Spurred by the progress of a bill moving through Congress that will speed new drugs to market for conditions lacking cures, called The 21st Century Cures Act, the FDA has scheduled a public meeting this summer to address concerns by the drugs industry that regulatory restrictions on what they can say about the off-label use of their products violates their First Amendment right to free speech. Language in the bill is ratcheting up the pressure on the agency to relax its guidelines.
Drug makers were emboldened on its prospects of changing the rules after an appeals court overturned the conviction of a pharmaceutical sales representative in 2012 who was convicted of promoting off-label uses of the narcolepsy drug Xyrem. The court ruling was based on First Amendment protection for truthful and non-misleading off-label speech.
The American Medical Association supports the need for physicians to have access to accurate and unbiased information about off-label uses of drugs since it already accounts for as much as 20% of prescribing, with higher levels in oncology and pediatric rare diseases.
Giving drug firms more leeway in this arena has its detractors, however. Rita Redberg, M.D., Professor of Medicine at the UC-San Francisco says, "At my own medical center we have banned pharmaceutical reps from coming because we don't think they are a good source of information. You don't ask the barber if you need a haircut."
Pharma companies have a dubious history of breaking the rules governing off-label promotion. Over the past 10 years, 17 firms have paid more than $16B in settlements related to inappropriate off-label selling.
Further complicating matters is the fact that up to 75% of published pre-clinical trial results cannot be reproduced in later studies.
Drug manufacturers have long defended their pricing by citing the need to recoup ultra-high development costs. Now another price-raising scheme is drawing attention. In a report by the Wall Street Journal, there are a growing number of examples where companies acquire drugs they perceive as undervalued and then significantly raise the prices.
In February, Valeant Pharmaceuticals (VRX -1.5%) purchased the rights to two heart drugs, Isuprel and Nitropress, from privately-held Marathon Pharmaceuticals for an undisclosed sum. It then immediately raised the prices 525% and 212%, respectively, based on the recommendations of a consultant who researched the price/benefit of each. According to Truven Health Analytics, the cost of a 1mL vial of Isuprel went from $215.46 to $1,346.62 while a 2mL vial of Nitropress went from $257.80 to $805.61.
Last year, Mallinckrodt (MNK -0.7%) bought Cadence Pharmaceuticals to gain access to its Ofirmev pain meds. A short time later, the list price for a package of 24 vials jumped 2.5-fold to over $1,019.52.
In 2013, Horizon Pharma increased the price of Vimovo pain tablets, after acquiring the rights from AstraZeneca, almost 600% to $959.04.
Valeant is apparently leading the price-raising pack. It has boosted list prices by at least 20% about 122 times since early 2011.
According to the IMS Institute for Healthcare Informatics, U.S. spending for prescription drugs rose 13% last year to $374B, driven by manufacturer price hikes, pricey breakthrough medicines and millions of newly insured individuals under the Affordable Care Act. The increase was the largest since 2001. Total spending rose by $43.4B, including $10B from price increases and $20.3B from new drugs. led by Gilead Sciences' hepatitis C drug Sovaldi. Specialty medicines, those for complex, chronic disorders, accounted for one third of spending.
A record 4.3B prescriptions were filled last year. Those covered by Medicaid rose by almost 17% which accounted for 70% of the growth in prescriptions filled at retail pharmacies. 8.4M fewer prescriptions were filled at retail pharmacies compared to 2013 for patients with commercial insurance due to high out-of-pocket costs.
The number of novel medicines launched in 2014 was 42, including 18 for rare diseases. Ten were designated as Breakthrough Therapies by the FDA.
Generic drugs reduced spending by only $11.9B.
The IMS data is based on list prices for branded drugs. Wholesalers usually get discounts of ~6%.
The FDA will hold a public hearing April 20 - 21 seeking information and comment on the use of products labeled "homeopathic" and the agency's regulatory framework for such products. The hearing will cover prescription drugs, biological products and over-the-counter (OTC) medicines labeled homeopathic, a multi-billion dollar industry in the U.S.
There has been considerable debate over the years concerning whether homeopathic medicines actually work. Recently, an Australian government study concluded that they do not. Last week, the FDA issued a warning about the potential health risks of homeopathic OTC asthma products considering they have not been evaluated for safety and effectiveness.
Homeopathy is considered pseudoscience. No remedy has ever been proven to be more effective than placebo.
The European Commission approves Boehringer Ingelheim's Vargatef (nintedanib), in combination with docetaxel, for the treatment of adult patients with locally advanced, metastatic or locally recurrent non-small cell lung cancer (NSCLC) of adenocarcinoma tumor histology, after first-line chemotherapy. Adenocarcinoma is the most common form of lung cancer.
Nintedanib is an oral angiokinase inhibitor which simultaneously inhibits endothelial growth factor receptors (VEGFR), platelet-derived growth factor receptors (PDGFR) and fibroblast growth factor receptors (FGFR) signalling pathways. It is currently being investigated in various other cancers.
Earlier this month, Europe's Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion supporting approval of nintedanib, branded as Ofev, for the treatment of ideopathic pulmonary fibrosis (IPF). The FDA approved it for IPF in October.
Chinese authorities are reportedly considering letting market forces determine the prices of most drugs sold there according to a proposal sent to drug industry groups by the National Development and Reform Commission, although measures would be implemented to prevent prices from spiking when the changes take effect January 1, 2015.
Currently, the government contains costs via price controls and state-run tenders. Under the new plan, prices for branded drugs would determined via multiple party negotiations. No further details are provided as to how the process would work, though.
Price controls would remain in place for certain meds such as anesthesia drugs because their production and circulation are strictly regulated. For medicines covered under the government's health insurance system, payment standards would be implemented to guide the market.
Pharmacy benefits manager Express Scripts (NASDAQ:ESRX) is at the forefront of an increasing wave of resistance to the high prices of new drugs from pharma and biotech firms, some which cost as much as $50K per month. Pharmaceutical spending in the U.S. is $270B and may breach $500B in five years. ESRX's method of controlling costs is to refuse to pay for them. For 2015, for example, it is excluding 66 branded drugs from its main formulary, an increase of 18 from 2014's 48. On the list is Johnson & Johnson's (NYSE:JNJ) rheumatoid arthritis drug Simponi (golimumab) which costs $3K per month.
Other prescription benefits managers are employing similar tactics. CVS Health (NYSE:CVS) will exclude 95 drugs from its 2015 formulary including Pfizer's (NYSE:PFE) multiple sclerosis med Rebif (interferon beta-1a) which costs $5K for a four-week supply.
Governments are pushing back as well. Among 42 state Medicaid programs, 27 pay for Gilead Sciences' HCV med Sovaldi (sofosbuvir) only for patients with severe liver damage while others impose coverage limitations for patients with recent substance-abuse problems. In the U.S., the full regimen cost is $84K. Recently, Britain's National Institute for Health and Care Excellence (NICE) balked at recommending reimbursement for Roche's (OTCQX:RHHBY) blood cancer drug Gazyvaro (obinutuzumab).
Ninety percent of commercial health plans require pre-approval of specialty drugs, up from 82% in 2011.
CB Pharma Acquisition Corp. (CNLMU) files for an IPO of 4M units priced at $10 per unit. Each unit consists of one share of common stock, one right to purchase 1/10 of a share of common stock on the consummation of an initial business combination and one warrant to purchase 1/2 of one share of common stock at a full-share price of $11.50.
The firm is a blank check company formed by Coronado Biosciences (CNDO +0.5%). It will seek business combinations via mergers, share exchanges, asset acquisitions, share purchases, recapitalizations or reorganizations.
It has 18 months from the consummation of this offering to complete its initial business combination (up to 21 months in certain circumstances) or it will liquidate the trust account, distribute the proceeds to shareholders and dissolve.
Per NASDAQ listing rules, the target business for the initial combination must have a fair market value equal to at least 80% of the value of the trust account.
The Cystic Fibrosis Foundation sells the royalty stream it receives from Vertex Pharmaceutics (NASDAQ:VRTX) to Royalty Pharma for $3.3B. It plans to reinvest the money into new research efforts.
The transaction is an example of successful mission investing, a growing trend in philanthropy. Instead of giving charitable grants, the foundation acts as a business partner by making an investment and expecting a share of the profits.
Vertex's Kalydeco (ivacaftor), which helps CF patients with a rare genetic mutation, costs more than $300K per year. CF Foundation Chief Robert J. Beall says, "While the drug's high price may have boosted the royalties the foundation gets, we would give up our royalties in a second to drive down the prices. We were not at the pricing table." He adds that the foundation has no plans to subsidize the cost of drugs to patients. "What we're really excited about is the move to a one-time cure." At present, the meds are daily therapies.
According to IMS Health (NYSE:IMS) global drug spending will top $1T this year led by Gilead's (NASDAQ:GILD) Sovaldi (sofosbuvir) for hep C and new cancer drugs. The projected spend of $1.06T represents a 7% increase from last year.
By 2018, drug spending is projected to rise to $1.3T. Hepatitis C and cancer meds will add ~$100B each, while diabetes care will contribute $78B. Leading the charge in cancer will be Roche's (OTCQX:RHHBY) Perjeta (pertuzumab) and Kadcyla (ado-trastuzumab emtansine), Pharmacyclics' (NASDAQ:PCYC) Imbruvica (ibrutinib) and Amgen's (NASDAQ:AMGN) Kyprolis (carfilzomib).
The ever-increasing tab for meds will be controversial because payers' budgets are being stressed while drug developers defend their prices citing high development costs.
There is already some push back in cancer. Britain's National Institute for Health and Care Excellence (NICE), which sets guidelines for the National Health Service on costs, procedures and technologies, is balking at approving Roche's Gazyvaro (obinutuzumab) and Kadcyla citing their high cost relative to additional benefits over existing therapies.
The contract research organization provides clinical development services (Phases 1 - 4) to the biotech and pharmaceutical industries. Since 2000, the company has performed ~2,300 clinical trials.
KKR, one of the underwriters, acquired the firm in September, 2013 for $1.4B. KKR PRA Investors, L.P., will own ~64% of the stock after the IPO, ~59% if the underwriters' over-allotment is fully exercised.
The financials are difficult to compare due several transactions executed by KKR.
To assuage a nervous public, President Obama presented his plan yesterday for containing a potential outbreak of Ebola in the U.S. despite the fact that there are only three infected persons in the country.
Rapid response teams will now be on-site within 24 hours of any new Ebola diagnosis in the U.S. Also, CDC officials are reviewing every step taken by the medical staff at Dallas Presbyterian Hospital who cared for Thomas Eric Duncan to find out how the two nurses became infected. Doctors there misdiagnosed Mr. Duncan and sent him home after his initial visit. He was correctly diagnosed after returning to the ER in an ambulance.
The President said, "We are taking this very seriously at the highest levels of government. We are going to be able to manage this particular situation, but we have to look forward to the future."
A 40-subject clinical trial to evaluate an experimental Ebola vaccine commences in Mali. The vaccine, which uses an modified adenovirus vector, was developed by investigators at the Vaccine Research Center of the National Institute of Allergy and Infectious Diseases in Bethesda, MD. GlaxoSmithKline (GSK -2.1%) Biologicals is the manufacturer.
The trial began yesterday with the vaccination of the first subject, a Malian health care worker. Two more health care workers will be dosed today.