Shire links with US hospital for rare disease research

Shire has boosted its R&D capabilities via an alliance with Cincinnati Children's Hospital Medical Centre in the US, a national centre for rare disease research.

The two partners will work on the discovery and development of novel rare disease therapies over the next three years, with the initial objective of brining at least one development candidate during the period, according to the pharma company.

Pharma firms have become increasingly interested in rare and orphan diseases in recent years as they can often command premium pricing, lower marketing costs and fewer reimbursement obstacles than drugs for more common conditions, and also have longer exclusivity in the marketplace.

Cincinnati Children's Hospital has more than 50 research divisions that specialise in combining patient observations with laboratory work, including genetic sequencing, to develop new diagnostics and therapies for cancer, respiratory disease, gastroenterology, nephrology and neurology.

The hospital is in the midst of a $250m fund-raising exercise to support its research programmes and is scheduled to open a new clinical sciences unit later this year that will make it the largest paediatric research centre in the US.

It already has a rare and complex disease collaboration with Alexion Pharmaceuticals - which it describes as a "model for how industry and academia can better work together to bring health innovations from the bench to the bedside" - and has made no secret of wanting to expand its links with pharma.

On Shire's part, the deal bolsters its early-stage research pipeline in rare disease after a prolonged period of adding late-stage assets to its product portfolio.

For example, the company has been steadily building its rare disease franchise over the last couple of years, with deals such as the 2013 purchase of Viropharma for £2bn, which brought hereditary angioedema (HAE) Cinryze in-house, as well as its planned $5.2bn takeover of NPS that will add hypoparathyroidism drug Natpara.

"Shire has a deep commitment to patients with rare diseases, and our developmental expertise, combined with Cincinnati Children's research capabilities, should enable an acceleration of our discovery and development efforts," said Philip Vickers, the company's global head of R&D.

CROs and trial design • Early access and regulation • Value demonstration • Strategic communications