2013 11 ebsin spez clinicaltrials

European Biotechnology News Science & Industry

November 2013

II Clinical Trials

SPECIAL 31_EBN11_13_SPECIAL_Titel_tg.indd 27

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TE SAVE THE DA

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For further information please visit www.swissbiotechassociation.ch www.swissbiotech.org 01.11.2013 13:12:32 Uhr

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Clinical Trials Intro

According to GBI Research, the global R&D outsourcing market in 2010 represented 25% of total pharmaceutical R&D expenditure, and is predicted to reach 37% by 2018. The CRO industry brought in US$33.6bn of revenue in 2012, and is expected to hit over US$37bn this year.

Approximately two-thirds of that revenue was paid by pharmaceutical developers, while 27% came in from biotechs, medical device manufacturers, foundations and governments.

Growth in an era of consolidation As bio-pharma companies seek to maximise income from core assets, their strategic planning increasingly includes outsourcing to Contract Research Organisations (CROs) as a means of controlling non-core expenditures and complementing internal resources. Portfolio pressures have peaked with major patent expirations, and while industry-wide R&D spending has more than doubled to roughly US$50bn a year over the past decade, US and EU regulatory agencies have approved fewer new drugs (Fig. 1). Partnerships between Big Pharma or biotechs and CROs have been shown to strengthen research capabilities, and they can also offer efficiencies through both reduced development timelines and lower cost. According to the Tufts Center for the Study of Drug Development, clinical trials conducted by CROs are completed an average 30% faster than those conducted in-house at a drug developer. In real terms, that translates into time savings of some four to five months – and can increase revenue potential by US$120m to US$150m.

German-based Bayer Healthcare’s approach is an example of the direction many Big Pharma players are taking. It has outsourced all of its late stage trials (Phase IIIV) to Covance, a top-ten CRO that began operations in the mid-1980s. Of the around 1,200 CROs worldwide, the industry leaders (Quintiles, Parexel, Covance, Pharma­

ceutical Product Development (PPD), Icon, INC Research, and InVentiv Health Clinical) took in more than 60% of the global revenues for outsourced clinical research and drug development. Those CRO majors continue to grow, while smaller competitors are increasingly niche players in the expanding biopharmaceutical market.

53 New Molecular Entities (NME)

US$17bn

25

R&D Spending

2009

Fig. 1: Development of pooled R&D costs in the pharma sector and market approval of new molecular entities.

33-34_EBSIN11_13_Spezial-Intro.indd 33

© Roche

1996

US$47bn

Opportunities for smaller CROs While big CRO players are driving market consolidation by offering faster recruitment for late-stage trials through global alliances with leading hospital sites, smaller entities are seeing new market opportunities in early clinical testing. “In the past, there was a trend to outsource the entire clinical development to globally­ acting CROs,” says David Surjo, Vice-President Business Development at German CRS Clinical Research Services Andernach GmbH. “Now there‘s more of a trend to look for highly-specialised partners for early phases.” In late June, Bayer AG signed a pioneering partnership agreement with the specialist company for complex earlystage (Phase I-IIa) trials and exotic patient populations. The drug giant will transfer its two clinical pharmacology units in Berlin and Wuppertal to CRS and integrate them into the CRO‘s network of study centres, outsourcing all of its early cardiovascular and gynecology testing.

The market for early-stage clinical study services, however, is yet smaller than that for late-stage testing. According to an anonymous survey conducted among safety pharmacology representatives from the top 12 pharma companies, a majority of pharma firms still outsource less than 25% of their safety studies, with in-vitro profiling most commonly transferred to CROs. “Phase III trials continue to be an area of significant opportunity for larger CROs,” says Jamie Macdonald, COO at INC Research. “We find that we are also engaging with more customers for Phase I activities, as the outcomes and decisions from early development activity are becoming

31.10.2013 13:04:07 Uhr

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CLINICAL TRIALS increasingly critical for the remaining development stages.” Market leader Quintiles has already recognised this growth opportunity (see interview, p. 36). A heavy war-chest could give the CRO a leg-up in the global race. In May, Quintiles raised US$947.4m in the third largest IPO this year to date.

Current trends

55% Clinical trials are evenly split (50/50) between mature markets and emerging markets

14% Slightly more of our clinical trials (50–60%) are conducted in mature markets than in emerging markets

12% Slightly more of our clinical trials are conducted in emerging markets than mature markets

12% A substantial portion (60% or more) of our clinical trials are conducted in emerging markets

7%

© Wipro/2UBM TechWeb

Fig. 2: Split between clinical trials conducted in the US and western Europe, as compared to emerging markets* * Data: 116 biopharma professionals who say their companies have begun or plan to begin conducting clinical trials in emerging markets

and Scientific Officer for Quintiles (see p.

36). Additionally, sponsors are promoting adaptive trial design to speed up the drug development process. Overall, new dataintensive technological approaches and the globalisation of clinical trials is driving the need for electronic data capture and interpretation.

The Association of Clinical Research Organisations (ACRO) reports that approximately 46% of the clinical trials listed at clinicaltrials.gov are conducted in the US, 30% in Europe and the remainder in Asia, Latin America, Africa and the Middle East.

A recent analysis found that there were no statistically significant differences in data quality between or among regions or countries (DRUG INFORMATION JOURNAl, 46(4), 455-

Source: CenterWatch 2013 Global Survey of 2,032 Investigative Sites

While the biologics market continues to grow, more drug sponsors are now also requesting biosimilar comparability testing services, which is currently still limited to specialised companies with particular expertise in protein analytics. Evaluate Pharma predicts that nearly half of the top 100 drugs by 2016 could be biologics, with biosimilars making up a big piece of that cake.

Another trend CROs will have to come to terms with is precision medicine. Regulatory authorities and payors are increasingly requiring drug sponsors to identify non-responders via biomarkers, and CROs are expanding technical capabilities to meet those demands. “We are enhancing technical capabilities that allow us to control important technical aspects required for precision-medicine – genomic sequencing, biomarker validation and bioinformatics – as well as the knowledgebased expertise that is essential to appropriately utilise these technologies,“ explains Jeffrey Spaeder, Chief Medical

A substantial portion (60% or more) of our clinical trials are conducted in mature markets (US/Western EMEA)

Fig. 3: Average CRO ratings by region

33-34_EBSIN11_13_Spezial-Intro.indd 34

463). As the Asia/Pacific region as a whole and China in particular is expected to grow into the world‘s largest pharmaceuticals market within the next decade, sponsors have already begun to carry out clinical trials there. According to a survey conducted

by UBM TechWeb and E-clinical solutions provider Wipro Technologies, around three in five biopharma professionals said their firms had begun conducting clinical trials in emerging markets as of 2011. The remainder said their companies had plans to do so in the “next 12-24 months”. While 46% saw Asia as the most attractive region, 37% indicated they will start enrollment in Eastern Europe, followed by 36% who say they will do so in South America.

Capacities are therefore rising in the Asia Pacific area, but also in Russia, Brazil, Africa and the Middle East, which offer high recruitment rates and market potential.

Globalisation & eClinical progress According to ACRO, development is being driven by the ability to access larger patient pools for participation in clinical trials, which significantly speeds the development process – sometimes cutting the cycle in half. According to clinical trials information provider CenterWatch, CRO ratings do not significantly differ between traditional and growth markets (see Fig. 3).

With clinical trials now crisscrossing not just one country, but the entire globe – including far-off sites in emerging markets – and technological advances such as sequencing, electronic data capture, storage and analysis is growing in importance. “Operational approaches enabled by eClinical technology can be highly adaptive to the treatment effect or safety signals measured during the course of a trial. This enablement is only possible through process automation and access to real-time information that allows for data-driven decisionmaking,” says PPD‘s chief information officer Mike Wilkinson.

The latest trend to digitalise the patient promises to phase out on-site visits, and speed up data interpretation and clinical trials as a whole. 

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Clinical Trials Interview

Front-runner insights While smaller Contract Research Organisations (CROs) are beginning to feel the winds of market consolidation as customers focus intensely on cost efficiency, market leaders in the sector are gearing up for more growth. EuroBiotechNews spoke with experts from global market leader Quintiles about trends in contract research.

Euro|BioTech|News

?

The CRO market is undergoing consolidation, but it‘s also predicted to grow. What do you see as the main drivers of that transformation, and what are the most interesting opportunities for expanding Quintiles‘ business?

!

Paula Brown Stafford: Increased outsourcing is one of the main drivers impacting the CRO market transformation. Biopharmaceutical companies will require service providers to meet the demands of increasingly complex clinical trials, as well as the need for specific therapeutic and scientific expertise, as they in-license drugs outside of their core areas of expertise. We estimate that clinical develop­ment spending outsourced to CROs will grow annually by 5%-8%. As sponsors develop more personalised medicines, they will partner with service providers like Quintiles to leverage our deep and broad therapeutic and scientific expertise, along with our data analytics platforms, which provide more sophisticated approaches to managing trial complexity. Evolving technologies and approaches to developing medicine – genomics, bio­ markers, bio­logics, biosimilars, personalised medicine – are advancing how medicines are discovered, developed and delivered, and will continue to impact research and development in a big way. We view these as areas of great opportunity.

Euro|BioTech|News

?

Quintiles‘ current focus is on late-stage clinical development. How will the era of biomarker-based precision medicine influence the firm‘s current business strategy?

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!

Jeff Spaeder: Quintiles is a pharmaceutical services company that works with customers to improve the probability of success of development for new therapies that improve health, as well as other ways to improve healthcare. Late-phase clinical development is certainly an important component of this mission, but so too is our expertise in conducting observational studies, our ability to engage with patients, our commitment to assisting customers with data-driven planning and design c ap abil itie s , and our long his tor y of conducting early-phase development.

We have been anticipating the shift to a more precision-based paradigm for developing new therapies due to the rationale of such therapies and to our active engagement with our vast and increasingly diverse set of customers. In response to these insights and observations, we have been prudently enhancing technical capabilities that allow us to control important technical aspects required for precision medicine – genomic sequencing, biomarker validation and bioinformatics, as well as the knowledge-based expertise that is essential to appropriately utilise these technologies. Concurrently we have enhanced our ability to identify patients for more targeted therapies, and to conduct these complex studies more efficiently.

By combining scientific platforms, subject-level expertise and the enhanced operational delivery needed to conduct studies of precision therapies, we take a holistic approach to improve the probability of success for our customers.

Paula Brown Stafford, President of Clinical Development at Quintiles.

Jeff Spaeder, Chief Medical and Scientific Officer at Quintiles.

Steve McPhail, President and CEO of Expression Analysis, a Quintiles company.

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Clinical Trials ?

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How will strategic projects like genetic pre-profiling and acquisitions such as that of Novella contribute to improved patient recruitment?

!

Paula Brown Stafford: With our strategic projects and acquisitions, our primary goal is to create a streamlined and more efficient approach to developing new therapies. Quintiles has many different kinds of customers, ranging in both size and therapeutic breadth, and we know that sometimes being the best partner means engaging with a more specialised customer focus. That‘s why we offer different models to adapt to each customer‘s specific needs. One way that we have bolstered support for our smaller customers is with our recent acquisition of Novella Clinical, a full-service clinical research organisation focused on serving emerging oncology customers as well as medical device and diagnostics com-

panies. Novella will service these customers by giving them the attention and focus that will help them succeed. As we expand and customise our offerings, we are not only better able to meet customer needs, but – more importantly – we can better meet the needs of patients. For example, our recent collaboration with US Oncology to undertake a novel genomic “pre-profiling” feasibility study is showing some early progress in helping to identify higher-risk meta­static colorectal cancer patients, and also to aid in selecting better treatment options.

?

Euro|BioTech|News

How is the CRO market preparing for the implementation of new (genomic) technologies such as next-generation sequencing?

!

Steve Mc Phail: Quintiles recognizes the importance of leveraging the power of genomics, and

has taken steps to advance the company‘s capabilities and expertise. In 2012, Quintiles acquired Expression Analysis (EA), a premier provider of genomics testing and analysis. The acquisition is another step forward in our efforts to improve drug development and deliver greater value through the use of new diagnostic tools that have the potential to deliver safer, more effective therapies based on the genetic makeup of the disease and the patient. EA‘s depth of expertise in DNA sequencing and advanced bioinformatics is essential to understanding diseases and drugs at the molecular level. Quintiles‘ strategic approach is to use genomic data and advanced informatics, in conjunction with deep biological expertise in a variety of therapeutic areas, to yield actionable insights leading to more effective personalised treatments. Quintiles, through EA, is currently implementing DNA sequencing and other genomic services in a variety of ongoing clinical trials.

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CLINICAL TRIALS CONTRACT RESEARCH

Conducting clinical studies in Russia

By A. Globenko, A. Shigabutdinov, V. Arnautov, E. Moskaleva, G. Shukshina, NADIM LLC, Moscow, Russia

This report addresses the key issues involved in organising and conducting clinical studies in Russia. It references all of the major regulatory documents, and provides an overview of statistical data from 2012 results.

Amidst the favourable economic and political climate established in Russia over the last few years, the key infrastructure elements that are required for successfully conducting and administering clinical studies have been bolstered. The other factor that has facilitated attractiveness and increased numbers of clinical studies in the country is a well-developed legal base now supported by a federal law – “On the circulation of drugs” (No. 61-FZ, dated 12 April 2010). A large number of investigators with broad experience in clinical studies and a proven system for the administration of clinical centres is now steadily increasing Russia’s active involvement in the fast-growing, competitive field of drug development, and the country has grown increasingly attractive to multinational teams of scientists engaged in clinical studies.1 Clinical studies in Russia are regulated by the country’s Min-

istry of Healthcare – particularly by its Department for State Regulation of Drugs Circulation – and two expert organisations: the federally-financed “Scientific Center for the Evaluation of Medical Products” (FGBU NTsESMP) and the Council for Ethics.2

Russian legislation The primary regulatory documents governing how clinical studies are conducted in Russia are: – The Constitution of the Russian Federation14; – Federal Law No. 61-FZ “On the circulation of drugs” dated 12 April 2010 (Articles 3844) 3; – Federal Law No. 323-FZ “On the Fundamental Healthcare Principles in the Russian Federation” dated 21 November 2011 (Articles 74-75) 4;

1000 900 800

International multicenter studies

Local (non-BE) foreign-sponsored studies

BE foreign-sponsored studies

Local (non-BE) domestic-sponsored studies 212

BE domestic-sponsored studies

700 600 500 400

85

300

19 23

200 100

67 68

144

252

65

279

88 6 29 324

101 9 19 369

165

68

63

77

133 5 25

45

364

5

112

63 8 32

80

77 123

348

6 30

107 19 35

62

370

369

2011

2012

246

0 2004

2005

2006

2007

2008

2009

2010

Number of permits to conduct clinical trials in the Russian Federation 2004–2012.

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– Ministry of Healthcare and Social Development of the Russian Federation Order No. 748 “On the Approval of Procedures for Obtaining Permits to Conduct Clinical Trials of Drugs for Medical Use”5; – Ministry of Healthcare of the Russian Federation Order No. 266 “On the Approval of Rules for Clinical Practice in the Russian Federation” dated 19 June 20036; – GOST R 52379-2005 “GCP”11.

At present, filing an application at the Healthcare Ministry to obtain a permit to conduct clinical trial requires filling in the electronic form at the state’s “Register of drugs” portal 7. The time-related terms for obtaining a permit to conduct a clinical trial is specified in Federal Law No. 63-FZ (Articles 19, 20, 21) as 45 business days 3. In reality, however, the terms are frequently not met. In 2012, for example, obtaining a permit took about 80 business days on average 13.

The amount of duty that has to be paid is specified in Article 333.32.1., Part 2 of the Russian Tax Code. Costs are: RUB75,000 (a1,715) for an expert review of documents in obtaining the permit to conduct clinical trial of drugs for medical use, and RUB225,000 (a5,145) during state registration for a check of drug quality and evaluation of the ratio of expected usefulness to potential risk when taking the drug 8.

The ICH-GCP international ethical and quality standard for clinical trials is obligatory in the Russian Federation. GOST R 52379-2005 was approved by Order No. 232-st. (27 September 2005) from the country’s Federal Agency for Technical Regulation and Metrology 9. When conducting clinical trials on patients in the Russian Federation, life and health insurance is compulsory. The federal government’s Resolution No. 714 (13 September 2010) adopts standard insurance rules. The amount of insurance premium (for each respondent) lies between RUB1,445-9,811 (a33-a224) depending on the clinical phase and class of drug under trial. The amount of any subsequent insurance payouts depends on the severity of harm to the patient’s health, and generally falls between RUB300,0001,500,000 (a 6,860-a 34,298). A payout

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CLINICAL TRIALS could be more if a court judges this is appropriate10. Insurance for investigator liability is optional.

An ethical review of documents related to conducting a clinical trial of drugs for medical use is performed by the Council for Ethics2. This procedure is executed while the application for obtaining permit to conduct clinical trial is under examination (Article 17 of Federal Law No. 61-FZ).

Information about the decision made by the Council for Ethics is available to the public at: www.rosminzdrav.ru/docs. According to Chapter 3 of the GOST R 52379-2005 regulation on good clinical practice, local ethics evaluations are to be performed by an Independent Ethics Committee (IEC) for the trial site prior to the clinical trial11.

Inspections Inspections for controlling how clinical trials are conducted are carried out by the Federal Service for Supervision in the Sphere of Healthcare (Roszdravnadzor). These may involve either a desk audit (without a visit to the medical institution or sponsor’s office) or an on-site audit. On-site audits are required if a desk audit indicates that documents submitted by the sponsor of the clinical trial might not be authentic. Its activity complies with the law in force. On-site audits are also conducted when Roszdravnadzor receives data indicating actual or poten-

tial harm to life or health in a trial being organised or conducted. Scheduled inspections of institutions are carried out no more than once every three years, as per a plan approved by Roszdravnadzor annually. Conductors or organisers of trials are given notice of a scheduled inspection no later than three business days before it takes place 12.

Statistics

the Approval of Procedure for Obtaining Permit to Conduct Clinical Trial of Drugs for Medical Use” dated 26 August 2010 [6] Order No. 266 from the Ministry of Healthcare of the Russian Federation “On the Approval of Rules for Clinical Practice in the Russian Federation” dated 19 June 2003 [7] Register of drugs, URL: http://www.grls.rosminzdrav.ru (Stand: 21 Oct. 2013) [8] Tax Code of the Russian Federation No. 117-FZ dated 5 August 2000 [9] Order No. 232-st. from the Federal Agency for Tech-

According to the “Register of drugs” data portal, 915 permits to conduct a clinical trial were issued in the Russian Federation in 2012 (567 in 2011). Among them: 369 international multicentre clinical trials, 169 local foreign-sponsored trials (including 107 bioequivalence studies), and 377 local domestic-sponsored trials (including 212 bioequivalence studies) 7. 

nical Regulation and Metrology “On the Approval of National Standard” dated 27 September 2005 [10] Resolution No. 714 from the Russian Government “On the Approval of Standard Rules for Compulsory Insurance of Life and Health of Patients Participating in Clinical Trials of a Drug”, dated 13 September 2010 [11] GOST R 52379-2005 “Good Clinical Practice” [12] Control over pre-clinical and clinical trials: new rules, URL: http://www.cmslegal.ru/newsmedia/

References [1] Portal about clinical trials, URL: http://cra-club.ru/ content/view/567/ (Stand: 21 Oct. 2013) [2] Official site of the Ministry of Healthcare of the Russian Federation, URL: https://www.rosminzdrav.ru (visit date: 21.10.2013)

publications.ru (Stand: 21 Oct. 2013) [13] Information and analytical bulletin No. 6 from the Association of Clinical Trials Organizations, URL: http://acto-russia.org/files/bulletin_6.pdf [14] Constitution of the Russian Federation, URL: http://constitution.kremlin.ru

[3] Federal Law No. 61-FZ “On the circulation of drugs” dated 12 April 2010 [4] Federal Law No. 323-FZ “On the Fundamental

Contact Alexander Globenko

Healthcare Principles in the Russian Federation”

NADIM” LLC

dated 21 November 2011

8, ulitsa Tvardovskogo, Moscow, Russia, 123458

[5] Order No. 748 from the Ministry of Healthcare and Social Development of the Russian Federation “On

Tel.: +7-926-301-17-42; +7-499-550-66-53 alexander.globenko@nadim-cro.ru

Contract Contract Contra Contract Metronomia Manufacturing Manufacturing Manuf Manufacturing Excellence Excellence Excelle

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Nº 11 | Volume 12 | 2013

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Clinical Trials Risk Management

Secure the best value

By Heiko Werthmann, Gerd Grimm and Andreas Ries, KPMG AG, Stuttgart, Germany

The value of successful drug development multiplies from the preclinical phase up to proof-of-concept, and biotech leaders have developed strategies to capture a bigger piece of this cake. One approach is to start forward integration into early clinical trials. Due to the enormous investment needed to build up company-owned clinical operations, most biotech firms work together with partners to minimise the risks.

Outsourcing or cooperation with clinical research organisations (CROs) is necessary, but on its own is not sufficient to capture the value of an opportunity. One warning sign is that some big pharmaceutical companies encounter productivity and cost challenges in outsourcing activities. A lack of shared processes and communication between partners jeopardises a strategic objective.

Successful Business Partnership for biotech firms requires a common language, reliable processes and having a clear picture of the current and expected trial performance. The focus of this article is therefore on how to set up an information model that can overcome this challenge and help to implement a strategy of forward integra-

tion from a business point of view. Its objective is to give insights on best practices that minimise risks and secure value.

The business information model A major lever in tackling productivity challenges in the early phases lies within the business information model of clinical trials. In the course of a trial’s life cycle, changes are likely to occur – either driven by scientific needs or changes that arise in the field. It is important to understand the rationale and the impact in time to counter­act and assess the overall situation. An important management instrument, the business information model has three major components:

Behind schedule – over cost

Before schedule – over cost

In-time and in-budget

Behind schedule – under cost

Before schedule – under cost

Time Development Variation in recruitment progress measured in “days” Budget Performance Relation of actual costs to cost forecast, associated with recruitment milestones/targets

Diameter displays total trial budget

Time Development The easy-to-understand performance grid is a preferred management tool.

40_EBSIN11_13_Spezial-kpmg_bk.indd 40

© KPMG

Budget Performance

Performance Grid

Costing Scenario Management: Biotech firms should have a systematic approach to set up costing scenarios for trial execution – ideally with the help of dedicated models. Those models allow the evaluation of various options from a business perspective and support a cross­-functional assessment. For instance, the biotech strategist should be able to rely on an independent and internal opinion from the trial manager with support from purchasing or controlling.

Trial Scenario Management: As outlined above, cost is an important driver of productivity – but not the only one. Speed is an additional parameter that should be in focus as timelines – like those for registration – have tremendous impact on future commercial value. Scenario capabilities enable determination of whether there is room to maneuver to manage timelines and budgets, as some trials are time­ critical while others are cost-critical.

Business Intelligence: Bringing cost and speed together provides transparency, if the real generated value justifies spending.

If employed appropriately, a performance grid can serve as a powerful tool for evaluating a trial’s performance. It establishes transparency from clinical trial managers up to top management, and helps to identify where action is needed.

Conclusion Clients who have successfully implemented the business information model in their clinical operations or in outsourcing with CROs have established a platform for joint discussion and decisionmaking – focusing on key success factors and enabling the critical review of the trial performance at a single glance. This instrument provides stronger control of the estimated costs and the timely delivery of medical objectives. Additionally, the net present value of trials/ projects can be managed actively.

Transparent scenarios and a deep understanding of change-cost relationships are success factors for better and pro­active decision­making. This leads ultimately to a productivity increase and higher efficiency in scarce resource allocation, and reduces the risk of forward integration.

B

01.11.2013 13:17:24 Uhr