Riddling Alzheimer’s disease

Alzheimer ’s disease (AD) is a growing threat to the world’s ageing population and is a topic of fascination for the mass media and public alike – whether in the press (The Daily Mail 23 Jan), TV shows (Greys Anatomy) or film (The Iron Lady). And the scientific community is undoubtedly racking its collective brain to find potential treatments with clinical.trials.gov listing over 1000 registered clinical trials in AD as of January 2012. 

The need for new treatments is certainly present:  in 2010 there were an estimated 35.6 million people with dementia worldwide. Alzheimer’sDisease International estimate that by 2050 this will increase to over 115 million people.  According to the Alzheimer’s Study Group report, of these patients, 16 million could be in the United States, costing the government healthcare system over one trillion dollars if no effective treatments are found.  

Yet, despite the considerable efforts, effective treatments remain elusive. Just last week, Pfizer/Medivation ended the development of a potential candidate following another failed Alzheimer trial (see Health News Daily). And it won’t be the last.

Scrip suggests that there is a pending crisis, with investors shying away from Alzheimer’s due to the many unanswered questions around the disease: how do we diagnose patients accurately? Can we change the disease progression? What is the potential for reimbursement?

Nevertheless, small victories are gradually being achieved. BioCentury reports today that CHMP has adopted a final qualification opinion for a new biomarker allowing its use in patient selection for clinical trials.

No need to despair yet then. And as the press reports today: reading, writing and puzzle-solving can help stave off the disease. Let’s hope the academic community can follow these steps to unravel the riddle that is Alzheimer’s disease!

Authored by Juliet Burns

Rare Treatments for Rare Diseases

Rare diseases, such as unusual hereditary conditions and some less common forms of cancer, affect many more people than most of us would imagine. In the EU, diseases are defined as rare if they afflict no more than five in 10,000 people. But put that in the context of more than 6,000 rare conditions so far identified and the total numbers affected - including the families of those that suffer from them - and we are well into the millions in the UK alone.

But, despite the millions of dollars spent worldwide on developing new therapeutic drugs, the treatments approved to tackle these rare conditions remain pathetically few. The biggest obstacle to improvement is money. Most drugs are licensed for use in conditions where the drug manufacturers are both able to recruit enough patients into clinical trials and where the products will yield enough sales to justify running those trials in the first place. So, people with rare diseases find their access to effective medication either limited or non-existent.

The first genetic condition ever identified, Alkaptonuria (or AKU) is a case in point. It's over a hundred years since AKU, more commonly known as black bone disease - for the discolouration and damage it causes to the bones and cartilage - was identified. Yet there has never been an effective treatment for it and people with AKU, who see the symptoms appear typically in their 20s and 30s, suffer chronic health problems that often lead to destruction of the cartilage and early joint replacement surgery.

A relatively small number of people with AKU have been identified in the UK (around 70) but experts, who describe the disease as a severe form of arthritis, to which it's closely linked, believe there's a much larger number who are living with AKU undiagnosed. 

This month, the AKU Society - a small UK patient charity supporting those with the condition - launched a campaign to raise funds for a late-stage clinical trial of a drug that does promise a treatment for AKU. It could in turn give hope to some of the eight million in Britain who suffer from osteoarthritis, to which AKU is closely linked.

Dr Lakshminarayan Ranganath, co-founder of the AKU Society and one of the country's leading experts on the condition, says "Anything we can do to understand AKU will undoubtedly help us to understand other forms of arthritis."

The drug nitisinone, which is already marketed for another hereditary condition has been shown in previous clinical trials to relieve AKU patients' symptoms but because of the estimated £5m costs of mounting a late-stage clinical trial and the relatively small patient population, no organisation has yet put up the funding.

Taking the drug over this final development hurdle would create the first ever treatment for AKU, as well as a better understanding of osteoarthritis and its potential treatment - a hugely significant benefit in an area of unmet medical need. Some well-funded organisation, ideally a pharmaceutical company, should put the money up for the trial. Research into rare conditions such as this one really shouldn't be left to charity.

Authored by Peter Coe

Recently published in The Huffington Post UK

AstraZeneca paves the way for UK research

It appears that AstraZeneca was well ahead of David Cameron on the day he announced his Life Science initiative.

With the unveiling of his Strategy for UK Life Sciences, the British prime minister argued the need for new models of working between universities, hospitals and pharmaceutical companies. This, he claimed “would place the UK at the forefront of medical research now and in the future.”

Turn the page of your newspaper that day and you would have found that AstraZeneca are already beginning to do just that. As reported in The Daily Telegraph and the Financial Times, in a revolutionary move that could see the development of several new treatments, AstraZeneca has opened its doors to allow full and free access for UK researchers to more than 20 of its experimental drugs.

New research findings will be owned by the relevant academic institutions, with AstraZeneca retaining the rights to the chemical composition of the compounds. The company has stopped developing all of these compounds, many rejected as the market was too small or because the treatments fell outside AZ’s areas of interest.

It’s a radical move and a pioneering effort to boost medical advancement. The AstraZeneca initiative will allow researchers to examine compounds – many still on patent – and could broaden the potential of drugs that would otherwise have been neglected.

The chance to be more open and co-operative is a growing trend and clearly supported by David Cameron’s incentives. Sir John Savill, MRC chief executive said it is ‘win, win, win’ where the connections between AstraZeneca, academics and patients will be firmly fused.

Authored by Lisa Stevens

Swap pills for health solutions

A bill so big you can see it from space. Bigger than the decades-old bill that created the system the current bill is intended to replace. Two descriptions of the Health and Social Care Bill, a controversial set of proposals designed to overhaul the UK’s unwieldy and overly-bureaucratic NHS. After a rocky few months, the bill is at committee review stage in the House of Lords. Reformers hope it will reach royal assent in spring 2012 and become law soon after. This raises two questions. Once all the necessary amendments are made, what bill can we expect to emerge in a few months’ time? And what can the pharmaceutical industry do to prepare for it?

Companies can do little to influence the answer to the first question; the passage of the reforms is now in the hands of the peers. But the recent Wellards Annual Conference, held in London on 9 November, showed that there are plenty of experts willing to have a stab at answering the second. A day of presentations and discussions, aimed primarily at pharmaceutical marketing and other customer-facing professionals, underlined two new strategic priorities: the need for proactive engagement with an enlarged group of stakeholders and a shift from selling drugs to providing broad healthcare solutions.

The first priority is to recognise the stakeholders. The few constants in the new system will be patients, health providers and the Department of Health; virtually every other component of the UK healthcare landscape will change. Some 250 clinical commissioning groups (CCGs), member organisations led by GPs, will take over work currently carried out by primary care trusts and regional strategic health authorities. Monitor, the independent body that regulates NHS foundation trusts, will become an economic regulator responsible for overseeing access and competition in the NHS. Health and wellbeing boards (HWBs) will co-ordinate and integrate services and develop strategic-needs assessments. And there will be a shift from centralisation to localism. Come 2013, local governments will take on a long list of public health responsibilities, including dealing with key disease areas such as mental health.

Some companies have seen these changes coming and been quick to adapt. David Rogers, chair of the Local Government Association Community Wellbeing Programme Board, said he had been approached by firms asking how best to work with local government. A recent pharma roundtable discussion, organised by the HFMA, an association representing NHS accountants and finance directors, showed that drugs companies are willing to share ideas with influential new stakeholder groups. A few years back, pharmaceutical sales and marketing teams were already talking about key account management as an effective way of working with NICE and other payer groups. It would not be much of a leap to do the same in their interactions with local governments and HWBs.

The second strategy, rethinking traditional sales approaches, may have more of an impact. Senior NHS managers are tired of seeing sales reps trying to sell pills and fancy gadgets. They are frustrated that a health service that claims to be primary care-led ends up dealing with so many patients admitted to hospitals as emergencies. They are crying out for solutions so that patients stop using A&E departments as their first port of call. How to achieve this? NHS officials argue for the creation of holistic disease-management pathways, integrating diagnostics that can be used at home, the most appropriate pharmaceuticals, self-monitoring and management by patients, and better use of smart telemedicine tools.

These ideas are gaining currency among policymakers the world over. Barack Obama’s health reforms in the US have placed a premium on the ability to deliver cost-effective health solutions across the entire treatment spectrum. In its two most recent Progressions thought-leadership reports, Ernst & Young has promoted guiding principles that it calls “Pharma 3.0”. These include the need for pharma companies to focus on health outcomes, for example by forming non-traditional partnerships with firms outside the life sciences space.

Delegates at the Wellards conference also heard some frank admissions about where the NHS needs to change. One area is cultural: the rules of the NHS have created a culture of risk-aversion rather than risk-taking. This will hold back a system that desperately needs to get into better shape. Jim Easton, national director for improvement and efficiency at the Department of Health, admitted the NHS is often scared to back new ideas and timid when it comes to disseminating or adopting innovation.

Other reforms will be structural. Senior NHS officials are already speaking the corporate lingo of the pharmaceutical CEO. Dr Gordon Coutts, chief executive of Colchester NHS Foundation Trust Hospital (himself a former Lilly and Schering-Plough man), talked about weighing up market penetration versus market expansion, competition or collaboration, and vertical or horizontal integration. Dr Coutts is on a quest for efficiency that will lead to a shortening of patients’ length of stay in hospital. He also wants to implement just-in-time principles (used to superb effect in the car-making industry) in his hospital’s supply chain.

Under intense pressure to adapt, most big pharma companies have spent the past few years re-engineering their organisational structures. They could be a fount of knowledge for an NHS undergoing upheaval. In addition, the drugs industry is shy of neither risk nor failure; the two have always been integral to the pharmaceutical R&D model.

Conference chair Alan Jones was probably only half joking when he told a room full of pharmaceutical professionals: “The NHS is full of people who don’t like you.” If companies were to share with NHS managers their expertise in re-engineering large bureaucratic organisations and innovation through smart risk-taking, they could go a long way towards overturning such ingrained negative attitudes.

Authored by Pete Chan