Category Archives: UK Pharma

More than 300,000 new cases of cancer were diagnosed in the UK in 2013, and roughly half of all people in the UK born after 1960 will be diagnosed with cancer in their lifetime.

Cancer endurance and survival in England is lesser than the European average, which is moderately because patients in the country are being diagnosed at a later stage. However, there are signs of progress

According to data from the National Cancer Intelligence Network, in 2006 almost 25 percent of cancers, one in four, were diagnosed as an emergency, but in 2013 this figure had fallen to 20 percent, or one in five.

The National Institute for Health and Care Excellence has published a new quality standard method just to accelerate cancer diagnosis and save thousands of lives every year.

It is estimated that in England only, 10,000 lives could be saved every year through earlier finding of the disease and the use of more suitable surgery as prime treatment.

The procedure proposes that GPs have straight access to tests to speed up the process such as:

• Endoscopy
• Ultrasound
• MRI
• X-ray
• CT for people with suspected cancer

The solution to secure early diagnosis and reducing waste of resources is improving attendance rates for cancer service referrals, and so the Institute suggests giving patients written information to support them to attend.

The quality standard, which follows updated direction on the detection and recommendation of suspected cancer published last year, sets out clear tables linking signs and symptoms to possible cancers and comprising simple suggestions about which tests to perform and the type of referral to specialist services that should be made.

According to Professor Gillian Leng, deputy chief executive of NICE, “When we published our updated suspected cancer guidance last year we said the best way to successfully treat cancer was to make an early diagnosis,”

Date: 4^th July 2016

http://www.pharmatimes.com/news/nice_quality_standard_to_boost_earlier_cancer_diagnosis_1057206

The European Medicines Agency (EMA) and the European Society for Medical Oncology (ESMO) are co-organizing a workshop on the role of single-arm trials in the approval of new cancer medicines on 30 June 2016.

There is no specific regulatory guidance on cancer medicines development based on such trials and how to manage the remaining uncertainties optimally. In a single-arm trial every patient registered receives the new medicine. These trials have no detached group in which patients are given a placebo or another medicine to permit evaluation.

The workshop will give a platform to regulators, clinicians, academics, patients, medicine developers and health technology assessment specialists to discuss:

• Situations in which the marketing approval application for a cancer medicine could be based on results from a single-arm trial.
• Experience achieved with marketing authorizations based on single-arm trials data, the potency and shortcomings of different perspectives, and chances from data sharing proposals.
• Circumstances in which a medicine shows considerable anti-cancer activity in an area where patients have no treatment option or where the demeanor of standard trials with a relative arm is difficult, such as in rare cancers or selected populations for example, will be measured.
• Look at the views of diverse stakeholders groups and discuss the need for regulatory supervision with the eventual plan of optimizing the progress of new cancer treatments in these situations.

Dated:  27/06/2016

http://www.ema.europa.eu/ema/index.jsp

The European Medicines Agency (EMA) and the United States Food and Drug Administration (FDA) have created a new collaboration on patient engagement. It will give an opportunity to share experiences and best practices on the way the two agencies involve patients in progress, assessment and post-authorization actions related to medicines. The first meeting took place by teleconference on 22 June 2016.

According to Guido Rasi, EMA Executive Director, “Our aim as regulators is to make sure that patients have access to safe and effective medicines that improve their lives. It is important to engage to be able to fully understand their needs and priorities and make their voice heard along a medicine’s lifecycle,”

The collaboration established by EMA and regulators outside the European Union focus on definite topic areas where the parties involved could gain an immense exchange of information. The presently offered EMA/FDA collaboration discusses problems correlated to:

• Biosimilars
• Medicines to treat Cancer
• Orphan medicines
• Medicines for children
• Pharmacovigilance

The increased interaction will allocate EMA and FDA to exchange information to extent the future rendezvous with patients. The information exchange is covered by the confidentiality arrangements between the two regulators.

The main discussion topics will comprise:

• The processes for selecting and preparing patients to take part in the agencies’ activities
• How to ensure that patients are independent and representative
• How to report on the impact of patient involvement

It is expected that three to four meetings will be arranged per year via teleconference and will be chaired in cooperation by FDA and EMA.

Dated: 22/06/2016

http://www.ema.europa.eu/ema/index.jsp?curl=pages/news_and_events/news/2016/06/news_detail_002554.jsp&mid=WC0b01ac058004d5c1

In the meeting of the Pharmacovigilance Risk Assessment from 6-9 June 2016,  the Committee paying attention on the extensive range of its tasks and responsibilities which cover all characteristics and features of the risk management of the use of medicines.

The PRAC’s full range of work comprises

• Pre-authorization actions
• Consideration of risk-management plans for medicines under assessment
• Post-authorization activities
• Evaluation of periodic safety update reports(PSURs) to re-assess the benefit-risk balance of a medicine throughout its lifecycle on the basis of new data collected.

The PRAC also estimates safety signals, public-health functions that make sure that latest safety issues are swiftly detected, assess and, when suitable, product-labeling changes or limitations are introduced for the benefit of patients.

Date: 10/06/2016

http://www.ema.europa.eu/ema/index.jsp?curl=pages/news_and_events/news/2016/06/news_detail_002546.jsp&mid=WC0b01ac058004d5c1

The primary treatment for the rare bleeding disorder hereditary factor X deficiency has now been initiated across the UK.

The UK is the first country in Europe where Coagadex will be made available, via a Commercial Medicines Unit tender, which starts on July 1st.

Bio Products Laboratory’s Coagadex was accepted in Europe in March for the treatment and prophylaxis of bleeding as well as for perioperative management in patients with this state.

According to Dr Steve Austin, director of St George’s University Hospitals NHS Foundation Trust, London “The prior focus has been on treating Factor X deficiency with blood infusions of plasma. For the first time, patients with this exceptional bleeding disorder can receive a definite factor X replacement that has been proven useful and successful in clinical studies.”

Hereditary factor X deficiency has an impact around 700 patients in Europe, and is described by a lack of factor X protein in the blood, which plays a critical role in coagulation.

People with this insufficiency are at bigger risk of bleeding, including inside the brain, lungs or gastrointestinal tract in the most severe cases, which can be life-threatening.

The CMU works on behalf of the Department of Health and the NHS and in corporation with the people who buy pharmaceuticals for hospitals across the NHS in the UK.

Date:  7th June 2016

http://www.pharmatimes.com/news/uk_launch_for_first_hereditary_factor_x_deficiency_treatment_1035301

The European Medicines Agency (EMA) today published a report from a multi-stakeholder expert meeting held on 27 May 2016 to look at feasible ways to promote the advancement of ATMPs in Europe and increase patients’ access to these new treatments.

ATMPs include:

• Gene therapies
• Tissue engineered products
• Somatic cell therapies

 These medicines have potential to reshape the treatment of a wide range of conditions, mainly in disease areas where predictable approaches are insufficient. However, eight years since EU legislation on ATMPs entered into force in 2008, only five ATMPs are currently sanctioned.

According to EMA’s Executive Director Guido Rasi, ‘We have organized this meeting with all relevant stakeholders to discuss concrete proposals on how we can nurture a regulatory environment that encourages development of ATMPs, safeguards public health and, ultimately, facilitates timely access for patients to much needed treatments.’

The meeting brought together leading academics and researchers, representatives from patients’ and healthcare professionals’ organizations, small and large pharmaceutical companies, the investment community, incubators and consortium organizations, health technology assessment (HTA) bodies, national competent authorities and the European Commission. In their discussions they focused on four key areas:

• Assisting research and development areas.
• Optimizing regulatory processes for ATMPs.
• Moving from hospital release to marketing approval.
• Improving funding, investment and patient access.

The main ideas and solutions projected by the different stakeholders are sum up in the meeting report. EMA and its scientific committees, together with the European Commission and the national knowledgeable authorities, have started conversing the proposals made during the meeting. Tangible actions will be determined over the next few months and shared with stakeholders.

Date: 03/06/2016

http://www.ema.europa.eu/ema/index.jsp?curl=pages/news_and_events/news/2016/06/news_detail_002543.jsp&mid=WC0b01ac058004d5c1

The European Medicines Agency (EMA) has started a appraisal of the plan that describe first-in-human clinical trials and the data needed to allow their suitable design and allow instigation. This is being done in collaboration with the European Commission and the Member States of the European Union (EU).

The evaluation will recognize which areas may need to be revised in the light of the tragic incident which took place during a Phase I first-in-human clinical trial in Rennes, France, in January 2016. The trial led to the death of one participant and hospitalization of five others.

EMA’s assessment will take into relation the findings from two in detail investigations into what went wrong during this check, one carried out by the provisional Specialist Scientific Committee (TSSC) set up by the French medicines agency ANSM and the other by the assessment générale des affaires sociales (IGAS), the inspectorate for social affairs in France.

Both reports comprise a sequence of recommendations concerning the requirements for sanction and ways of first-in-human clinical trials for further examination by the international regulatory and public health community.

EMA’s work will be the hub on best practices and direction. The objective is to agree a concept paper by July identifying areas for change and proposals to further reduce the risk of similar accidents. The concept paper will form the basis for an EU-wide review of the guidelines. This process will comprise of targeted discussions with stakeholders and a public discussion on anticipated changes shortly in 2016.

The EMA appraisal has started with two groups of experts who are carrying out preliminary work. One group is looking at pre-clinical aspects and the data needed from laboratory tests or animal studies to safely start first tests in humans. The other group is looking at clinical aspects of the design of first-in-human trials and how these could be improved to better ensure the safety of human volunteers taking part in these trials. This will lead into one EU-wide expert group discussion on review of guiding principle.

Clinical trials are vital for the improvement of medicines and without them patients cannot expand contact to new potentially life-saving medicines. In the EU, the approval and conduct of clinical trials is within the remit of the relevant authorities of the European Member States.

EU guidelines are in place to ensure that these clinical trials are conducted as safely as possible.

Severe adverse reactions in healthy volunteers such as those experiential in the trial in Rennes are extremely rare during clinical trials. Since 2005, approximately 14,700 phase I clinical trials (with participation of 305,000 subjects) have been conducted in the EU, including 3,100 first-in-human studies. Only one other rigorous incident has been previously reported in that time in the EU

Date: 27/05/2016

http://www.ema.europa.eu/ema/index.jsp?curl=pages/news_and_events/news/2016/05/news_detail_002538.jsp&mid=WC0b01ac058004d5c1

The European Medicines Agency (EMA) has suggested the conceding of marketing authorizations in the European Union (EU) for two new permutation therapies against chronic hepatitis C virus (HCV) infection, Epclusa and Zepatier.

HCV infection is a foremost European public health confront. It affects between 0.4% and 3.5% of the population.

Epclusa and Zepatier are from new generation of medicines for chronic HCV disease, direct-acting antivirals, that give high rates of cure of HCV infection and that have, in the past few years, reform the way this disease is treated. These medicines obstruct the action of proteins which are necessary for viral imitation. Epclusa targets the proteins NS5B and NS5A, while Zepatier targets the proteins NS3/4A and NS5A.

These new treatment allow cure of patients with chronic HCV infection without the need for interferon medicines which are linked with poor acceptability and severe side effects. In spite of the rapid development of new therapies, including interferon-free regimens, doctors and patients can still gain advantage from substitute treatment options.

Epclusa contains sofosbuvir, already approved under the name Sovaldi and as a combination therapy with ledipasvir under the name Harvoni, and velpatasvir which is a novel HCV protein inhibitor. The safety and effectiveness of Epclusa was tested in clinical trials involving over 2,000 patients, to review that the HCV was no longer detected in the blood 12 weeks after the end of treatment with or without ribavirin. Majority of the patients across all genotypes had no evident virus in their blood 12 weeks after the end of the treatment and could therefore be considered to be cured of their HCV infection. The most common side effects reported in clinical trials were headache, fatigue and nausea.

Zepatier contains two novel HCV protein inhibitors, grazoprevir and elbasvir. This fixed-dose combination of direct-acting antivirals targets genotypes 1 and 4 of the disease.

The effectiveness and protection of Zepatier was tested in clinical trials involving approximately 2,000 patients. The medicine also showed a persistent virologic response. The safety of the medicine was also measured approving and the most common side effects reported in clinical trials were fatigue, headache and nausea.

Date: 27/05/2016

http://www.ema.europa.eu/ema/index.jsp?curl=pages/news_and_events/news/2016/05/news_detail_002537.jsp&mid=WC0b01ac058004d5c1

The European Medicines Agency’s (EMA) 2015 annual report published emphases on the Agency’s central chores which are basically:

• Assessment of medicines
• Promoting research
• Development of new and advanced treatments
• Checking of the benefits and risks of medicines in real life

In 2015, the Agency suggested marketing endorsement for 93 medicines for human use, which comprise 39 new active ingredients, and 14 medicines for veterinary use, containing seven new active elements.

About one in two applicants who received a positive view for their medicine had received scientific guidance from EMA during the progress phase of their product; this figure rises to 85% for medicines containing a new active ingredient. Scientific guidance is EMA’s key tool to stimulate the collection of high quality data on the advantages and risks of medicines.

This is a positive trend showing that patients increasingly make use of the opportunity to directly report on side effects they experience. For human medicines, more than 1.2 million reports on suspected side effects were recorded. Over 48,000 reports instigated from patients in the European Economic Area, a 30% rise compared to 2014.

In 2015, the product information on many medicines was modernized as a new safety information had become available.

EMA’s annual report also shows some of the main projects, initiatives and achievements in 2015 that have had an important influence on the Agency and the way it functions. These are:

The preparation for the launch of Priority medicines to the maintenance and the advancement of medicines that address unmet medical needs

Initiatives to help fight antimicrobial resistance and other public health threats such as Ebola as well as the execution of various new pieces of legislation including a range of new activities to further support the safety checking of medicines.

The report also covers three interviews with stakeholders and EMA representatives on topics of major interest in the area of medicines and health in 2015, comprising:

• Best way of alignment for data requirements from regulators and health-technology-assessment (HTA) bodies.
• The way immunotherapies bring new hope to cancer patients.
• Use of big data in healthcare is challenging or an opportunity.

Date: 17/05/2016

http://www.ema.europa.eu/ema/index.jsp?curl=pages/news_and_events/news/2016/05/news_detail_002531.jsp&mid=WC0b01ac058004d5c1

The rising and emergent issue of high medicine prices and its effect on the sustainability of health care systems is getting more and more consideration in many countries around the world. Regulators are ready to play their part in solving the crisis and in facilitating sustained access of patients to safe and useful medicines.

In the article published in the New England Journal of Medicine (NEJM) two representatives of the European Medicines Agency (EMA), i.e. its Executive Director and Senior Medical Officer, as well as Heads of two national agencies discuss possible regulatory interventions.

The pricing of medicines is clearly out of their dispatch, medicine regulators cannot overlook the recent debate on the cost of medicines and can make a role to reasonably priced care, give details the authors in their article entitled ‘Drug regulation and pricing – can regulators influence affordability?

The article, co-authored by EMA’s Executive Director Guido Rasi, its Senior Medical Officer Hans-Georg Eichler, the Executive Director of the Dutch Medicines Evaluation Board Hugo Hurts and the president of the German Federal Institute for Drugs and Medical Devices Karl Broich, is available in open access in the NEJM.

According to the authors of the article, there are five main ways European regulators can help:

• Facilitate the quick sanction of generics and biosimilars, as this facilitate competition and drives down prices;
• Works to guarantee ‘me-too’ products (medicines comparable to already approved options) keep on coming on the market at reasonable speed, again to drive down prices through augmented competition.
• Support companies to carry out clinical trials that both assure the needs of regulators (i.e. make obvious quality, safety and effectiveness of the medicine) as well as the health-technology-assessment bodies(i.e. support the revelation of the value once the medicine is authorized, to guide payers in their reimbursement decisions).
• Assist the collection of other data that are important for payers by taking their needs into account when asking companies to conduct post-approval studies.
• Maintain higher competence of research and development in the area of medicines: by development a better model of medicines, it is anticipated that companies would potentially be able to decrease the price of their medicines. This could also mean reflecting on new approaches to medicines’ development, such as the adaptive pathways approach that is being explored by EMA.

Date: 12/05/2016

http://www.ema.europa.eu/ema/