Straight talk with...Joe Selby

The US Supreme Court's ruling in late June to uphold most of the Patient Protection and Affordable Care Act of 2010 decided more than just the fate of the law's individual mandate for health care. It also allowed many of the other provisions of the legislation to safely go ahead. Chief among those was the establishment of a comparative-effectiveness research hub called the Patient-Centered Outcomes Research Institute (PCORI), located in Washington, DC. With a budget of $3.3 billion over the next seven years and an independent status, PCORI is now tasked with creating the evidence base to help patients and doctors make more informed decisions about their medical choices. Leading the fledgling institute is executive director Joe Selby. A family physician and clinical epidemiologist, Selby joined PCORI in July 2011 after 13 years as head of research at Kaiser Permanente, the California-based health provider. Even amid all the uncertainty that surrounded the future of US healthcare reform, Selby, together with PCORI's board of directors, managed to help craft the institute's first research agenda and to fund 50 pilot projects totaling $30 million, all in the spring of this year. Elie Dolgin spoke with Selby to discuss how PCORI will influence the debate about which treatments work best.     

Straight talk with...Joseph Schwartz

On 17 May, Human Genome Sciences (HGS) formally rejected a $2.6 billion unsolicited takeover offer by GlaxoSmithKline. The Maryland-based biotech firm had long partnered with the UK drug giant to develop drugs including Benlysta (belimumab), HGS's first drug on the market and the first new medicine approved to fight lupus in 50 years. But, at $13 a share, GSK's bid was deemed "inadequate" by the HGS board. Recent history in the biotech sector shows how high the stakes are in such negotiations. Since last November, six biopharma buyouts have exceeded $1 billion each, with Gilead Sciences' purchase last year of the hepatitis C specialist Pharmasset topping the charts at a whopping $11.2 billion, the highest ever paid for a clinical-stage biotech and an 89% premium to its share price at the time. More recently, in April AstraZeneca paid $1.3 billion for Ardea Biosciences, a company with cancer and gout drugs in the pipeline but nothing on the market.One person watching the upward clime of such deals is biopharma analyst Joseph Schwartz, a managing director at Leerink Swann in Boston. Mark Ratner sought out Schwartz, who was named the top stock picker for pharmaceuticals in the last year's FT/StarMine Analyst Awards, for his views on what's behind the recent buyout spending.     

Straight talk with... Mark Sculpher

By 2014, the UK will be changing the way it regulates the price it pays for medicines. Currently the country's National Health Service (NHS) uses the Pharmaceutical Price Regulation Scheme (PPRS), which controls the maximum profit a drug manufacturer can make on the basis of capital investments the manufacturer has made. A review of this system, carried out by the UK Office of Fair Trading in 2007, recommended that, instead, medicines should be priced according to the therapeutic benefits they offer to patients-an idea known as value-based pricing (VBP) that has similarities to systems already used in smaller countries such as Australia and Canada. This pricing scheme has been embraced by the UK government, and negotiations on how the system will work are due to begin this month. A British switch to VBP could have ripple effects throughout the global pharmaceutical industry. Even though the UK's share of the world drug market is relatively small-just 3%-drug prices in the country are important because a quarter of national governments reference British values to determine their own sticker prices. One of the most influential thinkers on the UK's proposed system is health economist Mark Sculpher, director of the Programme on Economic Evaluation and Health Technology Assessment at the University of York. As a regular advisor to the UK National Institute for Health and Clinical Excellence (NICE) and former chair of a task force on methods guidance for economic evaluation at the agency, Sculpher has had an instrumental role in guiding value-based decision-making in the country's healthcare system. Kate Ravilious met with Sculpher at his office in York to discuss the value of VBP.     

Straight talk with... Mahendra Rao by Dolgin Elie

In October 2005, Mahendra Rao shocked the scientific community when he quit his job as head of the US National Institute on Aging's stem cell section and announced plans to go into industry. Rao felt that a ban at the time on federal funding for most human embryonic stem cell research hampered researchers in his division and prohibited him from doing the job he was hired to do. So he joined the research-tool giant Invitrogen (which later became Life Technologies) as vice president of regenerative medicine at the company's Maryland facility. Six years on, times have changed in the field of stem cell biology: rules governing taxpayer-backed research involving embryonic stem (ES) cells have been relaxed in the US, and induced pluripotent stem (iPS) cells have come into the fray. Prompted by those changes, Rao opted to return to the US National Institutes of Health (NIH) in August to head the new Intramural Center for Regenerative Medicine. The $52 million center was launched in early 2010 by the agency to develop new therapies using stem cell approaches. With a heightened focus at the NIH on translational medicine, Elie Dolgin spoke to Rao to find out how he plans to turn stem cell discoveries into cell-based therapies.     

Straight talk with...Ellen 't Hoen

In July, the global health financing mechanism UNITAID established an intellectual property-sharing scheme focused on scaling up access to new and lower-priced antiretroviral drugs in the developing world. The initiative-called the Medicines Patent Pool (MPP)-aims to streamline licensing processes, drive the combination of multiple HIV medicines into one pill and foster the development of drug formulations for children. In September, the US National Institutes of Health (NIH) became the first contributor to the venture, licensing a suite of patents related to protease inhibitors that are used to treat HIV. The task of bringing drug firms and other key stakeholders into the fold now falls on Ellen 't Hoen, a lawyer who became MPP's executive director last month after previously heading up Médecins Sans Frontières' Campaign for Access to Essential Medicines. Asher Mullard spoke to Hoen about the challenges of encouraging companies to share their intellectual property in a normally guarded sector.     

Straight talk with...Leonor Beleza. Interview by Lucas Laursen

Portuguese businessman António Champalimaud surprised his family when his will, opened after his 2004 death, revealed that he was bequeathing €500 million ($690 million), about a quarter of his estate, to establish a foundation for applied biomedical research. He also surprised law professor and one-time Portuguese Health Minister Leonor Beleza, whom he named to lead the foundation. Beleza, who met Champalimaud just once, agreed in principle to run his proposed foundation during a phone call in 2000 but did not hear any further until his death. She has now returned from a global tour of medical research institutions and foundations lasting over a year to determine how best to spend Champalimaud's millions.On 5 October, the Champalimaud Foundation opened its seaside Center for the Unknown in Lisbon, Portugal. The center will host about 600 researchers and physicians and 300 patients when it reaches full staffing levels. Lucas Laursen recently called Beleza to ask how she laid the groundwork and what lies ahead.