美国食品和药物监管局(FDA)1月1日晚宣布，2014年核准的新药数量为41种，创18年来新高，为历史第二高纪录，仅次于1996年的53种。
与此同时，欧洲药监局去年核准的新药(含新仿制药)数量为82种，超过2013年的79种和2012年的57种。
Specialty drug approvals: Review
of 2014 and a forecast for 2015
Specialty Pharmacy Section
The impact of specialty drugs continues to increase in both drug utilization and spend. In 2012, specialty drug spend accounted for approximately $87 billion, or about 3.1% of national health spend in the United States. In 2020, the forecasted specialty drug spend is expected to be $400 billion, or about 9.1% of national health spend. In addition, within 4 years, analysts predict that 7 of the top 10 drugs in terms of sales will be specialty products.1 Given this expected growth, the specialty drug pipeline will be of particular interest to patients, prescribers, payers, and pharmacies.
New specialty drug approvals
As of December 3, 2014, FDA had approved 39 new molecular entities and new therapeutic biologics this year. While there is no universal definition of a specialty drug, approximately 19 of this year’s approvals fit commonly used definitions of specialty products (Table 1).6,7 Of these 19, oncology and rare diseases represented the majority of approvals, a trend that is expected to continue through 2015.2
In the pipeline
OncologyLooking ahead to the remainder of 2014 and 2015, the pipeline is again forecasted to have the highest number of approvals in oncology, with approximately one dozen expected approvals for a variety of cancers (Table 2).3 Programmed cell death–1 (PD-1) drugs stand out as an especially promising class of therapy. Pembrolizumab (Keytruda—Merck) was the first of this class to earn FDA approval for the treatment of unresectable or metastatic melanoma. Pembrolizumab, along with fellow experimental PD-1 drug nivolumab (Opdivo—Bristol-Myers Squibb), have earned breakthrough designations from FDA and are likely to see success expanding into other indications.
Trials are currently under way with PD-1 agents for a number of cancers, including lung, liver, brain, and solid tumors. Other pipeline drugs are in late stages of development for the treatment of breast, melanoma, non–small cell lung, ovarian, and pancreatic cancers, as well as hematologic malignancies such as leukemia and multiple myeloma.3
Hepatitis CHepatitis C (HCV) continues to have one of the most intriguing pipelines of any disease state. Within the last year, the approvals of sofosbuvir (Sovaldi—Gilead) and simeprevir (Olysio—Janssen) as individual agents have led to major changes in the treatment of HCV. Gilead’s Harvoni, the combination of sofosbuvir and ledipasvir, was approved in October 2014. In addition, sofosbuvir and simeprevir used together was approved in November 2014. Both of these combination therapies show high sustained viral response rates and are interferon-free, oral regimens.
Competition should come from the AbbVie “3D” HCV combination therapy compose of ombitasvir, paritaprevir, and ritonavir (Viekirax) used in combination with dasabuvir (Exviera). An FDA decision for this combination is expected by December 22, 2014. In addition, Merck’s HCV combination of MK-5172 and MK-8742 is also showing good effectiveness in clinical trials.
Tough competition from fast-paced innovation in the HCV market has taken a toll on other pipeline agents. Faldaprevir, a late-stage drug from Boehringer Ingelheim, was abandoned. Bristol-Myers Squibb’s daclatasvir and asunaprevir, taken together as a two-drug combination therapy, was discontinued. However, studies continue with daclatasvir as a component of other drug combinations, including a three-drug combination of daclatasvir with asunaprevir and BMS-791325. A two-drug combination of daclatasvir paired with sofosbuvir is also being evaluated in clinical trials.3
ImmunologyIn the immunology pipeline, top agents of interest include the IL-17 class of drugs: secukinumab (Novartis), brodalumab (AstraZeneca, Amgen), and ixekizumab (Eli Lilly). These agents are showing promise particularly for the treatment of psoriasis. In clinical trials, some IL-17 inhibitors have demonstrated efficacy that is superior to etanercept (Enbrel—Amgen), a commonly used drug in the treatment of psoriasis. These drugs are also being investigated for the treatment of rheumatoid arthritis, ankylosing spondylitis, and psoriatic arthritis. Sarilumab (Sanofi—Regeneron) is an additional late-stage pipeline agent being evaluated for the treatment of rheumatoid arthritis.3
Rare diseasesOpportunities in an area with limited competition have made rare disease treatments an attractive category for many manufacturers. Examples of these types of products include the recently approved drugs for idiopathic pulmonary fibrosis, pirfenidone (Esbriet—InterMune) and nintedanib (Ofev—Boehringer Ingelheim), as well as the experimental drugs eteplirsen (Sarepta) and drisapersen (Prosensa), which are currently being studied for the treatment of Duchenne muscular dystrophy. These drugs treat diseases wher currently available therapies are limited.
Growing trend
This trend toward the development of rare disease treatments is expected to continue growing. As of 2013, more than 450 drugs were in various stages of development for rare diseases.4 Further evidence of the growth in rare disease drug development is shown by the fact that in 2010, of the top 100 drugs in the United States, 23 were for the treatment of diseases with 100,000 or fewer patients. In 2014, that number of rare disease drugs had increased to 41. Meanwhile, drugs targeting diseases with higher patient populations became less popular. In 2010, of the top 100 drugs, 55 treated diseases with 500,000 or more patients. By 2014, that total was reduced to 35 drugs.5
The pipeline is stocked with exciting treatments for many disease states. While we can’t be certain how many of these agents will earn FDA approval, the possibility of bringing patients better treatments makes the drug pipeline worth watching.
http://www.pharmacist.com/specia ... 4-and-forecast-2015
A blueprint for health?A surge in approvals and a record year for M&A led many investors to believe the pharmaceuticals industry is poised for strong growth
©Marco Betti
Three days before Christmas, as most of the US government slowed down for the holidays, regulators at the Food and Drug Administration were still going strong.
Bristol-Myers Squibb, the US pharmaceutical company, received FDA approval for a much-hyped medicine called Opdivo — one of the first in a new category of cancer drugs that harness the immune system to fight tumours.
It was the 41st time in the year that the FDA had given the green light to a new therapy — the highest tally since 1996 and well above the annual average of 26 over the past decade.
Not all the medicines generated as much excitement as Opdivo. Treatments for ear infections, insomnia and fungal infections of the toe were among other, less heralded advances for medical science in 2014.
But the surge of approvals has increased the confidence of those who believe the pharmaceuticals industry is entering a new era of growth. Scientific advances unleashed by the decoding of the human genome a decade ago are finally reaching commercial fruition, these optimists say, promising a resurgence in drug research and development after the innovation drought of recent years. “The science has never been better,” says Bahija Jallal, head of AstraZeneca’s Medimmune research and development unit. “The next five or 10 years are looking very exciting.”
While few would dispute that the pace of innovation has picked up, the outlook varies from one company to another — and many appear to be relying more on mergers and acquisitions than homegrown R&D to deliver new products. A record $250bn worth of deals was struck in 2014, and bankers and executives predict more this year as companies tap plentiful cash and cheap credit.
To bullish investors in pharma, the dealmaking is a sign of confidence from an industry rearming for new growth. But there are still plenty of sceptics who instead see desperate companies resorting to financial engineering to cover up a broken business model.
Which of these is closer to the truth? The answer matters not only to investors but also to societies around the world relying on pharmaceuticals to help meet the growing healthcare needs of an ageing population.
The bullish view rests on a belief that big pharma is beginning to generate more value from the tens of billions of dollars poured into research and development each year. According to a report by Deloitte last month, return on investment from R&D rose in 2014 for the first time since 2010, to 5.5 per cent, up from 5.1 per cent the previous year.
. . .
Improving productivity should help the industry to meet — and profit from — the rising demand for its products that is flowing from two big trends. The first is demographic: the number of over-65s on the planet is forecast to triple between 2010 and 2050 to 1.5bn, bringing with it a commensurate increase in age-related diseases such as cancer, heart disease and diabetes.
The second is economic: growing wealth in the developing world is increasing the ability of countries such as China and Brazil to provide high-quality healthcare — and increasing the demand from their burgeoning middle classes for access to modern medicines. Chinese per capita spending on pharmaceuticals is forecast to grow by more than 75 per cent in the next five years, according to the IMS Institute for Health Informatics. This will contribute to a 30 per cent increase in global spending on medicines to $1.3tn over the same period, IMS predicts.
“From a commercial business point of view you couldn’t be in a better industry,” says Severin Schwan, chief executive of Roche. Most investors appear to share his optimism: the S&P pharmaceuticals index is up 29 per cent in the past year. So why are some people unconvinced?
Producing new medicines remains among the most costly, protracted and risky ventures in global business. only about 7 per cent of experimental drugs in early-stage trials reach the market. The average development cost of those that make it is $2.6bn, according to the Tufts Center for the Study of Drug Development in Boston. This is three times the previous Tufts estimate in 2003, reflecting the greater cost of developing the new generation of biological medicines that are supplanting less complex chemical-based pills.
Sceptics of the pharma renaissance story question whether drug companies will be able to sustain the high prices needed to keep profits up. Instead of seeing an ageing population as a growth opportunity, they fear it will squeeze the industry as overburdened health systems battle to contain costs.
Disputes with publicly-funded European providers, such as the UK’s National Health Service, over access and pricing for new medicines are already commonplace. Last year’s high-profile backlash by US insurers and politicians against the price of new breakthrough treatments for hepatitis C — particularly Gilead Sciences’ $1,000-a-day Sovaldi — suggests that America’s bloated health system is also becoming more cost-sensitive.
. . .
China, too, is trying to keep a tight lid on prices in a bid to make its expanding health budget stretch further. India, meanwhile, has taken an aggressive approach to challenging drug patents in hopes of widening access to cheap generic medicines for the country’s poor. If such a stance were replicated by other big developing countries it could weaken the foundations of the intellectual property framework on which the industry’s economic model is based.
“This industry is financially falling apart because we do not know how to discover drugs in an affordable way,” says Chas Bountra, professor of translational medicine at the Nuffield Department of Medicine at Oxford university.
This gloomy view seems at odds with the average 67 per cent profit margin reported by the 10 biggest pharma companies in 2013. However, this was down from 74 per cent a decade earlier because of patent expiries and pricing pressure, according to Lloyds Banking Group. Companies have shielded shareholders from this squeeze by cutting operating costs and increasing dividends and share buybacks. “Shareholders are expecting growth and a return of cash, which is like having your cake and eating it,” says Philipp Gutzwiller, head of healthcare at Lloyds. “My question is how long will this be sustainable.”
A closer inspection of the 41 new medicines approved by the FDA this year provides further cause for caution. Fewer than half came from traditional pharma groups. The rest were developed by the smaller biotech companies that increasingly provide the strongest engine of industry innovation.
Even those drugs brought to market by big pharma often originate from smaller companies. According to Deloitte, almost 60 per cent of forecast revenues from the late-stage R&D pipelines of the 12 biggest drugmakers involve assets brought in through acquisitions or partnerships. “There is an inverse correlation between scale and innovation,” says Julian Remnant, head of Deloitte’s European R&D advisory practice. “If 60 per cent of your innovation is coming from outside the company do you really need so much fixed infrastructure?”
These pressures explain the sense of upheaval in pharmaceuticals as drugmakers scramble to restructure. Companies have been making choices about which diseases and product areas to focus on — and how much R&D to keep in-house and how much to outsource. This has spurred the rush of dealmaking as non-core assets are offloaded and others acquired to bolster innovation. These decisions will help determine pharma’s winners and losers in the decades ahead. As for the industry as a whole, Mr Remnant says: “We see some signs that a new era of growth could be on the horizon but it would be premature to declare victory.”
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US
Companies: Johnson & Johnson, Pfizer, Merck & Co, Gilead, Abbott, Eli Lilly, Amgen, AbbVie, Bristol-Myers Squibb
Outlook: The fortunes of US big pharma are as diverse as the companies themselves — from old guard Pfizer, Merck and Eli Lilly battling to refresh their product pipelines to fast-growing young biotech companies. Standard-bearer for this latter group is Gilead Sciences, whose hepatitis C medicine, Sovaldi, broke industry records in 2014 by generating about $10bn of sales in its first year.
2014 deals: Merck bought biotech companies cubist and Idenix for a combined $13.4bn; Botox-maker Allergan was bought by Dublin-based Actavis for $66bn in the biggest pharma deal of the year.
Pipeline: Merck and Bristol-Myers Squibb are racing for leadership of a new category of cancer drugs, called PD-1 checkpoint inhibitors, which boost the immune system’s ability to attack tumours.
UK
Companies: GlaxoSmithKline, AstraZeneca
Outlook: AstraZeneca has long been junior partner to GSK in Britain’s pharma industry. But, in a reversal of recent fortunes, it is AstraZeneca whose R&D pipeline is showing greatest promise. GSK is battling to stem decline in its core respiratory drugs business as prices come under pressure in the US, while facing fallout in China from a damaging corruption scandal.
2014 deals: AstraZeneca repelled a hostile $117bn takeover from Pfizer; GSK agreed a $20bn asset swap with Novartis to boost its vaccines and consumer healthcare businesses.
Pipeline: Much of the optimism around AstraZeneca stems from an experimental cancer medicine, called Medi4736, which is in late-stage trials with the company targeting peak annual sales of up to $6.5bn.
Switzerland
Companies: Novartis, Roche
Outlook: Situated on opposite sides of the river Rhine in basel, Switzerland’s rival pharma superpowers are among the industry’s best-performers. Roche has focused on two markets — oncology and diagnostics — and dominates both. Novartis is also strong in cancer as well as cardiovascular medicines, eyecare and generic drugs.
2014 deals: Roche agreed to pay $8.3bn for InterMune, a US biotech company whose main drug treats a previously incurable lung disease. Novartis sold its sub-scale animal health unit to Eli Lilly while trading its vaccines business for GSK’s cancer business.
Pipeline: A new Novartis heart drug, called LCZ696, looks set to be one of the biggest launches of 2015, with analysts forecasting peak annual sales of up to $10bn.
France
Company: Sanofi
Outlook: France’s sole representative at the top table of global pharma is facing turbulent times. Christopher Viehbacher was fired as chief executive in October after a downturn in its core diabetes business and a falling out with chairman Serge Weinberg, who has taken temporary charge.
2014 deals: Sanofi has steered clear of the M&A frenzy but a dispute with the board over an abortive plan to sell some of the company’s older drugs helped seal Mr Viehbacher’s fate.
Pipeline: New products are finally arriving to replac those such as its Plavix blood thinner and Ambien sleeping pill which have lost patent protection. Sanofi predicts an “unprecedented” period of product launches with up to 18 new drugs by 2020, including the first vaccine for dengue fever and a new treatment for high cholesterol.
Germany
Companies: Bayer, Merck KGaA
Outlook: Germany’s pharma groups are sometimes overlooked because they form parts of broader industrial groups. Yet, they are two of the world’s oldest drugmakers and remain important players. Bayer, the larger of the pair, has invested heavily in emerging markets, building a top four position in China. Its sprawling healthcare division ranges from glucose monitoring meters to animal medicines.
2014 deals: Bayer is planning to spin off its plastics business to focus on healthcare and crop science. It paid $14.2bn for the consumer business of US-based Merck & Co, which shares a name with its German counterpart but is a separate company. Merck of Germany paid $17bn for US speciality chemicals company Sigma-Aldrich.
Pipeline: Bayer has 18 medicines in late-stage trials but Merck, without a major drug launch since 2003, is still struggling to revive its pipeline.
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