While Republicans and Democrats in the U.S. House are at odds over proposed changes to the Affordable Care Act, their colleagues in the Senate are working together to rein in drug prices and ensure that incentives for orphan drugs are not being abused.

At the same time, they want to make the patient voice part of every step of the drug approval process, especially for therapies intended to treat rare diseases.

Those bipartisan efforts are likely to impact the future confirmation hearing for the FDA commissioner and the final shape of the next drug user fee agreements. During a colloquy on the Senate floor this week, Sen. Orrin Hatch (R-Utah) said he would stress in the confirmation hearing "the importance of incorporating a balanced and flexible approach when weighing risks, benefits and outcomes" in the evaluation of new drugs intended to be used in small populations of patients facing rapidly progressing prognoses.

While it's important for the FDA to use its authority to accelerate the evaluation and approval of drugs for rare diseases, Congress also must provide the proper incentives for R&D in the rare disease space, Hatch said.

Patients with rare diseases, and their families, should have greater clarity about the FDA's process for evaluating and approving drugs, and "they ought to be included and informed every step of the way," Sen. Amy Klobuchar (D-Minn.) added.

"All too often, as we rightly focus on evidence-based medicine, we can lose sight of the human experience of these diseases and different therapies. What may seem simple in a lab may be overwhelming or difficult when applied to real patients in real-life situations – all the more so when children are involved," Klobuchar said. "The FDA and all agencies should ensure that they have appropriate processes to seek and incorporate this vital input."

But it's not enough for new therapies to be developed. "It is critical that treatments that do exist for those with rare conditions are accessible and affordable," Klobuchar said, as she called on Congress to protect such patients from discrimination in insurance coverage and to work to bring down drug costs.

IS ODA WORKING?

Klobuchar and Hatch, the co-chairs of the Senate Rare Disease Caucus, also urged their colleagues to ensure that incentives to spur the development and accessibility of drugs for rare diseases are not being abused by drug manufacturers.

Just a few days earlier, Hatch had joined with Sens. Chuck Grassley (R-Iowa) and Tom Cotton (R-Ark.) in asking the Government Accountability Office (GAO) to look into whether drug companies are taking advantage of the multiple designation allowance in the orphan drug approval process. The requested review is to include a list of all orphan-designated drugs approved since the Orphan Drug Act (ODA) was passed in 1983 and a list of drugs for which there was a request in the past year for orphan drug designation, along with all non-orphan indications for each of those drugs.

The senators also asked the GAO to assess whether the FDA has the resources to keep up with the volume of orphan drug requests and whether the evaluation criteria are consistent from one reviewer to the next. As part of its report, the GAO is to include patient experiences and its recommendations on regulatory or legislative changes needed to maintain the integrity of the ODA and stimulate the development of rare disease drugs.

PAP VS. HIGH PRICES

Meanwhile, Grassley continued his siege on drug prices Wednesday with a letter to Spencer Williamson, president and CEO of Kaléo Pharmaceuticals Inc. Grassley quizzed Williamson about the patient assistance programs (PAPs) and the $4,500 list price for the company's Auvi-q, an epinephrine auto-injector intended to compete with Mylan NV's Epipen.

When Mylan raised the list price of Epipen to more than $600 last year, it spurred public outrage that resulted in congressional hearings and the launch of an authorized generic two-pack that sells for $300. Mylan defended its pricing, pointing to its PAPs for Epipen, which counters severe allergic reactions, and school giveaways. (See BioWorld Today, Aug. 30, 2016.)

Noting that the Richmond, Va.-based Kaléo is relying on its assistance programs to make Auvi-q a competitor, Grassley told Williamson, "Your pricing of Auvi-q appears to draw parallels with concern about Epipen's pricing structure. . . . Your pricing structure may simply shift the burden and cost to another entity within the health care system."

A number of insurance companies and pharmacy benefit managers have indicated they don't support Kaléo's pricing strategy and they won't be adding Auvi-q to their formularies, Grassley said.

He then asked Williamson to explain the company's decision-making process on the Auvi-q price and how the pricing structure would result in reduced prices to patients. He also requested a list of entities in the health care system that may have to pay the full price for the auto-injector.

When Kaléo first announced the mid-February relaunch of Auvi-q and its $4,500 price tag, company officials defended the price, saying it would allow Kaléo to "absorb the cost of Auvi-q for patients who would otherwise not have access." In other words, some patients and payers would be subsidizing the PAPs. (See BioWorld Perspectives, Jan. 26, 2017.)

Despite its price or because of its PAPs, Auvi-q accounted for more than 12 percent of auto-injector prescriptions in the first few weeks after it hit the market, according to Athenahealth.

Grassley's letter isn't the first to call out Kaléo on its pricing. Klobuchar wrote a similar letter to Williamson last month, asking for an explanation of the price hikes for Auvi-q and Evzio, a device that administers naloxone to counter opioid overdoses. She called the company's pricing "disturbing" and noted that competition should mean lower, rather than higher, prices.

Senators weren't the only ones addressing drug prices this week. President Donald Trump met Wednesday with Reps. Elijah Cummings (D-Md.) and Peter Welch (D-Vt.) to find common ground on which they could join forces to battle rising drug prices.

During the meeting, the president reiterated his commitment to lessening the regulatory burden and reforming the FDA, according to the White House. Cummings used the opportunity to seek the president's support on his bill that would allow Medicare to directly negotiate drug prices with manufacturers.

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