While it's far from dead on arrival, the proposed 21st Century Cures Act that seeks to streamline the development and approval of new drugs and devices in the U.S. could be waylaid by the clamor over escalating drug prices.

Citing Mylan NV's Epipen (epinephrine auto-injector) as the latest example of industry price gouging, several labor unions, advocacy groups and public watchdogs are calling on lawmakers to hold off on passing the bill until it includes steps to rein in the cost of prescription drugs. Congressional sponsors of the legislation, which also would provide more money for NIH-funded research, are pushing to get it passed during the lame duck session that follows the November election.

But moving forward this year would be a missed opportunity to address unaffordable drug prices, the organizations said in a letter to Democrat leaders in the House and Senate. "There is no justification for moving forward with legislation that provides substantial benefits to the drug industry without asking for something in return," according to the letter, which was signed by 13 groups, including the Center for American Progress, AFL-CIO and Doctors for America.

Whether pressure from such groups will delay the bill remains to be seen. At a news conference last week, Rep. Nancy Pelosi (D-Calif.) voiced support for Cures, adding that some Democrats in the House feel differently. "But if the leadership takes it up, I think it will pass," she said.

Fred Upton (R-Mich.), chairman of the House Energy and Commerce Committee, which shaped the bill, remains confident that Cures will pass in the lame duck session, as support for the legislation has continued to grow. To date, more than 700 organizations encompassing patient advocacy groups, rare disease groups, cancer centers, technology groups, universities, biopharma companies and device makers have rallied behind the bill, according to the committee.

"We're closing in on what's truly a once-in-a-generation, transformational opportunity to change the way we treat disease," Upton said.

Meanwhile, government scrutiny of all the players in the drug pricing chain is intensifying. In filing its third quarter earnings this week, pharmacy benefit manager (PBM) Express Scripts reported it has received a "civil investigative demand" from the U.S. Attorney for the Southern District of New York for information regarding its relationships with biopharma manufacturers and prescription drug plan clients, as well as payments made to and from those companies. PBMs serve as go-betweens for insurance companies and drug companies, setting formularies, negotiating rebates and discounts, etc.

In a separate Justice Department inquiry, the PBM has been asked for information about relationships among drug manufacturers, specialty pharmacies and independent charitable foundations providing cost-sharing assistance to beneficiaries of federal health care programs.

Express Scripts, the largest PBM in the country, reported $722.9 million in net income for the third quarter, a 9 percent increase over the $661.7 million it generated last year.

While some members of Congress have called for hearings about the role PBMs play in drug pricing, a bill that would force transparency standards on PBMs has been stalled in several House committees since early last year. Sponsored by Rep. Doug Collins (R-Ga.) with bipartisan support, H.R. 244 would impose the standards on PBMs involved in Medicare Part D and Tricare programs.

In another effort to tackle pricing issues in the short lame duck session, Sen. Chuck Grassley (R-Iowa), chairman of the Senate Judiciary Committee, will convene a committee hearing Nov. 30 on the reported $465 million settlement between Mylan and the Justice Department over the Canonsburg, Pa.-based company's misclassification of Epipen as a generic for Medicaid rebate purposes. (See BioWorld Today, Oct. 11, 2016.)

Mylan announced the settlement earlier this month, but Justice has been mum about the details. "Americans deserve to know what the government is doing to hold Mylan accountable, recoup lost tax dollars and prevent similar behavior in the future," Grassley said Tuesday when he scheduled the hearing.

Besides scrutinizing the settlement, the hearing will explore whether the government is using its authority to prevent drug companies from misrepresenting their products for federally funded programs and how it holds offending companies accountable, Grassley said.

Cuban vaccine comes to U.S.

As tangible proof of the thaw between Cuba and the U.S., the FDA approved the first U.S. clinical trial of a Cuban therapy.

The three-year trial of Cimavax-EGF, a lung cancer vaccine, will begin next month at the Buffalo, N.Y.-based Roswell Park Cancer Institute as part of a joint venture between Roswell Park and the Havana-based Center of Molecular Immunology (CIM). The study is expected to enroll 60 to 90 patients.

The joint venture is licensed to undertake research, development and commercialization of biomedical technology and will foster expanded collaboration between the two medical centers, according to New York Gov. Andrew Cuomo, who announced the clinical trial Wednesday.

More than 4,000 lung cancer patients in global clinical trials have been treated with the vaccine, which is already in use in Cuba, Bosnia and Herzegovina, Colombia, Paraguay and Peru. A recent randomized study conducted by CIM indicated that patients treated with the vaccine have significantly improved overall survival and quality of life, with minimal side effects.

Roswell Park hopes to develop Cimavax-EGF to prevent primary lung cancers and potentially as a treatment for other cancers, such as head and neck, colon, breast, prostate and pancreas cancers.

Where's the enforcement?

In light of the worsening opioid epidemic facing the nation, two U.S. senators are questioning a reported decrease in enforcement activity at the Drug Enforcement Administration (DEA) aimed at preventing diversion of prescription opioids.

In a letter to the Justice Department, Sens. Patrick Leahy (D-Vt.) and Ron Wyden (D-Ore.) noted that the DEA's civil case filings against prescription drug distributors, manufacturers, pharmacies and doctors "plummeted" from 131 in fiscal 2011 to 40 in fiscal 2014. Meanwhile, sales of the painkillers nearly quadrupled from 1999 to 2014, they said, citing CDC figures, even though Americans haven't reported an overall increase in pain.

The senators seek information on DEA policies and standards that may be factors in the reduction in enforcement. They also want specifics on settlements the Justice Department has reached with drug companies regarding sales of controlled substances. For instance, the department agreed to a settlement with Cardinal Health Inc. four years ago, but the negotiations continue and no fines have been levied, according to the letter.

Patent rules proposed

The U.S. Patent and Trademark Office (PTO) is proposing revisions to two rules – one on the materiality standard for the duty to disclose information in patent applications and reexamination proceedings and the other on petitions for the revival or reinstatement of abandoned applications and canceled or expired registrations.

The duty to disclose rule would bring the agency's regulations in line with a 2011 Federal Circuit Court decision. The PTO first proposed the changes in 2011, but given the passage of time since the comment period ended five years ago, the agency decided to open it to further comment before finalizing the rule.

The rules are slated for publication in Friday's Federal Register. Comments on both rules are due by Dec. 27.