Testing times for clinical trials - Perscetives on personalised health care

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Pesrpectives on how clinical trials framework is being modernised (WPF 2012/2/Sep/2012)

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Testing times for clinical trials - Perscetives on personalised health care

  1. 1. Insight > Clinical trials Testing times for clinical trials Clinical trials face more hurdles than ever – not least protracted timelines and soaring costs But with study design constrained by outdated regulations, rising to these challenges is proving difficult. Abi Millar talks to the FDA and Mats Sundgren, principal scientist in clinical development for AstraZeneca, to discover how the clinical trials framework is being modernised. 86 WorldPharmaceuticalFrontiers | www.worldpharmaceuticals.netWPF022_017_Clinical Trials.indd 86 25/09/2012 12:40
  2. 2. Insight > Clinical trials I n April 2012, the FDA held a public consultation, ‘Modernising the regulation of clinical trials and Mats Sundgren Mats Sundgren is a principal scientist in global approaches to good clinical practice’. With its broad medicine development, biometrics and information range of speakers, drawn from the full spectrum of sciences, AstraZeneca R&D. He has over 26 years’ experience in the pharmaceutical industry. stakeholders, the hearing provided rich grounds for debate. All had their own ideas about how the clinical trials framework could best be overhauled. One aspect, however, was unequivocal: with current regulations Case study: clinical trial of the future dating back to the 1970s, there is little case for maintaining the The goal of this trial is to develop a set diagnostic tool and status quo. Tailored towards a hugely different environment, biomarker for diabetes. This involves a large amount of data – internal and external. existing strictures are poorly suited to trials in 2012. While the project is primarily driven by a pharmaceutical “There have been dramatic changes in the clinical trials company, the R&D stage takes place as a collaborative enterprise,” says the FDA’s Michelle Bolek. “These include venture with the healthcare industry. During this phase, the increased size and complexity of trials, rises in the number tool is supported by sophisticated reuse and data mining of health information, including electronic health records (EHRs). of trials performed globally, greater use of CROs, participation Its success in the phase I and II trials depends on the of vulnerable populations, and numerous scientific and outcomes of a finely tuned group of patients, who, in turn, technological advances.” have been selected through EHRs. These studies are carried Faced with this cultural sea change, the industry cannot be out using devices and sensors, handled by the patients themselves, which monitor glucose levels in real time. An content with minor tweaks. With costs continuing to rise – interactive patient evaluation, the studies provide far more average expenditure has tripled over the past 12 years – information than is usually captured in clinical trials. healthcare systems running trials are creaking under the strain. Furthermore, outmoded policies make it hard to integrate new technologies. It is clear that purging the inefficiencies from the will help identify issues that may inform additional system will amount to all-out reform. harmonisation efforts.” For now, though, the challenges posed by drug development Personalised healthcare is the show few signs of abating. As such problems become entrenched, bodies outside the FDA are urgently seeking solutions of their own. approach now taken by the industry. We are no longer trying to produce Narrow the field drug products that treat the majority For Dr Mats Sundgren of AstraZeneca, the situation requires radical thinking. Principal scientist in clinical development, he of the population. is also one of 33 partners of the Innovative Medicines Initiative’s Electronic Health Records for Clinical Research This, of course, is not the first time these issues have been (EHR4CR) project, and the coordinator on behalf of raised. The Critical Path Initiative, aimed at improving clinical AstraZeneca. A four-year undertaking with EU backing, the trial practice and policy, was introduced as early as 2004. project endorses a full-scale paradigm shift. Similarly, the Clinical Trials Transformation Initiative (2007) came “The pharma industry has been unable to develop innovative about in response to sky-high prices and stifled innovation. products for the last two decades,” explains Sundgren. “The Unfortunately, such initiatives have come under flak for not attrition rate is a big problem, with only 2–5% of projects being transformational enough. Speaking at the FDA hearing, actually hitting the market. So there’s huge pressure on the Doug Peddicord from the Association of Clinical Research industry, and that’s reflected in clinical trials.” Organisations alleged that their “research on research” Sundgren is emphatic that the next few years will see approach was unlikely to “facilitate any significant change to important changes in the healthcare industry. As older current practices, let alone transformation of the enterprise”. drugs lose their patents and advances in genomics make He went on to lay out suggestions for how the FDA might themselves felt, healthcare is moving into an area of progress. The Clinical Trials Transformation Initiative, he said, personalised medication. should perform comparator studies that pit old approaches “Personalised healthcare is the approach now taken by the against new. Their goal should be to demonstrate “actual industry,” he says. “We are no longer trying to produce drug savings in development time and cost for a given product”. products that treat the majority of the population of the planet. And patient needs should always be given precedence This means we need to target and understand the right over the whims of corporations or institutions. Since the patients. To test if our inclusion/exclusion criteria make sense, hearing took place, the FDA has been busy assessing the we need to have much more external health information and points that were raised. patient data.” “We will consider the suggestions received at the April The trend is towards a culture in which each disease- hearing, as well as our stakeholders’ suggestions for management model is adapted to a patient’s genetic profile. improving the regulations,” says Bolek. “Input from the public Often described as ‘translating bench science to bedside WorldPharmaceuticalFrontiers | www.worldpharmaceuticals.net 87WPF022_017_Clinical Trials.indd 87 25/09/2012 12:41
  3. 3. Insight > Clinical trials clinical practice’, this movement into personalised healthcare way,” he says. “This is a very sensitive area in which all holds great promise for treatment and diagnostics; however, stakeholders must be transparent. We also need to find a the industry will need to push beyond its limitations before any sustainable and scalable solution. Pharma companies should significant changes can take place. be able to choose the hospitals that are preferred for a certain Under the present framework, personalised medicine is drug or patient category, and one of the levers will be platforms difficult to accommodate in clinical trials, and patient that allow virtual integration of data from different sources.” recruitment is a case in point. Even with present levels of Looking back several decades, there is an analogous selectivity, late-stage clinical trials can take up to a year to situation within the banking sector. Faced with their own locate their subjects. Narrowing down the criteria to certain dilemma – how best to leverage information technology to genotypes is hardly poised to simplify matters. make trustworthy financial transactions – banks worked This issue is one of the key dilemmas being addressed by together to create the standardised messaging network SWIFT. EHR4CR. In essence, the project’s aim is to find ways of While this analogy is imprecise, it usefully illustrates how reusing existing electronic health records (EHRs) of hospital competitors can join forces. patients in clinical research. A rich seam of data, previously In these financially beleaguered times, the real question to untapped by pharma companies, EHRs could greatly accelerate ask is whether pharma companies can afford not to collaborate. patient recruitment and shrink the development timeline for The EHR4CR project, after all, does not just stand to benefit new medicines. patient recruitment. It is relevant to protocol feasibility, clinical “We have identified the top-ranking services that the trial execution and drug surveillance reporting. All these areas pharmaceutical industry is asking for in order to enhance the will reap rewards from a more data-intensive and efficiency of clinical trials, and one of these is finding the right multidisciplinary approach. patients,” says Sundgren. “We are aiming to develop a new “Over the next ten years, the industry will conduct smaller business model in order to ensure we can reuse electronic but more data-rich trials, with exactly the right patients,” says health records to support clinical trials.” Sundgren. “Clinical studies will be conducted in closer collaboration with hospitals, in which observational studies The attrition rate is a big problem, and data from EHRs inform the pre-design of studies and the with only 2–5% of projects actually way they’re conducted. I also foresee that they will involve considerably fewer patients, which will help address the hitting the market. So there’s huge extremely high economic price that studies face today.” pressure on the industry, and that’s This emphasis on data is not confined to static hospital reflected in clinical trials. records. It also extends to real-time patient monitoring, using sensors connected to mobile devices. This should provide a much clearer tool for understanding how the drug is working The advantages are obvious. With more information required and help steer its passage to the clinic. than before, sourcing such data in isolation is likely to prove “Everyone has to win in this situation,” says Sundgren. time-consuming and costly. Reusing EHRs represents nothing “We can probably improve the attrition rate and heighten less than a breakthrough opportunity for the industry. our success in taking drugs to market, although what will Of course, this undertaking is not without its pitfalls, legally, change is that each drug will have a very limited and ethically and technically. For the pharma industry to connect specified indication.” its internal health information domain with external records, it The EHR4CR project still has over two years left to run. As it will need to take an unprecedentedly collaborative approach. edges closer to developing a robust and scalable business “We need to connect more closely to healthcare, not only model, its points of focus are indicative of a broader shift because healthcare institutions are the original owners of within the industry. No longer are pharma companies content information sources like EHRs, but also because developing to stick within outdated frameworks. They are united in their new biomarkers will bring us into close collaboration with search for building blocks that will help a drug move more many partners simultaneously,” says Sundgren. “There is an effectively towards its launch. opportunity for pharma companies to work not just with Encouragingly, top-down change is happening in tandem hospitals but also with other pharma companies in with bottom-up. If the FDA has been slow to evolve, a more precompetitive arenas.” welcoming approach to innovation is on the cards. “We are evaluating our regulatory approach to clinical trial Collaborative incentive oversight to ensure it meets our objectives without being Sundgren does not believe that pooling information will unnecessarily burdensome or unduly impeding implantation of come about through sheer goodwill. Rather, those involved innovative approaches,” says Bolek. will be offered a financial incentive, along with an element As these approaches take shape, the stage is set for a new of accreditation. type of clinical trial – one in which the design is better suited to “The new business models will need to govern and regulate a field undergoing constant shake-ups. For the industry, this is a the financial implications of reusing data in a trustworthy cost and time-saver. For patients, it’s a genuine revolution. ■ 88 WorldPharmaceuticalFrontiers | www.worldpharmaceuticals.netWPF022_017_Clinical Trials.indd 88 25/09/2012 12:41

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