Category Archives: NHS

ABPI expert urges to find new ‘blockbuster treatments’ for brain tumors

Wax Selection – Leaders in Pharma, Biotech & MedTech Recruitment

With the Government set to invest an additional £20 million into the research, diagnosis and development of treatments for brain tumours, we need to talk more about how we are going to find the next blockbuster treatments for these devastating diseases.

Nearly 11,500 people are diagnosed with a brain tumour every year in the UK with fewer than 15% surviving beyond 10 years. This week’s announcement from the from the Department of Health and Social Care – following the death of Dame Tessa Jowell – that they would be doubling investment for brain cancer research to £40 million is a welcome commitment to helping achieve a goal our industry shares: finding innovative new treatments and cures for these diseases.

The science is advancing in laboratories here in the UK and around the world, funded and supported by charities, universities and the pharmaceutical industry, collectively we are working to fight back against this terrible disease.

Among the 7,000 medicines currently being developed by the global pharmaceutical industry, there are 58 medicines in the pipeline for brain tumours, including gliomas. Companies are actively working to find better ways to speed up medicines development to get treatments to patients sooner.

In her speech to the House of Lords in January, Dame Tessa Jowell talked candidly about her glioblastoma diagnosis and called for greater collaboration in the fight against cancer. She also talked about the speeding up of drug trials by testing more than one at a time, saying: “I am not afraid, but I am fearful that this new and important approach may be put into the ‘too difficult’ box.”

The type of clinical trials Tessa Jowell talked about have many different names: adaptive randomisation, drop-the-loser, adaptive dose-finding, adaptive seamless and the list goes on.

The one thing they all have in common is flexibility. In trials like this – that we call adaptive design clinical trials – researchers can see how patients are responding to treatments and then change or stop parts of the trial in real time.

When used appropriately, trials like this may improve efficiency, reduce cost, maximize information gained and minimize risk to the patients and sponsors. Ultimately, drug development can be accelerated so that the right treatments can be delivered rapidly to the right patients. The UK is seen as a pioneer of innovative clinical trials and this involves collaboration between academia, the NHS, industry and medical research charities –  we must ensure we keep it that way in the future.

The issue is that these clinical trial types are not easy to design, plan or execute. An adaptive design will not rescue a poorly planned trial or ineffective treatment.

We need to make sure the regulatory authorities in the UK are not seen as a barrier to innovation; the MHRA and HRA are open to discussion and we need to encourage researchers and pharmaceutical companies to start conversations with them early in the process of planning an innovative clinical trial.

We think that adaptive design clinical trials could be the solution to speeding up the research and development of not only brain tumor treatments, but for all sorts of diseases. Research into small or rare patient populations could really benefit from these trials since they help us quickly rule out the drugs or drug combinations that aren’t working and give more patients the option to contribute to research and clinical trials.

We’re not alone. In February, the Department of Health and Social Care published their brain tumor research report which stated that, because brain tumors are one of the areas that have small patient populations, we need to think differently about how we conduct clinical trials and incorporate innovative trial designs.

The report provided practical recommendations for how we can work collaboratively to make quicker progress in this area. The next steps are to build on the UK’s existing strengths, ensure we have access to researchers with the right skills, and make sure that the right infrastructure is in place for us to make really make progress in this area.

Alongside their funding announcement, we welcome the Government’s commitment this week to accelerate the use of adaptive design trials. When used appropriately, drug development can be accelerated so that the right treatments can be delivered rapidly to the right patients – and that’s where the real benefit lies.

As we look to the future of cutting-edge research and development for blockbuster treatments, we know we need to make the case for innovative clinical trial design, talk more about the amazing science our researchers, companies and NHS are pioneering and encourage them to have open conversations with the UK regulators to ensure that innovative clinical research is safe and effective.

Together, we won’t rest until devastating brain tumours are a thing of the past.

SOURCE: www.news-medical.net/news

Life Sciences sector responds to report on the impact of Brexit

Wax Selection – Leaders in Pharma, Biotech & MedTech Recruitment

The Business, Energy and Industrial Strategy (BEIS) Committee report calls on the Government to secure a post-Brexit deal to protect patients and the UK’s pharmaceutical industry.

“The impact of Brexit on the pharmaceutical sector’ makes several recommendations which industry welcomes. This includes the need to secure the closest possible regulatory alignment with the EU as well as minimum border friction. Patients are at risk of harm and the UK pharmaceutical sector could lose its status as a world leader,” the report says.

The Committee also concluded that “what little benefits there may be from regulatory divergence, these would be greatly overshadowed by the costs and loss of markets and influence the UK would face.”

A joint statement by the Association of the British Pharmaceutical Industry (ABPI) and the UK BioIndustry Association (BIA) – whose chief executives, Mike Thompson and Steve Bates, provided evidence to the Committee – said:

“Every month, 45 million packs of medicine move from the UK to the EU, with 37 million moving the other way.

Today’s Select Committee Report is right – a Brexit ‘no deal’ would significantly damage public health, patient access to medicines and the UK’s leading pharmaceutical sector. This must be avoided at all costs.

“Securing cooperation on the regulation, trade and supply of medicines must be a priority for both the UK Government and the EU.”

The ABPI represents innovative research-based biopharmaceutical companies and is recognised by government as the industry body negotiating on behalf of the branded pharmaceutical industry, which supplies more than 80% of all branded medicines used by the NHS.

SOURCE: www.manufacturingchemist.com/news

SMC approves licence for liver cancer treatment

Wax Selection – Leaders in Pharma, Biotech & MedTech Recruitment

Stivarga® (regorafenib) has been accepted by the Scottish Medicines Consortium (SMC) as a monotherapy for the treatment of adult patients with hepatocellular carcinoma (HCC) who have been previously treated with Nexavar® (sorafenib).1

Regorafenib is the first medicine to be specifically licensed for second-line use in patients with HCC who had formerly been treated with sorafenib, the German multinational pharmaceutical company Bayer has announced.

The medicine is taken orally and works by slowing down the growth and spread of cancer cells by cutting off the blood supply that keeps cancer cells growing.2

Judi Rhys, chief executive of the British Liver Trustsaid: “A diagnosis of hepatocellular carcinoma (HCC) is truly devastating – it is a horrendous type of liver cancer that is often diagnosed very late with few treatment options.

“We are delighted that the Scottish Medicines Consortium (SMC) has accepted the Trust’s evidence on behalf of patients and agreed to the use of this drug for patients in Scotland.

Evidence shows that outcomes for people with advanced liver cancer are particularly poor, so this is an important step.”

She added the decision “highlights a two tier system where patients in other parts of the UK are denied access to this new treatment that can improve outcomes”.

The positive SMC announcement follows the recent decision from the National Institute for Health and Care Excellence (NICE) to not recommend the use of regorafenib on the NHS in England.3

Amanda Cunnington, head of patient access, Bayer UK said regorafenib was “the first advancement in licensed treatment for liver cancer patients in nearly a decade”and that it offers “the first and only approved second-line systemic treatment option which could significantly improve patients’ overall survival”.

Regorafenib is licensed based on data from the international, multicentre, placebo controlled Phase III RESORCE [Regorafenib after Sorafenib in patients with hepatocellular carcinoma; NCT 01774344] trial. The trial investigated patients with HCC whose disease had progressed during treatment with sorafenib.4

In the trial, regorafenib plus best supportive care (BSC) was shown to provide a statistically significant and clinically meaningful improvement in overall survival (OS) versus placebo plus BSC (10.6 vs. 7.8 months, respectively, (HR 0.62; 95% CI 0.50-0.79; p=0.000017)) which translates to a 37% reduction in the risk of death over the trial period.4

Adverse events observed in the RESORCE trial were generally consistent with the known safety profile of regorafenib.4 The most common (>=30%) treatment-emergent adverse events were hand–foot skin reaction, diarrhoea, fatigue and hypertension.4

HCC is the most common type of primary liver cancer.5 Liver cancer is a difficult-to-treat cancer with an annual mortality rate of 48,000 in the EU.6 Globally, it is the second leading cause of cancer-related deaths.6In the UK, there are over 5500 new cases of primary liver cancer diagnosed each year, which is around 15 patients each day.7

References

  1. SMC. regorafenib 40mg film-coated tablets (Stivarga®). SMC No 1316/18. Bayer plc. April 2018. Available at: http://www.scottishmedicines.org.uk/files/advice/regorafenib__Stivarga__FINAL_March_2015Revised_250315_for_website.pdf (Last accessed May 2018).
  2. European Medicines Consortium (EMC) Stivarga® Patient Leaflet. Available at: https://www.medicines.org.uk/emc/files/pil.1263.pdf (Last accessed April 2018).
  3. National Institute for Health and Care Excellence (NICE) Regorafenib for previously treated advanced hepatocellular carcinoma. Technology appraisal guidance [TA514] Published date: 21 March 2018.  Available at: https://www.nice.org.uk/guidance/ta514/chapter/1-Recommendations  (Last accessed April 2018).
  4. Stivarga® (regorafenib) Summary of product characteristics. Bayer HealthCare. September 2017. http://www.ema.europa.eu/docs/en_GB/document_library/EPAR_-_Product_Info… (Last accessed April 2018).
  5. Cancer Research UK. Liver Cancer Types. Available at: http://www.cancerresearchuk.org/aboutcancer/liver-cancer/types. (Last accessed April 2018).
  6. GLOBOCAN 2012: Estimated Cancer Incidence, Mortality and Prevalence Worldwide in 2012.http://globocan.iarc.fr/Pages/fact_sheets_cancer.aspx (Last accessed April 2018).
  7. Cancer Research UK. Liver Cancer Incidence Statistics. Available at http://www.cancerresearchuk.org/health-professional/cancer-statistics/statistics-by-cancer-type/livercancer/incidence#heading-Zero  (Last accessed April 2018).

SOURCE: www.hospitalpharmacyeurope.com/editors-pick

Vertex says ‘some way’ to CF drug price deal after NHS meeting

Wax Selection – Leaders in Pharma, Biotech & MedTech Recruitment

Representatives of Vertex Pharmaceuticals and NHS England met last week in an attempt to end a two-year long stand-off over the price of cystic fibrosis drugs – but there is still a long way to go before the matter is resolved.

Pressure from patient groups forced a parliamentary debate on access to Vertex’s CF drugs, after NICE rejected the company’s Orkambi combination therapy in 2016.

An online petition attracted more than 100,000 signatures, the threshold for a discussion in Parliament, and ministers have written to Vertex asking for the matter to be resolved.

Vertex is asking the NHS for a deal covering the price of its portfolio of CF drugs, including those that are yet to be approved, and will expand the proportion of the disease population who will be eligible for treatment.

The company confirmed in a statement that Vertex met with representatives of NHS England last Wednesday.

But a spokesperson added: “Both parties recognise there is still some way to go to reach an agreement and Vertex is committed to working together to achieve this. We share the cystic fibrosis community’s sense of urgency and have agreed to meet again in the coming weeks. There’s lots of work to do on both sides ahead of this to progress discussions as quickly as possible.”

However there is hope that there will be further progress in meetings over the coming weeks.

pharmaphorum understands that the biggest stumbling block is that NHS England wants to pay the same price as Vertex’s already-approved and NICE-backed Kalydeco (ivacaftor) for the company’s drugs.

Kalydeco’s list price is around £182,600 per year, although the company has agreed a confidential discount.

Cystic Fibrosis Trust public affairs manager Lynsey Beswick said: “We welcome the news that Vertex and NHS England have had further talks on the company’s medicines. This appears to be positive progress in our goal to gain access to the most advanced new medicines for people with cystic fibrosis at the earliest possible opportunity.”

SOURCE: www.pharmaphorum.com/news

Final NHS nod for Roche’s RoActemra

Wax Selection – Leaders in Pharma, Biotech & MedTech Recruitment

Roche/Chugai’s RoActemra should be routinely offered throughout the NHS to adults with giant cell arteritis (GCA) within the next three months, following a final green light from cost regulators.

Almost 15,000 patients develop GCA in the UK every year. The condition is a potentially life-threatening form of vasculitis that results in inflammation of blood vessels, which can be difficult to diagnose because of its wide range of symptoms, including severe headaches, scalp tenderness and jaw pain. If left untreated it can lead to blindness, aortic aneurysm or stroke.

To date, management of GCA has been limited to long-term high-dose steroids, but this can cause skin problems and weight gain, as well as diabetes and osteoporosis in the long-term. There have been no treatment advances for GCA for nearly 60 years.

RoActemra (tocilizumab) is an anti-IL-6 receptor licensed for the treatment of adult patients with moderate to severe active rheumatoid arthritis, polyarticular juvenile idiopathic arthritis and systemic juvenile idiopathic arthritis in children two years of age and older, and for the treatment of GCA in adults.

Clinical trial results show that after having RoActemra plus a tapering course of glucocorticoids for one year, more people stay in remission and need lower doses of glucocorticoids compared with people having glucocorticoids alone.

The National Institute for Health and Care Excellence is recommending funding for one year’s treatment with the drug for patients who suffer flares of their GCA or may not respond fully to steroids, as their disease is most difficult to control.

SOURCE: www.pharmatimes.com/news

Skin conditions cost NHS £723m a year

Wax Selection – Leaders in Pharma, Biotech & MedTech Recruitment

New research shows that more than three million primary care hours are spent on skin conditions, at a cost to the NHS of £723 million each year.

The research, carried out by the Association of the British Pharmaceutical Industry in collaboration with an independent Dermatology Expert Working Group (EWG), highlights the burden of skin conditions on both patients and the NHS.

According to the findings, around 13.2 million people in England visited their GP with a skin problem in 2016, of which 85 percent said the psychosocial aspects of their condition were a significant part of their illness.

The research also showed that almost 60 percent of patients with severe psoriasis could lose up to 26 days of work a year because of their skin condition.

“Today’s findings bring into sharp focus the staggering amount of time and money the NHS spends on the management of skin conditions in primary care and the significant impact skin conditions can have on people’s lives,” said EQG chair Rt Hon Professor Paul Burstow.

“Ignore dermatology and we miss a huge opportunity to make real and immediate gains for the NHS and for people’s quality of life.”

The group’s report, Making real our shared vision for the NHS: optimising the treatment and care of people with long-term skin conditions in England, makes a stream of recommendations designed to drive efficiencies and improve patient outcomes.

It calls on NHS England to promote and support successful self-management through patient education programmes for specific long-term skin conditions, led by suitably trained healthcare professionals (HCPs), as well as promote promote and incentivise the adoption of technology such as email guidance and smartphone apps.

NHS England should also put in place suitable incentives to encourage commissioners to implement teledermatology pathways to triage patients with skin lesions appropriately and free up face-to-face time for clinicians to see patients with inflammatory skin conditions, it said.

SOURCE: www.pharmatimes.com/news

Five new medical schools to open under ‘biggest ever expansion’ of medical training

Wax Selection – Leaders in Pharma, Biotech & MedTech Recruitment

Five new medical schools will launch across in England in September as part of the government’s ‘biggest ever expansion’ of the NHS medical workforce, the DHSC has announced.

The new medical schools will open in Sunderland, Lancashire, Canterbury, Lincoln and Chelmsford. They have been ‘chosen as part of a rigorous bidding process to help place more medical students in areas which traditionally struggle to attract doctors, particularly rural and coastal areas,’ the DHSC said.

As part of the plans to increase the medical workforce the government is also funding 100 additional places at Aston University’s medical school in Birmingham. Twenty further medical schools will also have additional student places funded.

The DHSC said that by 2020 there will be an extra 1,500 doctors in training. Some 630 of these places will be available from September, taking the total number of medical students for 2018/19 to 6,701, which the DHSC said was the highest on record.

The five new medical schools will be based at:

  • University of Sunderland
  • Edge Hill University in Lancashire
  • Anglia Ruskin University in Chelmsford
  • Lincoln – The University of Nottingham in partnership with the University of Lincoln
  • Canterbury – Joint medical school between Canterbury Christ Church University and the University of Kent

Health and social care secretary Jeremy Hunt said: ‘Setting up five new medical schools is part of the biggest ever expansion of our medical and nursing workforce; which will help us deal with the challenges of having around one million more over 75s in ten years’ time.

‘These schools are being set up in parts of the country where it is can be hard to recruit and attract new doctors – but will benefit doctors everywhere as we start to eliminate the rota gaps that add so much pressure to their work.’

SOURCE: www.gponline.com

Row over CF drug funding as NHS rejects Vertex offer

Wax Selection – Leaders in Pharma, Biotech & MedTech Recruitment

A deal covering new cystic fibrosis drugs is unlikely to happen unless the manufacturer Vertex agrees to drop its prices, NHS England has indicated.

Vertex is seeking a deal to fund national access in England to its cystic fibrosis (CF) drug, Orkambi (lumacaftor+ivacaftor),  a combination which extends the number of CF whose underlying disease can be treated.

Over the last 12 months NHS England has taken a lead role in negotiating directly with pharma companies, and has created a dedicated Commercial Medicines Unit for the task.  Faced with static NHS budgets and growing budget pressure, it is taking a harder line on prices, but also says it is willing to negotiate deals based on outcomes.

However no such deal has been struck on this occasion. In fact CF campaigners have reacted angrily to this NHS England rejection, and Vertex is critical of how the budget holder has conducted the discussion, saying NHS England has only communicated via email.

MPs are set to debate availability of cystic fibrosis drugs in Parliament later today, after a petition calling for them to be funded by the NHS attracted more than the 100,00 signatures.

But for now NHS England has said it is not interested in a deal covering cystic fibrosis drugs, despite the growing pressure from campaigners.

Vertex is keen to get its CF drugs funded in England and the wider UK because of the high number of patients with the disease here.

With 10,000 patients affected, the UK has the second highest number of CF patients in the world and as such would be an important and valuable market for Vertex.

The manufacturer is seeking a deal that would cover Orkambi and any other future CF drugs that it develops as part of a “portfolio approach”.

This was sparked by NICE’s decision to reject Orkambi in 2016, which was too expensive for NICE despite discounts and greater flexibility in funding for rare disease drugs.

Although the negotiations over pricing are confidential, campaigners united under the hashtag #ukneedsorkambi are speculating that NHS England wants Vertex to expand the number of patients receiving treatment from 400, to almost 4,000 but without receiving any extra funding.

Pharmaphorum quizzed NHS England on the details of the negotiations, and a spokesperson responded with a short statement.

The spokesperson said: “The NHS can only offer treatments which are both effective for patients and offer good value for taxpayers, so it’s crucial that drugs companies work with the NHS to get a positive outcome.”

“Following advice from NICE, the NHS has asked this particular drug company to review its proposed pricing, and unless this happens further progress at this time is frankly unlikely.”

A Vertex spokesperson said the company is “astonished and dismayed” with NHS England’s response.

“It amounts to a refusal to make any additional funding available for Orkambi or future medicines,” a company spokesperson said.

“Cystic fibrosis (CF) is a devastating disease where half of people die by the time they are age 31. The situation with CF in the UK is unique and needs a unique solution – this is what our portfolio approach that we proposed in February offers.”

“Negotiations over email are rarely productive and CF patients do not have time to waste. We call on all parties to come to the table to engage in meaningful dialogue about a way forward – on behalf of CF patients.”

SOURCE: www.pharmaphorum.com/market-access-2

Blood test could predict response to breast cancer drug early

Wax Selection – Leaders in Pharma, Biotech & MedTech Recruitment

A new study has found that a blood test for cancer DNA could predict if a woman is responding to the new breast cancer drug palbociclib, months earlier than current tests.

Scientists from The Institute of Cancer Research, London, and The Royal Marsden NHS Foundation Trust, say the test could detect in two to three weeks whether the drug is working, although they caution that the results need replicating before they are used clinically.

The research, published today in the journal Nature Communications, was largely funded by the Medical Research Council (MRC). The researchers tested women with oestrogen receptor positive breast cancer – the most common kind – who were taking part in a clinical trial of palbociclib for advanced breast cancer.

In November 2017, palbociclib was approved for use on the NHS by NICE for women with previously untreated advanced breast cancer.

Currently, women must wait two to three months to find out if palbociclib is working, via a scan.

The new blood test instead looks for circulating tumour DNA – fragments of DNA shed by the cancer that have entered the bloodstream. The DNA mutations associated with the cancer can be detected in these samples.

The researchers found that they could predict if the palbociclib treatment would work by comparing the amount of a gene PIK3CA detected in a blood test before treatment and 15 days after starting treatment. In the study, 73 women had the PIK3CA mutation and were given blood tests before and after starting palbociclib treatment.

In these women, the researchers found that those who had a small decrease in PIK3CA circulating DNA at 15 days had a median progression-free survival (the length of time the patient survived and the cancer did not get worse) of only 4.1 months, compared to women with a large decrease in PIK3CA, who had a median progression-free survival of 11.2 months.

The test could allow the women in the first group for whom the treatment is not as effective to be identified early, so that they could consider altering their treatment.

Professor Nicholas Turner, senior author and Professor of Molecular Oncology at The Institute of Cancer Research, London, and Consultant Medical Oncologist at The Royal Marsden NHS Foundation Trust, said: “Palbociclib is one of a new class of drugs that delays cancer progression for patients with advanced breast cancer, but it’s not effective for everybody. The problem is we have to wait for two to three months before doing a scan to see if the therapy is working.

“Our new study found that a blood test for cancer DNA in the first two weeks of treatment indicated whether the drug was likely to be effective. Having an early indication of how likely a treatment is to work might allow us to adapt treatment – switching some patients to an alternative drug that is more likely to benefit them.”

Dr Nathan Richardson, Head of Molecular and Cellular Medicine at the MRC, said: “This study provides early evidence that might help us understand sooner when a drug is successfully treating breast cancer, and if not, it can be discontinued and better approaches pursued.”

The research also received funding from the charity Breast Cancer Now and the pharmaceutical company Pfizer.

SOURCE: www.pharmafield.co.uk/Pf-Fox-News/General/2018/03

Eisai disappointed after NICE rejects earlier use for breast cancer drug

Wax Selection – Leaders in Pharma, Biotech & MedTech Recruitment

Eisai has been dealt another disappointment from NICE, which says its breast cancer drug Halaven should not be regularly used on the NHS.

The company noted that the Halaven (eribulin) continues to be available for patients in third line – but more recently NICE had been assessing the drug after one chemotherapy regimen.

In the final guidance NICE said it was unclear whether a 4.6 month overall survival increase compared with capecitabine chemotherapy was down to treatments given after Halaven.

NICE noted that the drug did not increase progression free survival compared with chemotherapy, and that Halaven therapy is usually stopped once the disease progresses.

The uncertainty caused means that the cost per Quality Adjusted Life Year for Halaven in this use is around £69,800, according to NICE’s calculations.

This is too expensive for the NHS, even though NICE has extra leeway above its usual £30,000 threshold.

But Gary Hendler, Chief Commercial Officer Eisai Oncology Business Group, and chairman and CEO Eisai EMEA said he was “disappointed” after another knock-back from NICE.

Although NICE recommended funding for Eisai’s Lenvima (lenvatinib) in thyroid cancer last month, this was after a wait of more than two years primarily because of a change to the way NICE and the Cancer Drugs Fund are organised.

Halaven was also removed from the Cancer Drugs Fund under previous arrangements in 2015 because it was overspent. However this decision was eventually reversed and the drug was eventually rubber-stamped for regular NHS funding in late 2016.

Hendler said in a statement: “Eisai is yet again extremely disappointed with a decision from NICE. Metastatic breast cancer patients can only currently access a limited number of new treatments in England, and as eribulin has been shown to significantly improve overall survival in women with this disease it is an important option that they should have access to as early as possible.”

“Denying earlier access to it for these patients will affect their outcomes and as a company focused on making a positive difference to the lives of patients and their families, NICE’s decision concerns us greatly. Thankfully patients can still access eribulin in the third line.”

Eisai, which has built a multi-million pound manufacturing base in Hertfordshire, has threatened to stop investing in the UK because of difficulties getting drugs reimbursed.

This is despite pharma-friendly initiatives such as tax breaks on drugs that are developed and manufactured in the UK.

SOURCE: www.pharmaphorum.com/news