The price is right?

by John Pinching 24. October 2016 08:22

Pricing, access, transparency – the common denominators which draw the sectors together and often tear them apart


Pharma pricing, medicine access and data transparency are the tags subliminally graffitied across every medical institution, hospital and government building across the UK.

Indeed, I was at the Westminster Health Forum in Central London for a frank discussion on these three ubiquitous subjects.

Proceedings were reassuringly held together by a ‘Churchill’ – in this case, Jo, MP for Bury St Edmunds. She is a proper politician whose two battles with cancer inspired her to make a difference. “It is so important that patient outcomes lie at the heart of all decision-making,” she insisted.

Among the speakers was Nicole Mather – Director of the Office for Life Sciences. She seemed optimistic about the post-Brexit scene. “The UK is a magnet for life sciences and we are the best in the world in the area of genomics,” she said. “GSK is still investing £350m in their Cambridge development and the message we have to the world is ‘Britain is open for business’.”

She clearly stated that the Accelerated Access Review would not be changed as a result of leaving the EU and that it would be focussing on collaboration between the NHS and innovators to create a vision that works for patients. She also emphasised that the collection of data on patient outcomes would be the first step in building a new strategy.

On the subject of evolution, Dr Ian Hudson, Chief Executive, Medicines and Healthcare Products and Regulatory Agency, outlined some of the challenges: “We should be developing innovation appropriately to bring products to market, while maximising development opportunities.”

He also recommended the greater use of real world data monitoring, adding: “Early access should be weighed up against the risk and knowledge of a product.”

Meanwhile, Professor Gary Ford, Chief Executive, Oxford Academic Health Science Network, raised the subject of pricing. “We need to have another look at prescription charges. Why should a 20-year-old with asthma pay for a prescription, when a 61-year-old Chief Executive pays nothing?”

This was perhaps the best ‘counter’ argument I have ever heard.

“There is not much data on ‘appropriate prescriptions’ – are patients getting the right drugs?” he inquired. “More patients need to be involved in decision making.”

Professor Karl Claxton from the University of York is a sort of punk rock Professor of Economics, with an earring and a refreshingly defiant pro-pharma chorus. “There is a discrepancy between global changes and what the NHS is prepared to pay,” he rallied. “NICE does assessments well, but it is not enough. Innovative medicines are still rejected or thrown into the Cancer Drugs Fund.”

He was adamant that there was a solution: “Discounts impact on the global market and manufacturers must be protected. Early uptake should be incentivised, innovations should be rewarded and rebates removed. We have an opportunity to repair the system – patients, clinicians and pharma can all win.”

And Karl’s right – his argument strikes at the beatless heart of a ‘Poundland’ Britain where short term tat is preferred to long-term quality. Desperately bartering with pharma companies, even after a considerable discount, is not going to endear them to research and develop in the UK. In contrast, paying a fair price has long-term healthcare, but also socio-economic benefits, which – in the blur of austerity – politicians would do well to recognise.



A view from the expert

Julian Given is Chief Officer, Washington Community Health Care (GP federation) and Project Manager, City and Vale Alliance (CAVA, GP Federation)

Access to medicine has always been variable from CCG to CCG. Now there is a focus on what is termed ‘rationing’, which started with the failed attempt – by New Devon CCG in 2014 – to restrict surgery to smokers and the overweight, until patients had made health improvement gains.

This has now re-emerged with CCGs looking at areas that ‘can only be paid for by the local NHS in certain restricted circumstances’, which includes surgery for sleep apnoea and hysterectomy for heavy menstrual bleeding. Meanwhile, North Staffordshire CCG has decided not to routinely fund hearing aids for patients with 'mild' hearing loss.

As the 44 Sustainability and Transformation Plan (STP) footprints mature – with their planning documents being published this year – we are likely to see more uniformity across STP constituent CCGs in terms of medicine access.

One of the main objectives of the STPs is for all sectors of health and social care to work closer together, with the view of delivering cost savings and better care for patients and, if this is to be achieved, data transparency and sharing has to be key. Currently, patient data between secondary care, primary care, social care and other patient-facing services is not always routinely shared.

Although many remember the recent failure of, a successor – currently known as the new ‘digital services platform’ – is being taken forward with a view to bridging this gap of information continuity. With the phasing out of Read Codes, and the move across to the SnoMed coding system – which has the ability to be used by primary, secondary and social care – the direction of travel is only one way.

Meanwhile, with the change in focus moving to collaborative working between partner organisations, pharma pricing is going away from the old style view of ‘what is the cheapest product’ – which simply protected the medicines management budget – to now looking at the financial impact on the whole system. Therefore, a slightly more expensive product, which is more efficacious, easier for the patient to administer and supports improved patient compliance would, perhaps, be considered more favourably than it may have been in the past.



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Pf Magazine: October issue out now! Read it here.

by John Pinching 11. October 2016 10:29





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Can ancient needle therapy really turn back time?

by Emma 10. October 2016 08:34


Don’t believe the hype: health headlines dissected




When two researchers at Wuhan University in China were reviewing a couple of published studies – from 2012 and 2013 – which showed that people who had undergone acupuncture performed 10% better in mental tests, they hit upon a new hypothesis. If lower mental test scores are linked with earlier progression of dementia – and acupuncture improves those mental test scores – could the ancient Chinese treatment also slow down its progression?




Researchers Min Deng and Xu-Feng Wang conducted a systematic review and meta-analysis of five earlier Chinese studies, which involved 568 patients. All of the trials involved subjects with mild cognitive impairment (MCI), a condition which leads to a decline in memory. Around one in 10 people with MCI will go on to develop a form of dementia within a year, usually Alzheimer’s disease.

The review’s aim was to collate the available evidence from previous randomised – or partially-randomised – controlled trials (RCTs), which addressed the safety and efficacy of acupuncture for treating MCI.

The trials compared a group who received acupuncture for MCI – on its own or with another treatment – with a control group, which was given another type of active treatment. A group of 288 received acupuncture and 280 people in control groups received nimodipine. Meanwhile, the acupuncture group in two of the trials were also given nimodipine.




The review and meta-analysis revealed that those in receipt of acupuncture scored better on two recognised cognition tests than those who took nimodipine alone.

Combining acupuncture and nimodipine also significantly improved scores when compared to the dementia drug alone.

The researchers concluded that acupuncture: ‘has a significant positive effective on cognitive and memory function’.




Although the results of the review appear encouraging at first, there were a number of shortcomings which cast doubt on the Daily Mail headline that suggests acupuncture staves off dementia. Only five relatively small studies were included, all in China. No trials used placebo/sham acupuncture, so therefore it must be assumed that both subjects and scientists were aware of the treatment being given, which could introduce bias.

Furthermore, the trials only looked at changes in cognitive test scores, and did not assess whether an outcome of acupuncture treatment – combined with nimodipine or not – actually affected an outcome of diagnosed dementia.

Commenting on the research, Dr James Pickett, head of research at the Alzheimer’s Society, said: ‘The lack of strong and convincing evidence makes it difficult to reach any meaningful conclusions.’


What the press said:

‘Could acupuncture stave off dementia?’ MailOnline; ‘Acupuncture may stop memory loss that precedes dementia’

‘Acupuncture prevents memory loss?’ World Report Now





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In the balance: Looking into NHS’s dilapidated purse

by Claudia Rubin 6. October 2016 09:19


Claudia recklessly stares into the NHS’s dilapidated purse

Nearly 70 years ago when the NHS was established – principally as an urgent care service – the population of Britain was about 47 million. Life expectancy for men was 66 – it’s now 77, infant mortality was 24 per 1000 live births – now down to five, and there were three mass immunisation programmes – that figure is now more like 15.

Since then, starting with the rapid development of antibiotics from the 1940s, through to the advancing of scientific research, healthcare is almost beyond recognition today, as are the demands on the service that delivers it.

With more than half of people over 70 living with at least two long-term illnesses –  and a quarter with at least three – it is no surprise that 70% of NHS spending now goes on managing that situation.

As a reflection of this, the NHS that operates today is not the same as the one set up in 1948 – numerous ‘top-down reorganisations’ have tinkered with it, or attempted wholesale change. Some fundamentals do, however, remain – the key one being the NHS’s system of funding.

A taxpayer-funded system, entirely free at the point of use, is a core NHS principal. Few in Britain argue that we need abandon this model entirely, but as with any complex infrastructure, there are no black and white choices, but a range of options within. While we know the NHS lacks funds, and that it has experienced these crises before, it is entirely possible that we have now reached a new low. Nine out of 10 Trusts are spending beyond their budgets, with an overall NHS funding gap of £20bn beckoning, by 2020.

Since most people have some awareness of the current NHS finances, it is astonishing that there hasn’t been a fundamental discussion about what the NHS should provide, what the public’s expectations are of free specialist treatment, and whether it’s ever going to be acceptable to charge for some services.

Indeed, whenever a known treatment or service is not made universally available – the new meningitis B vaccine for example, or various chemotherapy treatments, or GP appointments made at the whim of patients – some feel outrage without hesitation. Evidently the public do have an opinion.

Headlines in recent weeks lamenting the ‘rationing in the NHS’ and implying that it is something new, highlights the paucity of debate and lack of public awareness. Specific issues do trigger discussion from time to time, such as the recent survey by the British Dental Association, which revealed that 600,000 people a year seek a free GP appointment for toothache – costing the NHS at least £26 million a year.

Broadly speaking, however, consumers of NHS services come armed with a culturally inbuilt sense of entitlement with little regard to resource rationing. The latest headlines were triggered by a survey of doctors that found that seven out of 10 had witnessed restriction in approved NHS services and treatments in the past year, mostly for financial reasons.

Rationing of NHS services is not new and takes place across the board. From laboratory to hospital bedside, clinically effective new treatments are often denied entirely to NHS patients or – as this survey found – as a necessary ad hoc reaction to a budget deficit. This is precisely why NICE was set up in 1999 – to reduce variation in the availability of services – and why it continues to regularly make decisions based on cost, denying patients access to potentially worthwhile treatments.


Nostalgia tip: Claudia consigns sentiment to clinical waste

To be clear – no amount of efficiency savings or structural reorganisations will cut it – more money is needed if we want the same service, let alone an improving one. Or instead, let’s agree that we don’t. But headlines decrying the NHS for ‘forcing’ people to re-mortgage their homes to pay for expensive cancer treatment, while the same paper’s readership consistently refuses to accept any tax increases to fund these treatments, smacks of hypocrisy.

If we are so fiercely protective of our NHS, regularly topping as it does the list of things that make us most proud to be British, then why is it so politically risky to put forward the case that we all need to pay more for it?

And what of the NHS being the envy of the world? True perhaps, for the fondness its customers feel for it, but not in its service delivery or outcomes. Though there are metrics of which the NHS can be proud – our vaccines rates for example – we still fall behind in cancer, stroke and heart disease survival rates.

When researchers make scientific breakthroughs, unearth new technologies and unlock genetic secrets with the aim of developing long sought after cures, they want to see them benefit patients, and we want them for our families.

So, if you were that researcher, that scientist striving for the next big discovery, would you want your industry to engage with this wide debate? When your operation is delayed or your wife/husband refused treatment, do you want your political representatives to lead in helping us to understand why the NHS makes these decisions and consider what alternatives there may be?

The pharmaceutical industry should absolutely be doing all it can to reduce the prices it charges for medicines and increase the value they deliver. But should pharma also drive forward the message that the incredible improvement in healthcare that has transformed our lives – and in which it has played no small part – can continue at an ever-increasing rate, only if we are willing to pay more for it?

The discovery of new vaccines, diagnostics, medical technologies and medicines is in all of our interest. If we want GP surgeries to be open at all hours, hospitals to offer operations at weekends and equal access to the best medicines when we fall ill, we might even conclude that we are willing to handover more of our personal and national wealth to help the NHS deliver it.




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Welcome to the real world

by Amy Schofield 26. September 2016 08:42



The UK is poised at the rock face of real world data, but how successfully can it be mined?


Real world evidence (RWE – don’t try and say it) is transforming drug development. The use of large data sets – to determine how new drugs perform – beyond the limited reach of pre-approval clinical trials, helps pharmaceutical companies, healthcare providers and payers to determine the safety, efficacy and cost efficiency of a medicine. All this is can be achieved by extracting real world data from the records of many thousands of patients.

The evidence from data – generated through a range of resources across real life settings, including patient registries, electronic health records, insurance data, the web and social media – has the potential to accelerate the development of innovative medicines. This will transform the existing approach to health and healthcare, by finding out the cost of using a treatment in the real world, and establishing the real pattern of a product’s use during its lifecyle.

But what are the opportunities and benefits to be gained by RWE, what challenges remain, and what part is the UK playing?


ABPI view: Lead and others will follow

The UK is viewed as a rich mine of patient data for the collection of RWE. Shahid Hanif, ABPI Head of Health Data and Outcomes, believes the UK occupies a unique position: “As well as having an innovative and thriving research science base, and the internationally-renowned National Institute for Health and Care Excellence (NICE), the UK is regarded as a great source of patient data for real world evidence research,” he explained. “The NHS has a unique nationwide role in both providing care and collecting health data throughout patients’ lives, which – if used appropriately – could transform that care in the future.”

The report from a RWE workshop, held by the ABPI in partnership with the Academy of Medical Sciences, stated that the UK is at the vanguard of the future direction of RWE: ‘The UK has a very real opportunity to develop leadership in this field, and influence the development of a consistent approach across global regulators and other stakeholders.’


Home advantage

Consultant pharmaceutical physician Dr Martin Goldman, from Iatros Consulting, suggests that, despite the concerns about UK pharma post-Brexit, the move could actually consolidate its position as a global centre for the collection of data for RWE.

“The UK is the best territory set up for the collection of RWE. It has a number of pre-existing databases of healthcare that can yield retrospective information, for example the ‘Yellow Card’ system, and the General Practice Research Database (GPRD),” he said.

A wide range of UK and international organisations currently use or commission research from the GPRD, including the pharmaceutical industry, universities, charities, government departments and the NHS.

In addition – due to the nature of some RWE collection methods – this large amount of already-held data gives the UK an additional advantage: “Using more than one language makes for problems and it has always been the case that the ways of recording data in member states is heterogeneous,” added Dr Goldman. “I would envisage the UK becoming a power house for RWE studies, if sponsors have the appetite. Certainly RWE is far more easy to use for home markets. You would only need to deal with one language and one major platform for the delivery of healthcare.”

This mine of UK data is already being used to benefit the nation’s health. “We work closely in collaboration with the NHS, data providers – such as NHS Digital – government and academia to better understand the use of patient data, identifying how it can help improve understanding of the UK population’s health and improve access to the medicines they need,” said the ABPI’s Hanif.

“For the researchers in our companies, this data offers us three benefits; to help develop a greater understanding of a disease and its impact on a patient, identify how to improve the way patients receive their treatment, and show the effectiveness and safety of medicines over time,” he concluded.

In terms of how RWE is being utilised in the real world, Dr Goldman noted that many initiatives were trying to use real world and real time data to evaluate the management
of diabetes. “It fits very well with early access to medicines,” Dr Goldman said. “The drug regulators are interested in real world data on drug safety post-approval to evaluate the use of medicines that may have been approved on the basis of limited data.”


Getting real

One of the greatest challenges to optimising the use of RWE is acceptance – getting healthcare professionals to accept the validity of the information gathered and investigating whether it can be used for regulatory purposes.

According to Dr Goldman, one way to do this is by developing guidelines so that results are consistently credible to drug regulators and purchasers. “There needs to be more studies of different designs and more discussion with the purchasers of healthcare, advertising authorities and drug regulators.”

The ABPI, along with the European Medicines Agency and the Food and Drug Administration in the US, recognises the value of using more traditional data alongside RWE.

According to Hanif, it is also public confidence which is crucial to the future of RWE research: “None of this is possible without the support, understanding and trust of the public,” he insisted. “For the use of real world evidence to progress, patients need to have control over their data, understand its use, and be reassured that their data is protected, shared and used appropriately. Only then will patients feel confident that their health information has the power to improve healthcare and medicines for them and future generations.”


The views of Dr Martin Goldman are his own and do not necessarily reflect those of Iatros Consulting


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Modelling could set trends in drug development

by Edmundo Muniz 20. September 2016 08:41


The game changer: Modelling and simulation in drug development could change the commercial and regulatory landscape forever

It could easily be said that the current drug development process is broken. Tufts Center for the Study of Drug Development reports that the average cost to develop and gain marketing approval for a new drug is more than $2.5 billion. Furthermore, only 10% of drugs entering clinical trials achieve regulatory success. The current approach to developing new drugs, employed by most biopharmaceutical companies, clearly requires an overhaul.

Sponsors need to be able to determine a new drug candidate’s likelihood of success, at important milestones, throughout its development, so they can ‘course correct’ if necessary. They have to better understand the probability of success for everything, from proof-of-concept studies, to interactions with regulatory agencies and acceptance on formularies. By increasing those odds, using proper scientific approaches and technologies, they can intelligently inform core decisions.


Enhanced decision-making

In the quest to improve drug development’s return on investment (ROI), biopharmaceutical companies have sought methods, technologies and operational programmes to reduce costs and increase efficiency. Most of those approaches, however, have focused on improving clinical trial management, rather than systematically enhancing the research and development (R&D) decision-making process, especially in early-phase development.

Modelling and simulation (M&S) – also known as biosimulation, or ‘model-informed drug discovery and development’ (MID3) – can deliver significant business and scientific value to companies that fully integrate it into their drug development strategy. M&S combines two transformative and constantly-evolving technologies: computer-aided mathematical simulation and biological sciences. It uses pre-clinical and clinical data, combined with published industry data, to determine the relationships between drug exposure, drug response and patient outcomes.

M&S can influence every phase of the drug development process, including commercial decisions around the benefits of bringing a specific drug to market. It can also be used to compare the safety and efficacy of drug candidates; select dose and dose regimen to be used in clinical trials; and identify potential drug-drug and drug-food interactions. Knowledge of drug-drug interactions is particularly important for patients who are being treated for multiple conditions, and M&S is increasingly enabling that information to be included on drug labels.

In addition, M&S can be used to investigate how a new drug candidate will behave in patient populations which, for practical or ethical reasons, have traditionally been considered untestable. This includes paediatric patients, pregnant women and patients with rare diseases.

Certara has developed specific models of those patient populations within the Simcyp® Population-based Simulator. This simulator integrates knowledge of the physiochemistry of a drug with human biology, anatomy, physiology and genetics to simulate how that drug will behave in virtual patient populations.

Proprietary databases enable Certara to create virtual patient populations, which also consider different environmental circumstances, and adjust enzyme levels to reflect genetic differences.

The simulator combines that knowledge with pre-clinical and clinical data to predict differences in pharmacokinetic and pharmacodynamic response between individuals. Pharmacokinetics describes how a drug moves through the body during absorption, distribution, metabolism and excretion, while pharmacodynamics looks at the relationship between drug concentration and effect.


Working with global regulators

Like the tortoise and the hare, M&S has slowly and steadily impacted both the efficiency and effectiveness of drug development. It does this by informing a myriad of crucial decisions along the development path, imbuing them with more reliability and predictability.

Furthermore, M&S is now encouraged by global regulators, who have referred to M&S as ‘a regulatory necessity.’ The US Food and Drug Administration (FDA), the European Medicines Agency and the Japanese Pharmaceuticals and Medical Devices Agency (PMDA) all use the Simcyp Simulator to inform drug label language. In 2015 alone, Certara leveraged its M&S technology and expertise on nine of 21 orphan drugs and four of the 10 breakthrough drugs approved by the US FDA.

Using M&S, Certara was able to advise the drugs’ sponsors regarding trial design, helping them to develop the smallest and most targeted trials possible, and assisting them in identifying the correct drug dose to use from a safety and efficacy standpoint.

In many cases, Certara was able to eliminate the need for specific studies by simulating how particular drugs would interact in virtual patient populations. This provides more precision and ultimately reduced risk for patients. Dosing a virtual patient, after all, will always be less risky than dosing a real patient.


The results business

M&S informs biopharmaceutical companies’ most crucial drug development decisions, while optimising the data available for their crucial go/no-go drug and portfolio decisions. Ultimately, it permits a much larger number of potential drug-drug interactions to be investigated than could ever be undertaken in a clinical setting. All this additional high-quality data reduces the risks associated with bringing a new drug to market to the benefit of sponsors, payers and patients alike.


Edmundo Muniz, MD, PhD is Chief Executive Officer at Certara, a leading provider of decision support technology and consulting services for optimising drug development and improving health outcomes. Go to




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Pf Magazine: September issue out now! Read it here.

by Hazel Lodge 5. September 2016 10:46

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Precision medicine set to revolutionise outcomes

by Amy Schofield 4. September 2016 09:30

Currently, many available drugs are used to treat patients under the assumption that ‘one-size-fits-all’, but drugs don’t work the same way for every patient. The precision – or personalised – medicine approach, based on comprehensive diagnostic and clinical characterisation, ensures that the right patient gets the right treatment at the right time. 

Precision medicine involves using various technologies including genomics, diagnostics and data analytics. Diagnoses are subsequently adapted to the detailed information that is emerging about our specific genetic make-up. Pharmaceutical solutions tailored to our DNA – rather than one-size-fits-all medicines aimed at the mass-market – are the future of medical treatment, which treats the person, not the disease.

Dr Sandra Horning, Roche’s Chief Medical Officer and Head of Global Product Development, says: “The confluence of new medicines, sophisticated diagnostics and advanced technologies has created an unprecedented opportunity to improve outcomes for patients today and in the future.”


Accelerating the process

As people live longer and diagnosis and treatments improve, the prevalence of non-communicable diseases, such as cancer and diabetes, present a significant challenge as lifestyle choices act together with the genetic make-up of a patient. The need to treat the individual, rather than the group of individuals with the condition, is the catalyst for the personalised medicine approach.

Is the pace of development able to keep up with the evolving needs of the patient population?

Steven Shak, MD and chief scientific officer, Genomic Health, told Pf Magazine: “There is no question that innovations and new discoveries are being made at an accelerated pace today, compared with the past. As researchers and caregivers in the world of cancer, we know that patients have an incredible urgency for us to move even faster to make sure that we identify both drugs and tests that really work, and make sure they are used appropriately in clinical practice.”

NHS England’s National Director Sir Bruce Keogh announced the transition, to a personalised approach, in September last year, while outlining his emerging strategy for personalised medicine in the health service. He called it “the future of the NHS”.

He said that the work already being done, through the NHS’s contribution to the 100,000 Genomes Project, must be capitalised on, and fast.

“The shift to personalised medicine is already underway – our role as a system leader and commissioning organisation is to consider how this transformation can be accelerated,” he said. Embracing technology and innovation is clearly key to the success of this shift. 

An example of this comes from Genomic Health, which has developed the Oncotype DX® Breast Recurrence Score™. The therapy has now been used in over 600,000 patients around the world, to help them decide whether they need chemotherapy or not, when they’re first diagnosed with breast cancer.

“With new technologies we can now look at tens of thousands of genes and at their expression. We used a very innovative and creative approach to find and identify the genes that mattered,” said Shak. “In precision medicine it’s incredibly important that innovations are developed, used appropriately and reimbursed and paid for, so patients can benefit.”


Gene genie

Scientists cracked the human genome code thirteen years ago, resulting in the evolution of genomics. This discipline studies each individual’s complete set of DNA which, in turn, helps in the understanding of the person’s predisposition towards genetic diseases, and helps to inform the personalised treatment that is best for that patient.

Chief Scientific Officer for England, Professor Sue Hill, recently delivered an overview on how genomics and personalised medicine is set to transform the NHS. With the knowledge gained from the project, researchers are learning how inherited differences in genes affect the body’s response to medicines. These differences will then be used to predict whether a medication will be effective for an individual and help to prevent adverse drug reactions.

Shak points out, however, that innovations take time to reach those who need them: “Healthcare innovations can take 10 to 20 years to disseminate into actual practice. One of the things that’s going to be very important for bringing innovation into clinical practice in the future is to better take advantage of technology.”


Catapulting innovation

The Precision Medicine Catapult’s (PMC) aim is to make precision medicine a reality in the UK. It has six regional centres of excellence, all working on locally driven programmes, using the PMC network to harness the breadth of UK expertise, while developing innovative technologies and solutions for broader use across the UK’s healthcare sector.

It is headquartered in Cambridge, where AstraZeneca is creating a new research centre, led by Dr Menelas Pangalos, Executive Vice President of AstraZeneca’s Innovative Medicines and Early Development Biotech Unit.

Earlier this year the PMC announced a strategic partnership with the UK Pharmacogenetics and Stratified Medicine Network to drive the growth of the UK industry and accelerate the delivery of precision medicine to patients. It has over 400 individual members drawn from academia, medicine, industry, regulatory authorities and patient groups across the UK and elsewhere.

Meanwhile, pharma pipelines that include precision medicine-based targeted therapies are continuing to grow, with the added benefit of the maximisation of patient populations, for clinical trials, by the use of genomic information as an integral part of trial enrolment.

Precision medicine is gaining momentum, which has the potential to reshape how therapies are developed in the future, and industry is perfectly placed to capitalise on incorporating huge amounts of genomic and clinical data in their R&D processes, creating greater success in drug development and the understanding of disease. 





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Pf Magazine: August issue out now! Read it here.

by Hazel Lodge 14. August 2016 08:22





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Dangerous liaisons: GPs are failing transgender patients

by Admin 8. August 2016 09:41


GPs are failing transgender patients with a lack of compassion and understanding that must change, according to Dr Helen Webberley

The recent massacre in Orlando served as a grim reminder that discrimination against minority groups continues to play a very real part in our so-called ‘modern society’.

Of course, these episodes are extreme, but while this degree of hatred is mercifully rare, members of the LGBT community have come to accept prejudice as an everyday occurrence. Nowhere is that more true than if you are a trans man or a trans woman.

Every day I hear stories through my online clinic – GenderGP – from members of the trans community, about the battles of bigotry, prejudice and humiliation they face. But what shocks me most, is when that sentiment comes from members of the health profession.

Following significant concerns raised by the House of Commons Women and Equalities Committee in their recent report on Transgender Equality – specifically in relation to the lack of awareness and consideration shown by a great many doctors in treating transgender patients – I was delighted to see the General Medical Council (GMC) publish guidelines on managing transgender patients.

The guidelines state that GPs can prescribe hormones to patients suffering with gender identity disorder, under the following ‘exceptional circumstances’:



  • The patient is self-prescribing with hormones from an unregulated source 
  • The bridging prescriptions are intended to mitigate risk of self-harm or suicide
  • The GP has sought the advice of a gender specialist and prescribed the lowest acceptable dose



This was met with a variety of concerns from NHS GPs, and I am disappointed at the reluctance to openly embrace the news that we can now do more to help our trans patients.

Dr Chaand Nagpaul, CBE Chair of the BMA General Practitioners Committee, penned his concerns to the GMC, raising a number of emotive points. His main uneasiness being that specialist prescribing would place GPs in a difficult position, forcing them to prescribe outside the limits of their competence.

In response, Susan Goldsmith, acting chief executive of the GMC, provided reassurances, which included the point that GPs would be expected to ‘acquire the knowledge and skills to be able to deliver a good service to their patient population’. This, she explained, may mean undertaking training. It was clear that the GMC does not consider care for patients with gender dysphoria as a highly specialised treatment area requiring specific expertise.

Goldsmith goes on to endorse a firm view of mine: that these patients actually require very simple care and well-known medication.

The medication for transgender care includes oestrogen therapy used for treating female menopause (estradiol), injections that are typically given to women with endometriosis or men with prostate cancer (GNRH analogues) and a diuretic used for heart failure (spironolactone). Furthermore, anti-androgens used in contraceptives (cyproterone acetate), medication for benign prostate hyperplasia (finasteride) and testosterone replacement therapy, generally used for the management of the male menopause, can also be prescribed.

These are medications that are well known to GPs, and we are well versed in their potential side effects and effective monitoring.

So why is it that GPs are reluctant to take this on? There have been some well-publicised legal cases of doctors trying to help transgender patients – Dr Russell Reid was criticised for a lack of caution in initiating hormonal and surgical gender reassignment treatment, without more careful and thorough investigation and assessment. Meanwhile, his successor, Dr Richard Curtis, was also subjected to a lengthy investigation.

Fear of litigation is at the forefront of any doctors’ mind, but should this impair our duty to help our patients in the best way we can? GPs prescribe hormone patches for menopausal symptoms regularly, but if a trans man asks you to do the same, the response he experiences is often one of horror.

Not all trans people want to go through lengthy assessments, procedures, hoops and surgery – they just want the right hormones to suit their true gender.

These are actually very safe treatments, and delays in receiving care from GPs and NHS gender clinics mean that people are turning to illegal sources and unsafe forms of medication. In the very worst cases, death appears to be the only answer.

The other essential aspect of transgender care is listening, hearing, caring, educating, protecting – bread and butter to GPs. Often the key healthcare provider for the transgender patient, however, is an endocrinologist. Experts in their field yes, but have they honed their patient skills to the same extent as a GP? I would argue not.

So many of my patients tell me about the long journey they have to take, before even feeling brave enough to confide with their GP that they are suffering from gender variance, only to have their feelings dismissed or told that they will ‘grow out of it.’ 


Carry On Doctor


I have the following simple advice for GPs, which might just help any transgender patients they may come into contact with.


  • If you don’t know, look it up. There are many training resources and literature sources on gender care. Go to
  • Listen to your patients, they are not mentally ill, they are gender incongruent
  • Their medical needs are often very simple – some hormone replacement therapy and a listening ear
  • The treatments are those that we use every day – put any prejudice aside and, if you think your patient is suffering from gender variance, get your prescription pad out
  • The cost of treating these patients is far less than the loss of life and distress caused by refusing them very simple, basic care



Dr Helen Webberley MBChB MRCGP MFSRH is a GP specialising in the treatment of transgender patients on the NHS and via her private online clinic. Go to



An online petition calling for a reduction in waiting times for appointments at Gender Identity Clinics currently stands at over 5000 signatures. Add your signature to the petition.

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