Category Archives: ABPI

From molecule to medicine, the importance of persistence

Wax Selection – Leaders in Pharma, Biotech & MedTech Recruitment

Here, Dr Sheuli Porkess, deputy chief scientific officer, Association of the British Pharmaceutical Industry (ABPI), outlines how the pharmaceutical industry takes a substance from molecule to medicine and how the process requires persistence.

A report last week from the Office of Health Economics (OHE) shows the amazing impact medicines have had on the NHS and more widely. The antipsychotic chlorpromazine, first used in the NHS in 1954, paved the way for deinstitutionalisation and community-based care for people with mental illness. In 1948, there were almost 400,000 cases of measles in England and Wales, and 327 people died. By 2015 the number of cases of measles in England and Wales had fallen below 1,200.

These medicines, and others, had a variety of benefits including better clinical outcomes, saving lives, improving quality of life, greater health service efficiency and wider societal impacts. But making medicines is a complicated and costly business. It costs billions of pounds and can take decades. Successes can change the world; failures are an inevitable part of the discovery and development process. But when medicines get through the development process, they can clearly change millions of lives.

There are broadly three stages to creating a new medicine: research, development and approval. Here’s how it works:

Drug discovery and development

The process usually starts with chemical compounds or biological molecules. With advances in technology over the last few years, we can screen compounds that have the potential to become treatments faster than ever before. AstraZeneca — a British pharmaceutical company — launched a new screening robot in 2016 called ‘NiCoLA-B’ which is able to test 300,000 compounds a day. Its job is to find those chemicals that show the slightest potential of being useful as a medicine.

The research stage benefits hugely from collaborative partnerships between the pharmaceutical industry, charities and universities, all working together to find a potential medicine. This stage can take four to five years and takes about 22% of the total budget it takes to find a treatment. Each compound has a less than 0.01% chance of success.

Preclinical research

From a batch of about 10,000 compounds screened in the drug discovery phase, only about 10–20 go into the pre-clinical phase, where scientists determine how safe a medicine might be through testing in cells and animals as well as using computational models.

Clinical research

If any of those 10-20 compounds show real potential of being turned into something useful, they’re developed in to a medicine that will move into clinical trial stage. There are three steps: Phase I involves about 20 to 100 volunteers. If medicines are successful here, they will move onto Phase II where they are tested in people with the disease.

Phase III can include up to 5,000 patients. Going through the three phases can take six or seven years. Over half, or about 65%, of the money it takes to make a medicine is spent in the development stage.

Phase IV clinical trials are after the medicine has a licence and are there to help monitor the medicine’s safety and help clinicians better understand how the medicine works in everyday life, not just in clinical trials.

Approval

The final stage is when regulators review the medicine and it can get ‘market authorisation’ — which shows the medicine is safe and effective. By this point, the manufacturing of the medicine has been scaled up. Only one medicine of 5,000–10,000 compounds discovered will make it to this stage.

The approval processes last anywhere from six months to two years. The medicine is continually monitored once it starts being prescribed for patients.

Researching and developing medicines takes a lot of time and work along the way; there is no guarantee that any particular medicine will make it through the various stages of this highly regulated process. The process is fascinating and once medicines get through this system, their impact can be huge.

Of course, the pharmaceutical industry is pioneering new ways to find treatments. The future looks exciting and how we detect, diagnose and treat disease is set to change significantly.

Advances in medical technology and the miniaturisation of diagnostics, wearables and devices will have a huge impact on our lives and could help people with chronic diseases to remain out of hospital.

Advances in understanding how cells monitor and repair damaged DNA enables us to develop game-changing treatments for cancer. Progress in immuno-oncology sees patients own immune cells used to attack cancer cells, and stem cell therapy is treating rare sight conditions.

We see AI and synthetic biology used for treating malaria, HIV and hepatitis. Gene-editing technology is happening in labs right now, identifying new disease targets, accelerating the discovery of novel treatments.

Passionate pioneers, such as those who invented the groundbreaking treatments in the report, have always been at the heart of our industry and it’s exciting to imagine what their successors could achieve in the next 70 years.

SOURCE: www.epmmagazine.com/opinion

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

EMA initiates divorce process from the UK’s MHRA

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The first stage of separation for the EMA from the UK came with the decision to relocate its headquarters to Amsterdam, and now the second stage has taken place whereby the EMA has reassigned the MHRA’s role vetting medicines to other countries.

All of this has been brought about by Brexit; the surprise decision to go ahead with uncoupling the EMA’s medicine verification process from the MHRA immediately after the deadline for Brexit will have brought even more uncertainty for UK industry.

It was expected that this decision may have been left until the end of the transition period, which will continue until the end of 2020. However, the EMA has already been swift to react to Brexit – first by announcing immediately that it would be seeking new headquarters and now by preparing early for life possible without the aid of the MHRA by March 2019.

Mike Thompson, Chief Executive of the ABPI, said: “Given that the MHRA assesses up to 20% of EU medicines, it is clearly in the EMA’s interest to continue to draw on its expertise. It would also be disadvantageous for the EU to be unaligned with the UK as the largest biopharmaceutical cluster outside of the US.

“The UK Government has been clear that cooperation on medicines is a priority and we urge both sides to come to an early agreement in the interest of patients and public health.”

Theresa May had previously suggested, in a speech at the beginning of March 2018, that her government wanted the UK to be closely linked with the EMA, but this move by the EMA looks to have potentially disrupted that wish.

It could prove to be problematic to industry, if drug approvals are slowed down by the process of other member states learning to cope with new burdens, but the main concern for the UK is that it risks isolating industry based in the country, which includes giants such as GSK and AstraZeneca, as well as many other smaller companies.

The flipside to this equation is that, for the EMA, any uncertainty that is dependent upon political wrangling at the national level could spell disaster for the agency’s work if agreements are not reached. It is therefore understandable that the agency has forged ahead of relocating the 370 medicines that the MHRA and Veterinary Medicines Directorate (also UK-based) are currently rapporteur or co-rapporteur on.

SOURCE: www.pharmafile.com/news/517104

Vive la révolution! What is the future of biosimilars in the UK?

Wax Selection – Leaders in Pharma, Biotech & MedTech Recruitment

Harriet Lewis, ABPI’s Medicines Optimisation Lead, discusses the future of biosimilars in the UK asking the pertinent question: will they revolutionise healthcare?

The ABPI represents UK-based pharma companies researching the newest and most innovative medicines. Our members make both originator biological medicines and make biosimilar medicines so, for want of a better word, we have a foot in both camps.

We work to support the introduction of biosimilars but also work to ensure there is a sustainable and active competitive market. To understand why we talk about biosimilars and the biosimilar market in a unique way, it is important to take a step back.

I am a pharmacist — not a scientist — so I am constantly amazed by the science behind biosimilar medicines that is both fascinating and complex. Put simply, biosimilar medicines are made from living cells and since living cells are all slightly different, there are also variations between biosimilar medicines. This so-called heterogeneity is part and parcel of biological medicines: chemical difference that do not affect the clinical effectiveness of the medicine.

When producing biological medicines, companies must demonstrate the reproducibility of their drugs time-after-time and within certain parameters. Before they even get to the point of clinical trials in humans they are required to demonstrate successful reproducibility. As a result of the complexity of manufacturing and regulation, bringing these products to market is time-consuming and expensive.

One of the early key discussion points around biosimilars is how they should be defined and therefore how they should be treated. Biosimilars are neither generic medicines nor novel treatments. Definitive guidance from the European Commission sets out that they are in fact a ‘version of an active substance’ within an already approved medicine.

Manufacturers must convincingly demonstrate the similar nature of these products. Regulators require biosimilars to demonstrate a comparable clinical effectiveness to originator products. The European Medicines Agency (EMA) goes so far as to say that the ‘concept of biosimilarity is applicable to any biological medicinal product’.

Just as with any medicines, there are class-based risks associated with a biosimilar medicine’s pharmacology, potency or bioavailabilty over time, and these are monitored on an ongoing basis. Pharmacovigilance is a necessary component to the success of biosimilars and ensures that regulators can trace where biosimilar medicines are used to track immunogenic side effects should they arise.

We can only know so much about any medicine, including biosimilars, before they are licensed. Information and our understanding of how they work in patient populations is continually built-upon once the medicine become available to patients: pre-authorisation clinical studies are often insufficient in highlighting all adverse effects. Across Europe, the pharmacovigilence regulation requires that all adverse events for biologics are reported by brand name and batch number. In England, prescribers are encouraged to prescribe by brand name and NHS providers to record product name and batch number to provide an audit trial for product identification and traceability.

The impact of biosimilar competition and what comes next

The European biosimilars market has grown exponentially since the first medicine was launched back in 2006; there was a sudden uptick from 2012 and if you look at any timeline outlining the number of medicines launched what is abundantly clear among the many successes are also a number of failures.

Once you have started manufacturing a biosimilar medicine, it isn’t a done deal that you’ll have a successful medicine. In that respect, biosimiliars are indistinguishable from originator products.

There are also strict regulatory challenges for biosimilar medicines and regulators take time to assess and approve them. This is unlike generic medicines, which are often licensed relatively quickly and are made available to patients on much shorter timescales.

While we are seeing a fast-growing market in biosimilars in all areas, it is also becoming apparent that they might not always be commercially viable.

It has become clear from the rapid expansion of the biosimilars market, according to the QuintilesIMS European study ‘The Impact of Biosimilar Competition in Europe’, competition drives down price. The report also shows there is a relatively weak correlation between biosimilars market share and price; this is a fast-growing market that will inevitably provide complex medicines at more affordable prices, switching to biosimilar medicines may release savings in health systems that should be reinvested in patient care and not used to plug financial deficits.

We know that switching from originator projects to biosimilars is being heralded by the NHS as the next step in delivering savings to health systems, although the predicted savings on the scale suggested have yet to be delivered. Actually, it’s not switching per se but the introduction of competition, in which both the biosimilar producers and originator biologic manufacturers compete (as the IMS Health study has demonstrated). Regardless, the decision to switch should be clinically-led: considerations must be made to ensure patient access to innovative new medicines and that those are made between patients, clinicians and doctors.

There is also a burden associated with switching to biosimilar medicines both in the financial sense but also as a burden on the clinical support staff that need to manage the change. Education is a key part of these decisions. To support clinicians considering switch programmes there are a number of important documents setting out the process for clinician and patient engagement, including ‘Considerations for physicians on switching decisions regarding biosimilars’ a joint document from European Biopharmaceutical Enterprises (EBE) European Federation of Pharmaceutical Industries and Associations (EFPIA) and International Federation of Pharmaceutical Manufacturers and Associations (IFPMA), but also on the EMA website, to show clinicians some of these toolkits available to make informed decisions about switching.

At ABPI we are working closely with our partners: the BIA, the BGMA and the BBA, as well as the regulators, to make sure that we have a sustainable and active market so that patients do actually benefit from the innovation seen in this growing field of medicines.

We continue to work closely with NHS England on a range of activities to highlight how competition within the biologics market is beneficial for patients. The ABPI recently advised on a forthcoming NHS commissioning framework document which will provide guidance to NHS commissioners about biosimilar switching programmes. We look forward to seeing the final version and hope that our industry’s insight has been considered and that the benefits to patients of an effective biosimilars market here in the UK are included.

The phenomenally complex manufacturing process involved in making biosimilars is really leading contemporary science in this field and could potentially revolutionise the treatment of some conditions.

SOURCE: www.epmmagazine.com/opinion

ABPI bid to overturn NICE’s budget impact rules fails

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The UK pharmaceutical trade body has failed in an attempt to force a judicial review of the new evaluation process introduced by the National Institute for Health and Care Excellence (NICE) earlier this year.

The Association of the British Pharmaceutical Industry (ABPI) has been fighting a legal action to try to overturn NICE’s introduction of a £20m cost ceiling for new drugs that have already been assessed as cost-effective, a measure that could allow it to delay introduction for up to three years.

The High Court rejected the ABPI’s application to have the changes to the evaluation process struck down, which the trade body claimed were “inappropriate and unworkable”, would limit patient access to new treatments and lay outside the cost-effectiveness organisation’s remit. It said around one in five new medicines would be affected by the policy.

In a statement, it said: “The ABPI is disappointed that the judicial review application has been turned down. It’s now appropriate for us to take time to reflect on the judgement with our members and decide next steps.”​

NHS England said in a statement that the High Court “has rejected ABPI’s flawed legal manoeuvres which the judge said would ‘produce an absurd result”.

“Rather than attempting to further frustrate NICE and the NHS’ work to ensure patients and taxpayers get maximum value out of the £15bn being spent on drugs, it now makes sense to work together towards that shared goal,” it continued.

In addition to the £20m budget cap, NICE also introduced a fast-track approval mechanism for treatments that offer ‘exceptional value for money’, and new rules on how it evaluates treatments for very rare conditions, raising the upper threshold limit to £300,000 from £100,000 for treatments considered to offer substantial clinical benefit.

Last year, the NHS spent £16.8bn on medicines, according to the British Medical Journal, up 8% on the prior year and almost £4bn more than was spent in 2011.

SOURCE: www.pmlive.com/pharma_news