Nine months after U.S. House Energy & Commerce Committee chairman Rep. Fred Upton (R-Mich.) launched the 21st Century Cures initiative as a bipartisan effort, the committee released a discussion draft Tuesday without support from Democrats. Republicans can pass legislation in the House, but would need bipartisan support to pass a bill through the Senate.
Sen. Lamar Alexander (R-Tenn.), chairman of the Senate Health, Education, Labor and Pensions Committee, said in a statement that one of the HELP committee's "top priorities this year will be modernizing the Food and Drug Administration, helping to get treatments and medicines to patients as quickly and as safely as possible." Alexander added that he plans to engage in a bipartisan effort that will "have legislation to the President by the end of this year."
Rep. Diana DeGette (D-Colo.) joined with Upton in April 2014 to announce the initiative and expressed support for the project at over a dozen public meetings. "While I don't endorse the draft document, I know that with continued engagement, we can reach a bipartisan consensus to help advance biomedical research and cures," she said in a press release on Tuesday. She did not specify areas of disagreement.
Rep. Frank Pallone, Jr. (D-N.J.), the ranking Democrat on the Energy & Commerce Committee, said in a statement that he is "disappointed that the discussion document released today by Chairman Upton does not reflect true bipartisan collaboration." Pallone added that the "nearly 400 page draft could create more problems for our health care system than it solves." While he did not cite any specific concerns, he did note that the draft "does not include any real dollars to fund additional basic research at the National Institutes of Health."
Some provisions in the discussion draft are likely to be sticking points for Democrats, including proposals to exert more centralized control over NIH funding decisions by setting fixed, renewable four-year terms for institute directors, and making directors personally responsible for ensuring that the goals of every grant award are consistent with "a national priority and have public support."
FDA is likely to oppose scores of provisions in the discussion draft that would impose new mandates but do not provide additional funding. For example, the draft would require FDA and HHS to produce about 35 draft or final guidance documents, most of them on tight deadlines.
The committee left placeholders in the draft for several topics that are potential political flash points, such as communication about off-label uses of approved products, and regulation of diagnostics. (For more on 21st Century Cures, see Politics & Policy below.)
The Sandoz unit of Novartis AG (NYSE:NVS; SIX:NOVN) filed a motion in the U.S. District Court for the Northern District of California seeking an order to dismiss patent infringement and related claims from Amgen Inc. (NASDAQ:AMGN) related to Sandoz's application to market Zarxio (EP2006), a biosimilar of Amgen's neutropenia drug Neupogen filgrastim recombinant methionyl human granulocyte colony-stimulating factor (G-CSF).
In July, Sandoz announced FDA had accepted the company's BLA for Zarxio, making it the first disclosed biosimilar candidate to reach that regulatory milestone in the U.S.
In October, Amgen filed a lawsuit and a Citizen's Petition alleging that Sandoz refused to follow the patent resolution protocol laid out by the Biologics Price Competition and Innovation Act of 2009 (BPCIA) when it filed the BLA for Zarxio (see BioCentury Extra, Oct. 30, 2014).
BPCIA stipulates that a biosimilar applicant provide the reference product sponsor with a copy of the BLA and manufacturing information for its biosimilar within 20 days of FDA's acceptance of the application for review. The procedure is designed to identify and resolve potential patent disputes before a biosimilar is approved and commercialized. In its Citizen's Petition to FDA, Amgen alleged that Sandoz said it views the patent resolution procedure as "optional" (see BioCentury, Nov. 3, 2014).
In its motion filed last Friday, Sandoz argued BPCIA does not require it to supply its BLA to Amgen, and that Sandoz decided that it should not share the BLA with a competitor. Rather, said Sandoz, it chose to run the risk that Amgen would file a patent infringement suit, as is permitted under BPCIA and which Sandoz concluded Amgen would not win.
Sandoz also argued that it properly notified Amgen of its intent to market the biosimilar at least 180 days before it would be launched.
The company also stated that Amgen's suit "is inconsistent with the goals of the BPCIA" and would "cause unwarranted delay in providing lower-cost, effective drugs to cancer patients, with no countervailing benefits."
Sandoz is seeking an order declaring the company complied with BPCIA. Furthermore, it is seeking an order to dismiss Amgen's unfair competition and conversion claims against Sandoz, which hinge on Amgen's interpretation of BPCIA. The court is scheduled to hear the case March 12.
Earlier this month, an FDA advisory committee voted 14-0 to recommend approval of Zarxio. The product was the first biosimilar to come before an advisory committee (see BioCentury Extra, Jan. 7).
Steve Miller, SVP and CMO of Express Scripts Holding Co. (NASDAQ:ESRX), wrote in a blog post that the PBM's deal last month with AbbVie Inc. (NYSE:ABBV) covering HCV treatment Viekira Pak will save its clients "more than $1 billion this year" on HCV treatments.
Miller said the "industry-wide ripple effect" of its decision will save the U.S. healthcare system more than $4 billion in 2015. Several PBMs and payers have reached agreements with AbbVie and Gilead Sciences Inc. (NASDAQ:GILD), which markets its Harvoni ledipasvir/sofosbuvir and Sovaldi sofosbuvir, in which the drugmakers provide discounts for their HCV treatments in exchange for exclusive or preferred formulary status.
In December, Express Scripts granted exclusive status to AbbVie's Viekira Pak to treat HCV genotype 1 in exchange for undisclosed rebates on the treatment. It is the only PBM to disclose an exclusive deal for Viekira Pak, while several other payers and PBMs have selected Gilead's drugs (see BioCentury, Jan. 19).
Express Scripts spokesperson Brian Henry said the company is not disclosing the number of patients on which the $1 billion savings estimate is based.
Henry said that Miller arrived at the $4 billion estimate because Express Scripts has "visibility" into the deals made between other payers and AbbVie and Gilead. The estimate assumes that 170,000 patients will be treated in 2015, he said.
Dividing the $4 billion in savings over 170,000 patients suggests an average savings per patient of $23,529. That number would represent a 28% discount to the wholesale acquisition cost (WAC) for a 12-week course of Viekira Pak, which is $83,319, and a 25% discount to that of Gilead's Harvoni, which is $94,000 for 12 weeks.
Henry said Express Scripts is unaware of any other payer that has negotiated a better rate on the Abbvie or Gilead drugs than it has.
Viekira Pak includes a fixed-dose combination of 25 mg of ombitasvir (ABT-267), 150 mg of paritaprevir (ABT-450), and 100 mg of the booster Norvir ritonavir dosed once daily; and 250 mg of dasabuvir (ABT-333) dosed twice daily. Harvoni is a once-daily, fixed-dose combination of 90 mg ledipasvir, an HCV NS5A protein inhibitor, and 400 mg sofosbuvir, a nucleotide analog HCV NS5B polymerase inhibitor.
Biogen Idec Inc. (NASDAQ:BIIB) and the Google X Life Sciences innovative research unit of Google Inc. (NASDAQ:GOOG) have established a collaboration to study the symptoms and progression of multiple sclerosis (MS). The two are planning a study that will use Google's sensor platforms and bio-analytical tools to monitor patients and look for clues that can predict patient outcomes. The companies did not disclose terms of the collaboration or details of the study.
In a statement, the companies said researchers seeking clues about the differences in how MS affects individual patients have access to only a limited amount of data, often gathered via self-reporting or during office visits. They suggested that tracking tools that monitor patients continuously and provide information about characteristics of patients' immune systems, gait or sleep patterns might lead to insights that improve disease management.
Last year, Google partnered with Novartis AG (NYSE:NVS; SIX:NOVN) to develop contact lenses that can monitor glucose levels in diabetics (see BioCentury Extra, July 15, 2014).
Zosano Pharma Corp. (NASDAQ:ZSAN) gained a penny to $11.01 in its first day of trading after it raised $49.5 million in an IPO on NASDAQ through the sale of 4.5 million shares at $11. The price valued Zosano at $130 million, including shares purchased by Eli Lilly and Co. (NYSE:LLY) in a concurrent $15 million private placement at the IPO price. Ladenburg Thalmann and Roth Capital Partners are underwriters. Zosano initially proposed to raise up to $65 million in its offering last June.
In November, Zosano granted Lilly exclusive, worldwide rights to commercialize its Daily ZP-PTH patch, a microneedle-based treatment for osteoporosis in women. The parathyroid hormone (hPTH 1-34) transdermal microprojection delivery system has completed Phase II testing.
The U.S. House Energy & Commerce Committee released a discussion draft Tuesday of 21st Century Cures legislation that proposes sweeping changes to FDA regulation of drugs, medical devices, clinical research and reimbursement.
The draft includes a provision allowing FDA to approve a drug with breakthrough designation on the basis of a single Phase II study, contingent on postmarket clinical trials and/or data from observational studies and registries. FDA could withdraw breakthrough approvals if sponsors failed to provide the required data, if evidence emerged showing the drug was not safe or effective, or if the sponsor disseminated false or misleading promotional material.
The committee included provisions creating tight deadlines for FDA to assess applications for qualification of biomarkers and other surrogate endpoints. The draft also attempts to extend and formalize FDA's efforts to incorporate patient experiences and preferences into regulatory decisions, including using patient data in structured benefit-risk assessments.
The draft incorporates, in whole or in part, a number of bills that were introduced but not passed over the last two congressional sessions. It includes the Antibiotic Development to Advance Patient Treatment (ADAPT) Act, which would give FDA authority to approve antibiotics and antifungals for limited populations based on a smaller number of patients or alternative endpoints, as well as data from Phase II trials or pharmacologic or pathophysiologic data (see BioCentury Extra, Dec. 12, 2013).
It also incorporates legislation on compassionate access introduced by Michael McCaul (R-Texas), as well as the Modernizing Our Drug & Diagnostics Evaluation and Regulatory Network (MODDERN) Cures Act (see BioCentury Extra, Dec. 8, 2014 and BioCentury Extra Sept. 11, 2013).
The discussion draft has several provisions intended to make CMS decision-making more transparent and consistent, including a process for device and drug sponsors to appeal coverage decisions.
The U.K.'s NICE is relying on requirements around dose adjustments and stopping rules for clinical reasons to rein in costs to NHS of use of the rare disease drug Soliris eculizumab from Alexion Pharmaceuticals Inc. (NASDAQ:ALXN). In its final guidance, NICE recommended use of the humanized mAb against complement 5 (C5) to treat atypical hemolytic uremic syndrome (aHUS).
NICE had objected to the drug's high cost, yet in its appraisal admitted that Soliris provided significant clinical benefit. Since NICE's Highly Specialised Technology (HST) appraisal process does not evalute cost effectiveness, the agency had tried to force Alexion to justify the Orphan drug's price by providing information on Soliris' R&D costs -- information Alexion declined to share (see BioCentury, March 17, 2014).
The final guidance stipulates that Soliris use be coordinated through an expert center; that monitoring systems be established to record the number of aHUS patients diagnosed and treated with Soliris and the dose and duration of treatment; and that a national protocol be created for starting and stopping Soliris therapy for clinical reasons. The guidance also requires Alexion to conduct a research program to develop methods to evaluate when stopping treatment or a dose adjustment might occur.
The recommendations are in line with NICE's final evaluation determination (see BioCentury Extra, Dec. 1, 2014).
NICE estimates that Soliris will cost the NHS up to L58 million ($87.1 million) in the first year, rising to L82 million ($123.1 million) after five years. Soliris is the first Orphan drug NICE has evaluated since it took over responsibility for assessing high-cost drugs for rare diseases in 2013.
NHS England already covers Soliris for its other approved indication, paroxysmal nocturnal hemoglobinuria (PNH). Alexion lost $2.40 to $180.36 on Tuesday.
Not-for-profit biopharma consortium TransCelerate BioPharma Inc. (Philadelphia, Pa.) added Merck & Co. Inc. (NYSE:MRK) and Novo Nordisk A/S (CSE:NVO; NYSE:NVO) as member companies. Merck acquired TransCelerate member Cubist Pharmaceuticals Inc. last week.
The consortium also named as its chairman Briggs Morrison, who is EVP of global medicines development at AstraZeneca plc (LSE:AZN; NYSE:AZN). He succeeds Annalisa Jenkins, who became chair in February 2014 but stepped down last year after leaving the Merck Serono unit of Merck KGaA (Xetra:MRK), a TransCelerate member.
TransCelerate was founded in 2012 after a group of R&D heads concluded that a precompetitive collaboration was the best way to solve common R&D challenges and clinical trial bottlenecks. All 10 of TransCelerate's founding companies remain members (see BioCentury, Oct. 21, 2013).
In addition to Merck & Co., Merck Serono and Novo Nordisk, other non-founding members include Actavis plc (NYSE:ACT), Allergan Inc. (NYSE:AGN), Astellas Pharma Inc., Biogen Idec Inc. (NASDAQ:BIIB), Medgenics Inc. (NYSE-M:MDGN), Shionogi & Co. Ltd. (Tokyo:4507) and UCB Group (Euronext:UCB).
Former members Onyx Pharmaceuticals Inc. and Braeburn Pharmaceuticals S.p.r.l. (Princeton, N.J.) are no longer participating. Amgen Inc. (NASDAQ:AMGN), which is not a member, acquired Onyx in 2013.
The White House announced Tuesday that its FY16 budget proposal will include a request to nearly double spending on combating and preventing antibiotic resistance.
The $1.2 billion request would include an increase of more than $100 million for NIH, including funds for "an antibacterial resistance clinical trial network for rapid testing of new drugs to treat multi-drug resistant bacteria."
The Biomedical Advanced Research and Development Authority (BARDA) would receive an increase of over $100 million for antibiotic drug and diagnostic development. FDA would receive $47 million for antibiotics, including funding "to support evaluation of new antibacterial drugs for patient treatments and antibiotic stewardship in animal agriculture."
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