Stephanie Sutton was an assistant editor at Pharmaceutical Technology Europe.

Stephanie Sutton

GlaxoSmithKline (GSK) has provided further details concerning its new unit dedicated to rare diseases, which was 

 in February. According to the company, the new unit provides the necessary resources to create new medicines or approaches to tackling rare diseases. With more than 6000 rare diseases, however, Marc Dunoyer, Global Head of GSK Rare Diseases, explained in a 

 that the unit’s efforts will be very focused.

“We will target those diseases where we realistically believe we can make a significant contribution; ideally, we will be creating new medicines, but importantly by adding to the weight of scientific knowledge into rare disease to the benefit of all working in this area.”

A systematic approach will be used to select which diseases to target. According to Dunoyer, the aim is to create a list of approximately 200 priority diseases for the unit to target, although this list will probably change over time in light of new knowledge. “Other factors that will also influence our decision-making process will include our belief about the potential for a treatment to help manage a disease, its complications or even offer a cure,” said Dunoyer. “Not all rare disease are ‘reversible’ or can be significantly improved by therapeutic interventions and it will be vital that we are science-led in making these decisions.”

To ensure the right molecules are selected for development, the company has said it will leverage medicinal chemistry and biology expertise. “If we make the right decisions upfront, then ultimately the risk associated with discovery and development is generally lower as the conditions are often well defined and clinical development programmes tend to be small with robust endpoints. In most cases, the molecular target is known, making it easier for treating physicians to diagnose patients,” said Dunoyer.

GSK’s rare disease portfolio also received a further boost earlier this week with the 

 of an alliance with Fondazione Telethon and Fondazione San Raffaele, two institutes in Italy that have been collaborating in biomedical research. The alliance will research and develop treatments for rare genetic disorders using gene therapy carried out on stem cells taken from patients’ bone marrow. Under the terms of the agreement, GSK will gain an exclusive license to develop and commercialize an investigational gene therapy for ADA severe combined immune Deficiency, which affects approximately 350 children worldwide. 

The company will also co-develop with the two institutes six further applications of ex vivo stem cell therapy using a new gene transfer therapy that has the potential to treat a range of rare disorders.

In a statement, Philippe Monteyne, Head of Development and Chief Medical Officer for GSK Rare Diseases, described the alliance as an “important addition” to the company’s portfolio of rare diseases.

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GlaxoSmithKline (GSK) has provided further details concerning its new unit dedicated to rare diseases, which was launched in February.

GSK details rare diseases strategy

The European Generic medicines Association (EGA) has made a number of recommendations on how to ensure the quality of medicines in the globalised supply chain; in particular, the EGA spoke about API quality and quality supervision, and how these can be improved by transparent communication between authorities and industry. 

Speaking at the EDQM’s Quality of Medicines in a Globalized World: Dreams and Reality international conference last week, Suzette Kox, Senior Director of Scientific Affairs at the EGA, explained in a 

 that pharma manufacturers must exercise care when selecting API suppliers, and should monitor them carefully to ensure that only GMP-compliant APIs are used. She recommended both industry and regulatory authorities apply a common risk-based prioritization to API manufacturers and suppliers, as well as to increase focus on API audit programmes and reports during EU inspections. 

“Since the relationship with API manufacturers is an essential part of maintaining the integrity of the supply chain, business only with certified partners provides the guarantee of uninterrupted and timely exchanges of information and continuous quality assurance,” said Kox.

To overcome the issue of limited resources, the EGA also suggested the creation of a formal EU Inspection Group to optimize an existing network of national inspectors and to free up more inspection capability. Kox also explained the importance of industry access to inspection information such as inspection outcomes, GxP compliance and operator licenses, which can support risk assessment and audit prioritisation. “Access to a schedule of upcoming routine inspections by EU health authorities would allow optimisation of audit planning,” Kox also added. 

Regarding international cooperation, the EGA has recommended streamlining inspections at a global level by building on a number of existing platforms and agreements, such as leveraging the experience of the Pharmaceutical Inspection Convention and Pharmaceutical Inspection Co-operation Scheme to assess ‘equivalence of standards’ between countries. Additionally, the EGA recommended  developing bilateral agreements to facilitate exchange of information, training, common understanding and confidence building with key API exporting countries. 

In the long term, the EGA also hopes the industry can work towards a long-term global inspection framework. 

“The industry is dreaming of truly globally harmonized standards for pharmaceutical products and APIs, including pharmacopeias and why not a harmonized regulatory system, with harmonized registration requirements and assessments, and mutual recognition of approvals and inspections,” said Kox. “This EDQM international conference contributes to setting the scene and paving the road in that direction.”

The European Generic medicines Association (EGA) has made a number of recommendations on how to ensure the quality of medicines in the globalised supply chain; in particular, the EGA spoke about API quality and quality supervision, and how these can be improved by transparent communication between authorities and industry.

EGA calls for better API quality measures

The European Generic medicines Association (EGA) has made a number of recommendations on how to ensure the quality of medicines in the globalized supply chain. In particular, EGA spoke about the quality and quality supervision of active pharmaceutical ingredients (APIs), and how these can be improved by transparent communication between authorities and industry.

  Speaking at the European Directorate for the Quality of Medicine’s (EDQM)

 Quality of Medicines in a Globalized World: Dreams and Reality

 international conference last week, Suzette Kox, senior director of scientific affairs at EGA, explained in a 

 that pharmaceutical manufacturers must exercise care when selecting API suppliers, and should monitor them to ensure that only GMP-compliant APIs are used. She recommended that both industry and regulatory authorities apply a risk-based approach to the selection of API manufacturers and suppliers. The approach may also increase the focus on API audit programs and reports that take place on European Union inspections.

  To overcome the challenge of limited resources, EGA suggested the creation of a formal EU Inspection Group to optimize an existing network of national inspectors and to free up more inspection capability. Kox also explained the importance of industry access to inspection information.

“The online access by the industry to most information on inspection outcomes as foreseen today, GxP compliance and operator licenses (e.g., importers) is expected to constitute an invaluable tool for industry to support their risk assessment and audit prioritization,” said Kox. “Access to a schedule of upcoming routine inspections by EU health authorities would allow optimization of audit planning.”

  Regarding international cooperation, EGA has recommended streamlining inspections at a global level by building on existing platforms and agreements such as leveraging the experience of the Pharmaceutical Inspection Convention and Pharmaceutical Inspection Co-operation Scheme (PIC/S) to assess the “equivalence of standards” among countries. Additionally, EGA recommended  developing bilateral agreements to facilitate the exchange of information, training, common understanding, and and confidence building with key API exporting countries.

  In the long term, EGA said it hopes the industry can work toward a long-term global inspection framework. “The industry is dreaming of truly globally harmonized standards for pharmaceutical products and APIs, including pharmacopoeias and why not a harmonized regulatory system, with harmonized registration requirements and assessments, and mutual recognition of approvals and inspections,” said Kox. “This EDQM international conference contributes to setting the scene and paving the road in that direction.”

The European Generic medicines Association (EGA) has made a number of recommendations on how to ensure the quality of medicines in the globalized supply chain; in particular, the EGA spoke about API quality and quality supervision, and how these can be improved by transparent communication between authorities and industry.

European Generic Medicines Association Addresses Supply-Chain Quality Issues, Recommends Global Inspection Framework

GlaxoSmithKline (GSK)  provided further details concerning its new unit dedicated to rare diseases, which was 

 in February. According to the company, the new unit provides the necessary resources to create new medicines and approaches to tackling rare diseases. With more than 6000 rare diseases, however, Marc Dunoyer, global head of GSK rare diseases, explained in a 

 that the unit’s efforts will be  focused.

A systematic approach will help GSK select which diseases to target. According to Dunoyer, the aim is to create a list of approximately 200 priority diseases, although this list will probably change over time in light of new knowledge. “Other factors that will also influence our decision-making process will include our belief about the potential for a treatment to help manage a disease, its complications or even offer a cure,” said Dunoyer. “Not all rare disease are ‘reversible’ or can be significantly improved by therapeutic interventions and it will be vital that we are science-led in making these decisions.”

To ensure the right molecules are selected for development, the company  said it will leverage medicinal chemistry and biological expertise. “If we make the right decisions up front, then ultimately the risk associated with discovery and development is generally lower, as the conditions are often well defined and clinical development programs tend to be small with robust endpoints. In most cases, the molecular target is known, making it easier for treating physicians to diagnose patients,” said Dunoyer.

GSK’s rare-disease portfolio also received a further boost earlier this week with the 

 of an alliance with Fondazione Telethon and Fondazione San Raffaele, two institutes in Italy that have been collaborating in biomedical research. The alliance will research and develop treatments for rare genetic disorders using gene therapy carried out on stem cells taken from patients’ bone marrow. Under the terms of the agreement, GSK will gain an exclusive license to develop and commercialize an investigational gene therapy for ADA Severe Combined Immune Deficiency, which affects approximately 350 children worldwide. 

The company will also co-develop with the two institutes six further applications of 

stem-cell therapy using a new gene-transfer therapy that has the potential to treat a range of rare disorders. In a statement, Philippe Monteyne, Head of Development and Chief Medical Officer for GSK Rare Diseases, described the alliance as an “important addition” to the company’s portfolio of rare diseases.

GSK Details Rare-Diseases Strategy

Following the low growth of the global pharma market in 2010, IMS Health’s 

 now expects the industry to rebound somewhat in 2011, growing at a rate of 5–7% compared with only 4–5% in 2010 to reach $880 billion. 

However, it’s not all good news as the underlying constraints to growth, such as the impact of patent expiries and government initiatives to limit drug spending, will be stronger than ever in 2011.

“In 2011, products with sales of more than $30 billion are expected to face the prospect of generic competition in the major developed markets,” explains a 

. “In the US alone, Lipitor, Plavix, Zyprexa and Levaquin – which together accounted for more than 93 million prescriptions dispensed in the past 12 months and generated over $17 billion in total sales — likely will lose market exclusivity.”

Data also paint an uncertain picture for 2012 — as this is when patent expiries and the shift to lower-cost generic alternatives will have their full impact because of the timing and expected competitive intensity among generic entrants. 

2011 and beyond will also see pharma coming to terms with reductions in the drug budgets of public and private payers. “Multiple markets will be impacted by these measures in 2011,” said IMS. “Prominent examples include substantial reductions in the price of generics relative to their branded counterparts in Spain and in Canada, where generic pharmacy rebates are expected to be eliminated; new price negotiation requirements for brands launched in Germany; and across-the-board price cuts for branded products in Turkey and Greece.”

Although the future of the developed pharma markets may still be a little unsteady, it’s a different story for pharmerging countries, which are forecast to grow at a rate of 15–17% in 2011 to reach up to $180 billion. Many of these markets are benefiting from greater government spending on healthcare, as well as greater demand and access to medicines. In particular, China, which is now the world’s third-largest pharma market, is expected to grow by 25–27% to reach more than $50 billion next year.

In comparison, the predicted growth for developed markets is much lower. Japan is expected to grow 5–7% in 2011 as biennial price cuts are expected to have little impact, but the five major European markets (Germany, France, Italy, Spain and the UK) will collectively grow at a rate of only 1–3%. Meanwhile, the US is forecast to experience 3–5% growth. 

So where does pharma go from here? Particularly developed markets that seem to have tough times ahead? According to IMS, there is a “promising wave of innovation” and potential for several significant treatment options for stroke prevention, breast cancer and hepatitis C, among others. Five potential blockbuster products are also expected to be approved and launched globally by the end of next year.

“In 2011, we will see the loss of exclusivity for some iconic brands and a promising new wave of innovation,” Murray Aitken, IMS Senior Vice President, said in the press statement. “It also will be a critical year for gauging how healthcare reform initiatives in key markets evolve and play out amid the expected macroeconomic recovery. For pharmaceutical manufacturers, an unrelenting focus on bringing distinct value to patients and health systems will be essential to navigating this dynamic market.”

Following the low growth of the global pharma market in 2010, IMS Health?s IMS Market Prognosis now expects the industry to rebound somewhat in 2011, growing at a rate of 5?7% compared with only 4?5% in 2010 to reach $880 billion.

Global pharma market to rebound in 2011

Restructuring at sanofi-aventis will result in the elimination of approximately 1700 jobs in the US — about 25% of the company’s US Pharmaceutical Operations division workforce. The company employs 13000 people in the US; more than half of which are based in the Pharmaceutical Operations division. 

, the cuts and restructuring will “position the company for growth following the impact of patent expiries, increase business development and other innovative partnerships, and ensure investment in late lifecycle products”. Going forward, the focus of the division will be on diabetes, atrial fibrillation and oncology.

The positions to be axed will be finalised by mid-December. According to an interview with a company spokesperson, conducted by 

, however, the cuts will come from sales (1400 positions) and company offices in New Jersey (300). The spokesperson also reportedly added that the cuts are part of the 2 billion euro cost-reduction plan announced by the company in

“Given the serious challenges facing our organization and the healthcare industry, it is important to act decisively now so that our organization has greater stability moving forward and that our resources are allocated to our strategic growth priorities,” Gregory Irace, President and CEO of sanofi-aventis US/Canada Pharmaceutical Operations, said in a press statement. “This step in our transformation strategy will expedite decision-making, reduce business complexities and increase innovation, ensuring we rapidly respond to patient and customer needs.”

The news about the layoffs comes as sanofi attempts a hostile takeover of Genzyme (

sanofi-aventis Attempts Hostile Takeover of Genzyme

), which would provide sanofi with a pipeline of drugs for rare genetic disorders. According to an analyst from UBS in London (UK), who reportedly spoke to Bloomberg, however, the layoffs are not connected to the potential acquisition. 

Restructuring at sanofi-aventis will result in the elimination of approximately 1700 jobs in the US &#151; about 25% of the company?s US Pharmaceutical Operations division workforce.