Pfizer continues on rare disease path with new R&D unit

Pfizer continues its foray into rare diseases as it creates a new R&D unit to investigate novel therapeutics for conditions such as Haemophilia. The pharma giant joins GlaxoSmithKline and Novartis as companies looking into producing specialised drugs as a way to offset slowing sales growth and loss of blockbuster patent protection.

The new unit created within Pfizer’s Worldwide Research and Development operation aims to capitalise on Pfizer’s existing research into such conditions as Haemophilia. In the past, Pfizer have also expressed its interest in treatments for muscular dystrophy and other conditions caused by genetic mutations.

The creation of an R&D unit represents a significant strategy shift into rare diseases for the firm. In December of last year, Pfizer agreed to pay $60 million initially with a further $55 million to license the worldwide rights to Protalix Biotherapeutic's drug for Gaucher disease.

This shift also produced job cuts as the company announced last month its intention to shed roughly 6,000 manufacturing jobs worldwide over the next five years.

This includes approximately 300 at the Andover biotechnology plant Pfizer took over when it bought Wyeth Pharmaceuticals for $68 billion last year.

With the impending expiration of blockbusters and a slowdown in emerging pipeline candidates, industry insiders believe pharma’s passing interest in specialised markets is gaining increasing momentum.Drugs produced for ultra-orphan diseases with high unmet needs have an easier time commanding high prices. Genzyme’s Cerezyme, another drug used to treat Gaucher disease can command prices of up to $200,000 a year while medicines for cancer can cost $50,000 or more a year.

In addition, the growing trend of pharma companies using biomarkers to improve on current methods to define populations of patients increases the prospect of smaller markets suddenly becomes more attractive and financially viable.

Pfizer joins a growing band of companies who are currently pursuing treatments for rare diseases. In March, GlaxoSmithKline (GSK) steeped up its efforts to build a rare diseases business which saw the UK pharmaceutical major launch its own dedicated R&D unit.

Bristol-Myers Squibb has signed agreements with clinical development service providers, Icon and Parexel, for joint strategic, operational and capability support of its clinical development program.
As per the agreement terms, Icon and Parexel are expected to provide support for Bristol-Myers Squibb’s clinical studies to support its full development pipeline over the next three years.
Bristol-Myers Squibb is preparing for a large volume of clinical development work that is expected to require expanding upon its existing partnering approach for clinical development.
Bristol-Myers Squibb also stated that, consistent with the its BioPharma strategy, the agreements with Icon and Parexel are expected to complement its internal capabilities and capacity.
Brian Daniels, senior vice president of global development at Bristol-Myers Squibb, said: “These partnerships are expected to increase the operational capability of our clinical development organisation, and support our position in productivity and innovation.
“Working with Icon and Parexel, Bristol-Myers Squibb will enhance support for our pipeline and improve our ability to deliver medicines to patients with serious disease.”
John Hubbard, president of clinical research services at Icon Group, said: ‘‘We are delighted that Bristol-Myers Squibb has selected us to support its clinical development programs.
“Over the last ten years, we have forged a partnership by working hard to fully understand Bristol-Myers Squibb’s requirements. We look forward to moving this partnership to another level and continuing to support Bristol-Myers Squibb’s goal of bringing treatments to patients."
Josef von Rickenbach, chairman and CEO of Parexel, said: “We look forward to a deeply collaborative relationship with Bristol-Myers Squibb to help provide solutions for its growing pipeline, as well as leverage our capabilities and technology infrastructure to support the effectiveness of its clinical development programs."

US CRO Kendle sets up in Ahmedabad

US CRO Kendle has opened a new services centre in India, attracted by the country’s thriving contract research sector and favourable climate for clinical trials.
The 14,000 sqft centre, in the Ahmedabad-Gandhinagar special economic zone (SEZ), will provide clinical data management, pharmacovigilance and biostatistics services to sponsors running trials in the region.

Kendle has had a presence in India since 2004 with its unit in New Delhi and has been expanding to allow its clients increased access to the nation’s favourable research conditions.

COO Stephen Cutler said that: "Growth in India is a key component of our strategy and will be very important to the future" adding that an "expanded presence in Asia, and in India in particular, creates efficiencies in the clinical development process.”

In an interview in the Business Standard Cutler contrasted India with Asia’s other contract research hotbed China, explaining that “India's edge… lies in its favourable regulatory environment and research initiatives as well as investor-friendly duty structure.”

The new unit will begin operations with a staff of fifty although Kendle plans to scale up this up in the next few years, benefiting from the units “proximity to multiple universities” in and around Ahmedabad.
Aside from access to large population centres and proximity to a number of pharmaceutical manufacturers the Ahmedabad-Gandhinagar also provides Kendle with others benefits due to its status as an SEZ.

Operating in an SEZ gives contract research organisations (CROs) like Kendle, and GVK Biosciences who set up there in March , staged tax benefits, including a five year 100 per cent exemption on export income.

New norms for clinical research organizations soon in India.

The government will soon make it mandatory for all firms involved in clinical research to maintain minimum quality standards and register themselves with the country’s drug regulator.

Once the law is implemented, the names of all the approved Clinical Research Organisations (CROs) in the country will be uploaded on the website of Drug Controller General of India (DCGI). A violation of the norms could lead to the firm being barred from conducting clinical studies in India.

Several inspectors at the DCGI have been trained by the US Foods and Drug Administration to audit CROs. The country’s highest technical body on medicines, Drug Technical Advisory Board, has approved an amendment to the Drugs and Cosmetics Act to make CRO registrations compulsory.

Industry experts believe that this will regulate the entire clinical protocols, ethical norms and other practices. At present, there are 40-50 CROs in the country, a number that is increasing steadily. All these provide research services to drug companies in developing medicines, especially the clinical trials.

Admissions for the Next Batch

Admissions for the next batch of clinical research (2010-11) are now open. The application form can be downloaded from our website