April Spotlight

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Looking Back, Looking Ahead


Check both ways before proceeding — always good advice. Industry analysts at GlobalData and Mergermarket (an Acuris company) have reviewed 2017 and made predictions for the biopharmaceutical industry in 2018. Set against a backdrop of remarkable advances in technology and the growing relevance of biosimilars, last year was marked by both disappointments and noteworthy innovations.

GlobalData points to major regulatory approvals in “nearly every major therapeutic area.” These include CAR T-cell oncology treatments, the first biologic for atopic dermatitis, and the first drug for primary progressive multiple sclerosis. Global director of therapy analysis and epidemiology Claire Herman says, ‘‘Each fills a critical clinical unmet need and alters the treatment paradigm.’’ But R&D saw significant setbacks in 2017 as well, including two candidate failures for Alzheimer’s disease and two major approved drugs that did not meet endpoints in key oncology trials.

Meanwhile, biosimilars are growing competitive in immunology and oncology, with ophthalmology not far behind. But approvals have not yet led to high clinical uptake. ‘‘As physicians’ comfort with the class grows,” Herman says, “we expect increased erosion in 2018 and beyond.’’ She touts the impact of technology as well, with advances in digital health across drug life cycles — from clinical trials, to disease diagnosis, treatment selection, and patient monitoring. ‘‘With collaborations between pharma companies and technology giants such as Apple and Google making headlines, the pace of digital health innovation is unlikely to slow.’’

In its global mergers and acquisitions (M&A) roundup report for 2017, Mergermarket offered these key findings: M&A activity dropped, with US$265 billion spent across 1,490 transactions leaving the sector at its lowest value in four years. That accounted for 8.4% market share of global M&A (8.7% in 2016). The top 10 deals accounted for 41.6% of the sector’s entire combined value. Despite scientific milestones in cell and gene therapies, the industry continued to come under heavy pricing pressures and political challenges. But investor interest remains steady.

GlobalData predicts much more M&A activity in 2018, attributing much of that to the US tax bill passed last year. Healthcare analyst Rahael Maladwalasa says that includes a huge cut in the overall tax rate, pushing it down to <20% for most companies and leaving pharmaceutical giants flush with excess cash. “This has the potential to significantly alter the market as companies allocate the excess to various strategies, including opening new plants, rewarding stakeholders, and looking for M&A.”

M&As are pursued for four main reasons: to increase market share/penetration, add complementary therapies to existing portfolios (product development), build on existing competencies in a new market (market development, and add new products in a new market (diversification). Although some companies (e.g., Pfizer, Merck, and Roche) have stated that they are not rushing into M&A, they did not rule it out. Some may focus on organic growth.

“This strategy could see investment into research and manufacturing, which would in turn lead to an increase in the number of jobs,” suggests Maladwalasa. “Companies that grow through acquisition typically have to pay the standing value of the acquired company, as well as a takeover premium. And several studies have shown that organic growth can generate more value in the long term, although it can take more time and effort. However, with poor growth forecast for many of the pharma giants, it may not be too long before they start to look at M&A as a short-term fix.”

Sure enough, Sanofi announced two multibillion-dollar buys to boost its rare-disease pipeline; Celgene completed buyouts of Juno Therapeutics and Impact Biomedicines to expand its oncology pipeline; and Impax Laboratories and Amneal are merging to become the fifth largest generics maker in the United States. Healthcare analyst Ashwin Oberoi pointed to Merck, Pfizer, and Amgen as likely to expand through M&A using their large off-shore cash stores. “Companies that remain as likely targets generally include those with therapies that have recently gained approval or are likely to gain approval within high-impact disease areas. Generally, interest is strong in companies that focus in oncology, rare diseases, and gene-editing technologies, such as BioMarin, Clovis Oncology, Puma Biotech, and Bluebird Bio.”

GlobalData expects the M&A trend to continue throughout 2018. “As a result of improved earnings, economic growth, and recent deal activity, the biotechnology sector is poised to reach an all-time record high.”

In February AstraZeneca and Merck announced an $8.5 billion agreement to codevelop an oncology drug. AstraZeneca tops strategic alliance spending for 2017 with development deals involving Pieris Pharmaceuticals and MedImmune. Healthcare analyst Lisa Marris says, “Despite that, Merck had an arguably more successful year. It more than quadrupled its alliance deal values since 2016. And if its subsidiaries are added to the equation, its 2017 deal values rise to $9.5 billion, an increase of 248% from 2016, whereas AstraZeneca and its subsidiaries stayed almost stagnant at $11 billion.”

Allergan and Novartis both experienced a slower 2017 than 2016, with decreases in partnership/licensing deal values by 44% and 5%, respectively. Yet both companies closed big deals last year. GlobalData predicts that 2018 will be Merck’s year in terms of R&D collaborations.

Forum Addresses Continuous Processing


For several years, as continuous biomanufacturing has come to the forefront, many producers of biotherapeutics have tried to strategically integrate such approaches into their own bioprocesses. In a continuous process, a biological product stream moves stepwise through manufacturing as each unit operation is completed — without intermediate hold periods. Benefits offered by integrating such a concept could include increased productivity, enhanced process flexibility and agility, reduced facility and manufacturing costs, and improved product quality. Many continuous technologies have been incorporated on biomanufacturing floors already. However, some operations are still in development, and gaps remain before a fully integrated, end-to-end solution can be realized.

This year’s World Biopharm Forum focuses on reviewing many recent achievements in commercial implementation of continuous biomanufacturing as well as addressing the remaining challenges. Subtitled “Continuous Biomanufacturing: Achievements and Challenges for Commercial Implementation,” the conference will take place on 20–22 June 2018 in at the Lady Margaret Hall at the historic University of Oxford in the United Kingdom. It brings together leading industrial scientists, engineers, suppliers, and academics to address recent technological advancements and practical applications in continuous biomanufacturing — including new technologies facilitating the implementation of either hybrid or fully continuous bioproduction systems. The agenda highlights keynote lectures, case studies, and panel discussions on such topics as design of end-to-end modular automation; technology and materials to enable intensified perfusion culture; continuous downstream processing; and approaches for monitoring and control of integrated continuous bioprocesses. Challenges in implementation of single-use systems, integrated system process control, and the newest approaches to business-case development also will be discussed in detail.

Join such industry innovators as Merck, GE Healthcare, Novartis, Sartorius Stedim, ThermoFisher Scientific, Lonza, and the Max Planck Institute to review innovations and changes in this exciting area of biotechnology this June. For more information, visit http://www.subramanian.org.uk/Conferences.html.

Standards in Revision for Worldwide Description of Medicinal Products


Reliable, robust, and consistent exchange of medicinal product information is essential to meeting the objectives of both medicines and pharmacovigilance regulations. The Identification of Medicinal Products (IDMP) international standards are under revision to support that goal. Implementing these standards should simplify the exchange of information and enhance the interoperability of systems across the medical field. IDMP standards and technical specifications — ISO 11616, ISO 11615, ISO/DIS 11238, ISO/TS 20451, ISO/TS 20443, and ISO/TS 19844 — cover a range of regulatory activities related to development, registration, and life-cycle management of medicinal products as well as pharmacovigilance and risk management.

Christian Hay is a senior healthcare consultant for the GS1 Global Office and convenor of the International Standards Organization’s (ISO’s) working group for the pharmacy and medicines business. “IDMP standards are essential to the world’s increasingly integrated healthcare,” he explains. “They provide a precise architecture for computerization of information on medicinal products around the world. When regulators adopt IDMP, their capacity to interoperate with each other makes for safer patient care. This is, for example, a huge benefit for adverse-event reporting and for documenting medication in patient records.”

Publication of these standards in 2012 gave the global medical community help in understanding potential changes in diverse data models. After learning from users’ reactions, the IDMP project leaders initiated a further standards-development program consisting of working on implementation guides. Those are shifting some detailed information from the standards themselves to their corresponding guides. Users can expect educational materials and uniform implementation by both manufacturers and regulators. In parallel, IDMP provides a basis for existing or new information technology solutions: e.g., prescriptions, medication reports, product dictionaries for clinical use, and so on.

Using ISO IDMP within regulatory activities should benefit regulators, companies, and patients. “The trend toward global standards continues to increase,” Hay says. “I cannot imagine a world without IDMP, whose implementation program is going to last several years. Without IDMP, the existing information fragmentation by country or region would cause increasing risks to patients globally — not only those who travel, but those who are faced with mobile health [monitoring and treatment using mobile technologies] or because of the globalization of supply chains.”

The ISO IDMP standards were developed by ISO technical committee 215 on health informatics. Its secretariat is held by the American National Standards Institute (ANSI), ISO’s US member. Read more online at www.iso.org/news/ref2234.html.

Correction: December 2017


The authorship was incorrect for “Simplify Upstream Process Development and Scale-Up: Single-Use 5:1 Turndown-Ratio Bioreactor Technology” in BPI’s December 2017 issue. The authors are Benjamin Madsen, Jeff Hurd, Chris Brau, and Nephi Jones, all in single-use technologies at Thermo Fisher Scientific.

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