Califf gets the nod that counts
The likely appointment of Robert Califf as the next commissioner of the FDA – he was nominated by President Barack Obama this week – comes as little surprise.
Califf was appointed to his current role at the administration as deputy commissioner for medicinal products and tobacco in January of this year and was immediately touted as a potential successor to Margaret Hamburg. Her resignation just a week later provided further suggestion that Califf had been lined up for the top job; a view supported by the broad remit of his current role. Furthermore, he had been cited as a candidate for the position of commissioner prior to Hamburg's appointment in 2009.
President Obama's nomination is unlikely to face any significant opposition and appears to have attracted early support from both the healthcare and pharmaceutical sectors.
As Matthew Herper notes for Forbes, 2015 – rather than six years ago – could be a more suitable time for Califf to take up the role. He was previously overlooked in favour of Hamburg by the Obama administration given his close links to industry; Hamburg, however, was "not as tough" on the pharma sector as many had predicted. Focusing heavily on improved communication between the regulator and pharma companies, she oversaw a period of notably faster and higher drug approval rates.
With Califf's interaction with industry heavily dictated by the large-scale clinical studies he helped run in collaboration between drug companies and Duke University, he could give the FDA's broader drug approval process a slightly more conservative focus.
The sensor in a pill – can it revolutionise patient adherence?
An ever-converging relationship between the pharmaceutical and technology sectors has been highlighted by recent FDA acceptance of a new drug application for a version of Takeda's schizophrenia treatment Abilify, which incorporates an ingestible sensor allowing patients, physicians and caregivers to track whether and when a patient has taken their medication.
Designed primarily to tackle the significant cost burden associated with shortfalls in patient adherence, Takeda has partnered with Proteus Digital Health to develop this version of Abilify; it hopes in the future to use the technology in other drugs, with Otsuka US executive vice president Bob McQuade suggesting there is "conceptually, no reason why this technology could not be used in all oral medications."
Proteus CEO Andrew Thompson describes the solution offered by this new version of Abilify as "a new category of computing, one that allows pharmaceutical companies to combine ingestible, wearable, mobile and cloud-based elements to transform patient adherence."
Read more…Spotlight On Interview: Can a microscopic sensor-in-a-drug revolutionise patient adherence? – FirstWord discusses this breakthrough technology with Proteus CEO Andrew Thompson and Otsuka US executive vice president Bob McQuade.
Entresto's turn under the value spotlight
Amid the roll-out of multiple new drug classes, which have been cited by payers as a threat to already unsustainable levels of US pharmaceutical expenditure, Novartis' heart failure treatment Entresto became the latest therapy to be value-assessed by the Institute for Clinical and Economic Review (ICER).
Similar to the American Society of Clinical Oncology's value framework (see Spotlight On Interview: Chief medical officer Richard Schilsky discusses ASCO's new cancer drug value framework), these assessments are designed to stimulate discussion rather than dictate policy. The report on Entresto indicates that pharma may be working towards a more constructive relationship with payers - ViewPoints: ICER finds Entresto priced more appropriately than anti-PCSK9 mAbs.
In determining a price benchmark some 17 percent below the $4560 list price set by Novartis in the US, the ICER report concludes that Entresto is priced within the appropriate range, when further discounts and rebates are taken into account.
Speaking to FirstWord earlier this week, Steve Miller – senior vice president and chief medical officer at the pharmacy benefit manager (PBM) Express Scripts – applauded Novartis for being proactive in its approach in pricing Entresto.
Citing a lack of push back from the PBM towards Entresto, Miller described Novartis' pricing of the drug as "reasonable." While – as the ICER report indicates – the cost of Entresto would ideally be lower, added Miller, Novartis has "worked to understand the medical offset," associated with the therapy.
This extends to Novartis' efforts to offer outcomes-based reimbursement for Entresto. Express Scripts continues to work with the company on developing an outcomes-based scheme, added Miller, but such models have historically been fraught with challenges. "With the infrastructure of these programmes often so expensive it negates the benefit of the model, we are looking for a system that is both fair to all parties and simple to operate," Miller confirmed.
Added gloss on the ICER's assessment of Entresto is provided by the inevitable comparisons which have been made to the Institute's recent assessment of the PCSK9 inhibitor class; the ICER valued a pricing benchmark of $2177 per year for Amgen's Repatha and Sanofi/Regeneron Pharmaceuticals' Praluent, versus the approximate $14,000 list price set for both drugs.
One can argue that with no direct competitors to Entresto on the horizon, Novartis is better positioned to concede ground on price knowing that the product is likely to enjoy sustainable market share gain for an extended period of years. The almost simultaneous launch of Repatha and Praluent has played into the hands of the PBMs, with Miller confirming that Express Scripts remains willing to grant exclusive preferred formulary status for one of the PCSK9 inhibitors in exchange for competitive discounting.
Amgen hedges its bets
An uncertain pricing environment is one of the factors that is making it difficult for analysts to forecast revenues for the PCSK9 inhibitors.
Another is the possibility that oral medications offering sufficient efficacy to warrant their use ahead of the PCSK9 inhibitors reach the market. Esperion Therapeutics' ETC-1002 is one such candidate, as are the CETP inhibitors anacetrapib and evacetrapib, which are being developed by Merck & Co. and Eli Lilly, respectively.
Amgen appears to be hedging its bets, having announced this week it is toacquire the privately-held, Netherlands-based Dezima Pharma, in a deal initially worth $300 million which could rise in value to $1.6 billion.
Dezima CEO Rob de Ree described lead compound TA-8995 (formerly known as DEZ-001) as a "best in class CETP inhibitor," to FirstWord last year based on Phase III data; his enthusiasm is somewhat validated by Amgen's deal, even if the purchase looks relatively cheap for a late-stage asset.
Price reflects in part the reputational damage suffered by the CETP inhibitor class from high-profile setbacks to therapies being developed by Roche and Pfizer. Furthermore, as analysts at Goldman Sachs note, while the deal both hedges against the risk of orals to PCSK9 market share and complements Amgen's ownership of Repatha, TA-8995 trails both anacetrapib and evacetrapib for which Phase III data is expected next year. With Phase III studies for TA-8995 yet to commence, analysts at UBS estimate Amgen's new drug could be four years behind the competition.
Esperion saw its share price close down 2 percent on Wednesday, presumably reflecting some disappointment among investors that Amgen had chosen to look elsewhere. Alternatively, argues UBS analyst Andrew Peters, the deal highlights the strategic value of ETC-1002 as an oral option, with Sanofi, Regeneron and Pfizer mooted as potential acquirers or in-licensers. Improved trading on Thursday – with Esperion's share price up 8 percent at one point – saw the Amgen deal being viewed in a more positive light.
Can Intarcia deliver some much needed innovation to the diabetes market
While a wealth of new clinical data has been presented this week at the European Association for the Study of Diabetes (EASD) meeting, short-to-medium term evolution of the diabetes market looks poised to be primarily shaped by the launch of new products demonstrating incremental benefit versus existing therapies, combinations and data emanating from cardiovascular outcomes studies.
Recent and forthcoming developments encapsulate this trend; focus is shifting rapidly to likely FDA approval of Novo Nordisk's basal insulin Tresiba by the beginning of October (a notable event given FDA rejection two years ago), analysts are speculating as to whether Sanofi will use a priority review voucher acquired earlier this year to expedite approval of its LixiLan combination and the standout data at EASD is expected to be full disclosure of the recently top-lined EMPA-REG OUTCOME study.
See ViewPoints: On back of new Phase III data will Sanofi choose to make LixiLan a priority? and Physician Views Poll Results: Jardiance demonstrates a cardiovascular benefit – what impact on the oral diabetes market?
As it nears potential launch in 2017, therefore, one may expect enthusiasm to grow around Intarcia's ITCA-650, which combines the GLP-1 agonist exenatide into a small, matchstick sized pump capable of delivering regular doses of the drug over periods of six or 12 months.
A Physician Views poll run by FirstWord earlier this year showcased notable enthusiasm for the approach among endocrinologists and Intarcia CEO Kurt Graves hopes payers and patients will share this view. Speaking to FirstWord this week, Graves outlined a commercial strategy for ITCA-650, which will be centred on the adherence advantage that an implantable device can offer over other therapies – see FirstWord next week for the full interview.
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