Patient engagement has become more than a buzzword for the FDA and drug and device developers. But for payers, not so much.

When valuing new drugs and devices, payers often undervalue or ignore what they may consider convenience updates, giving little to no consideration to the difference a seemingly minor improvement could make to patients debilitated by fatigue, pain, the burden of treatment and the burden of a disease itself. They also may balk at giving patients access to transformative treatments and cures that come with hefty price tags.

Part of the problem is not listening to patients or understanding what life is like for them. “The North Star of drug development has to be the patient,” Insmed Inc. CEO William Lewis told BioWorld. The patient also is where the value conversation must begin. If payers and those making value assessments of drugs and devices spent time with patients, they would understand that, Lewis added.

William Lewis, CEO, Insmed

The object of medicine is to help a patient feel and function better, Lewis said. That can be a twofold goal that involves the eradication of an infection or disease and the alleviation of symptoms. Payers tend to focus more on a treatment’s value in eradicating the disease, whereas regulators also consider relieving symptoms. And that’s where patient outcomes generally come into play, Lewis said, as the ability to feel and function better is often connected to the relief of symptoms.

A symptom can become its own kind of disease in how it affects a patient. For instance, patients with refractory nontuberculous mycobacterial (NTM) lung disease caused by Mycobacterium avium complex (MAC) may suffer irreversible lung damage from the rare and often chronic lung infection. As a result, they may experience fatigue and coughing fits.

That’s not to say they’re just tired and coughing a lot. Their fatigue can be so bad that it’s impossible for them to walk a city block. And their coughing can come in rib-cracking fits that last 45 minutes at a time, Lewis said. A therapy that offers relief from those symptoms can be as valuable to the patient as one that clears the infection.

In developing Arikayce (amikacin liposome inhalation suspension) as the first approved treatment for MAC-NTM, Bridgewater, N.J.-based Insmed held listening sessions with patients and found consensus on the three or four most debilitating symptoms of the infection. It also hired artists who listened as patients described living with the illness and then developed artwork to express their struggles in a visual way that allowed others to glimpse the reality of the infection.

While companies like Insmed are benefitting from value conversations with patients, payers and organizations that assess the value of health technology too often are not. Meenakshi Datta, a partner at Sidley Austin LLP, attributed that in large part to payers’ short-term perspective.

In assessing value, many commercial and Medicare plans look at the impact over the current year rather than the long-term impact to the patient, Datta told BioWorld. That’s because they have no guarantee they’ll reap the long-term savings, as the patient may be on a different plan the next year. Thus, patient engagement activities that could positively move the needle on outcomes within a year would hold more sway with payers, Datta said. But plans may hold back on therapies and cures costing six figures or more because they wouldn’t recoup that cost in a year.

Impossible to quantify

Another issue is the difficulty of quantifying patient outcomes. The Institute for Clinical and Economic Review (ICER) grappled with that problem during a September summit on valuing potential cures and transformative therapies. At the summit, Michael Sherman, chief medical officer for Harvard Pilgrim Health Care, said the patient voice is important, but it’s hard to include it in a value assessment because it’s not easily quantified.

Esther Krofah, the senior director of Fastercures, presented a different perspective, stressing the need to include the patient voice in those assessments. In speaking with 14 diverse patient organizations, Fastercures found that the “value of hope” is stronger than ever as patients are beginning to realize the promise of cures and transformative therapies.

While patients are hopeful, they’re also “concerned that if the market doesn’t signal a willingness to absorb the high prices” of cures, the R&D investment will dry up, and the promise will end, Krofah said. Other patient concerns included payers’ limitations on who will be approved for expensive new treatments.

“In terms of disease progression, patient groups really expressed time and time again that any mechanism – any mechanism – that can delay the progression of disease, that can buy more time, that can maintain the ability to perform the activities of daily living was really extremely valuable,” Krofah said.

The patient groups stressed that the benefits of a cure or a transformative therapy extend beyond the patient, because of the economic impact a disease can have on the entire family. That impact is not just the cost of treatment and health care. For instance, when a child has a rare pediatric disease, one parent often must give up a job to stay at home to care for the child, Krofah said. That’s often overlooked in determining the value of a treatment.

Recognizing that they’re the most impacted by value decisions, patients told Fastercures that they want more input in the discussions, especially on critical endpoints used in value-based assessments.

Lewis recognized the difficulty of quantifying patient engagement, given the uniqueness of each disease and how it affects patients. Nevertheless, “it is absolutely essential to have the patient voice included in this dialogue,” he said.

“Increasing value is going to be placed on patient outcomes. … As the [patient] voice gets louder, you simply can’t ignore this,” Lewis said. Consequently, ICER and payers are going to have to find a way to address patient outcomes, he added.

Meanwhile, sponsors are going to have to be more cognizant early on of what payers want to see from a value perspective. In drug and device development, “the higher hurdle used to be the FDA,” Lewis said. “The higher hurdle is going to become reimbursement.”

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