disruption when EMA moved to its present headquarters in London, UK, to join Brexit. EFPIA and its co-signatories are publicly out of the discussions about the country that could host EMA post-Brexit, but according to their criteria they describe the type of place they want to win.

Three of the criteria relate to local regulatory capacity. EMA, with the support of the Medicines and Health Products Regulatory Agency (MHRA), business groups want to ensure that the national regulatory agency of the new host country can strengthen their participation in EU activities. This change should not detract from non-EMA workforce, business groups argue, and as such, the local government must be prepared to increase spending.

The three remaining criteria relate to the more general characteristics of the proposed sites. Commercial groups want the Commission to choose a location with easy access to the airport, communicated with the rest of the EU, adequate local hotel capacity and enough space for EMA meetings and events. Finally, the authors of the letter to the Commission to minimize disruption of personal EMA taking into account the availability of international schools, access to labor markets and social security.

The EFPIA criteria echo the cases submitted by some candidates to host the EMA to date. Denmark, the Netherlands and other countries have talked about their regulatory and scientific capacity and the viability of their cities for people traveling to London. If the EMA fails to convince the vast majority of its staff to move to London with him, the regulator could have trouble providing normal services to its small size. Business groups want to avoid that scenario.

As such, while the letter out of the debate on the city that will host the EMA criteria favor the countries of Western Europe that have the necessary regulatory capacity and facilitate resettlement.

The organizations argued their views in a letter to Xavier Prats Monné, Director General of Health and Food Safety Directorate-General. The responsibility to choose a new site for the responsibility of EMA Monne and the rest of the commission. Officials have yet to come up with a timetable to make the decision, but many people affected by resettlement want clarity this year.

Letter

EMA sharing guidelines on the risks to human health of veterinary medicinal products in groundwater

EMA has issued a directive on the toxicological risk that veterinary medicinal products in groundwater pose to human health. The text proposes a methodology to assess whether the concentration of drug in groundwater poses a risk to the health of humans who drink the contaminated liquid.

Today, regulators consider concentrations above 0.1 mg / l as unacceptable. At lower concentrations, no risk is expected and no regulatory action is required as such. However, EMA now consider whether this is the right approach.

The cut-off point of 0.1 g / l is obtained from the limit of detection of the analytical methods at the moment. The development of more sensitive tests can detect small amounts of contaminants has made possible a new approach. Because some drugs may affect concentrations below 0.1 mg / l, EMA believes that using these new, more sensitive tests is useful in making more informed judgments about the risks of groundwater contamination.

The EMA provides the industry uses two formulas that take into account the acceptable daily intake of an active ingredient, the daily consumption of drinking water, the expected concentration in the groundwater and other variables to determine if the risk to human health is acceptable . If the expected concentration in surface waters exceeds