Survey: Patients Want Low Cost, Under-the-tongue Treatment

When choosing on-demand treatments for “off” episodes, individuals with Parkinson’s disease prefer those with lower out-of-pocket cost and an under-the-tongue (sublingual) mode of administration, according to a survey of 300 patients living in the U.S.

Next in preferred features was faster onset, followed by longer duration of “on” periods.

These findings may help optimize an “off” episode treatment that fits Parkinson’s patients’ preferences, lifestyles, and circumstances, the researchers noted.

“Overall, respondents preferred treatments in which the mode of administration was a dissolvable sublingual film, with rapid onset, longer duration of effect, and low cost,” the researchers wrote.

The results of the survey were shared in a study, “Patient Preferences for Treating “OFF” Episodes in Parkinson’s Disease: A Discrete Choice Experiment,” published in the journal Patient Preference and Adherence.

While therapies such as oral levodopa and carbidopa are considered mainstays of Parkinson’s treatment, patients over time often begin to experience fluctuations in their motor and/or nonmotor symptoms. This is due to an increasingly faster wearing-off of the treatment’s effects, causing so-called “off” episodes in which symptoms occur.

“Moreover, “OFF” episodes may have a significant negative impact on patient quality of life and can make self-administration of treatment challenging, highlighting the need for on-demand treatments that can rapidly convert patients from “OFF” to FULL “ON” in a manner that can be managed by patients while experiencing symptoms,” the researchers wrote.

In the U.S., several on-demand treatments are available for managing these “off” episodes, including under-the-skin injections of Apokyn (apomorphine), now owned by Supernus Pharmaceuticals, inhalation of Acorda Therapeutics’ Inbrija (levodopa inhalation powder), and a sublingual, dissolvable formulation of apomorphine, marketed by Sunovion as Kynmobi (apomorphine hydrochloride).

“As the landscape for on-demand treatment of “OFF” episodes evolves, it is important to understand which features patients prefer among the available treatment options,” the researchers wrote.

Now, Sunovion’s researchers, along with RTI Health Solutions, used a discrete choice experiment to evaluate the preferences of Parkinson’s patients for features of theoretical on-demand, “off” period treatment options.

This type of survey is increasingly used in healthcare to elicit preferences from responders, including treatment features, without directly asking them to state their preferred options.

Responders were presented with a series of choice scenarios in which they were asked to choose between two theoretical on-demand treatments with varying features, forcing them to trade off between their preferred and less-preferred features presented in each choice scenario.

Varying features included the mode of administration — sublingual, inhaled, or injected — with and without mode-specific adverse events. Time to regain a full “on” state, with options of 15 minutes, 30 minutes, or 1 hour, was another choice, along with that of the duration of “on” periods, with times of one hour, 1.5 hours, or two hours.

Finally, patients also had to consider the out-of-pocket cost for a 30-day supply, with possible pricetags of $0, $10, $30, or $90.

Participants were invited to take part in the online survey, conducted between September and October 2019, through e-mails sent by a healthcare research recruiting firm.

A total of 300 U.S. adults responded to the online survey. These individuals were taking oral carbidopa/levodopa and had a self-reported physician diagnosis of Parkinson’s disease made at least five years prior, or before that.

Their mean age was 59 and 83% were white. Almost all (98%) reported experiencing “off” episodes, with half reporting at least one “off” episode per day, and 90% having more than one per week.

The survey results showed that respondents preferred a theoretical dissolvable sublingual film with no mode-specific adverse events over all other modes of administration, regardless of whether the other modes had associated adverse events or not.

The patients also preferred theoretical treatments with faster and longer-lasting effects, and with no out-of-pocket cost.

Further analyses revealed that the most important factor for responders was avoiding a possible $90 cost, followed by a change between an injectable treatment with possible injection-site reactions to a dissolvable sublingual film without adverse events, and a reduction in time to full “on” state.

The duration of “on” periods was found to be relatively less important, compared with the other features.

Also, the respondents were willing to pay $83 to change from an injectable treatment with possible injection-site reactions to a dissolvable sublingual film without adverse events. They also indicated their willingness to pay $28–$52 to switch from a least- to more-preferred mode of administration with associated adverse events.

Participants also were willing to pay $58 to reach a full “on” state in 15 versus 60 minutes, and $9 to increase the duration of “on” periods from one to two hours.

Since previous studies suggested that some patients may want to avoid daily injections due to fear of needles and also may have trouble assembling administration devices while having “off” episodes, “a theoretical sublingual film may have appealed to respondents,” the researchers wrote.

Among the study’s limitations, the team noted, were its recruitment strategy and the online nature of the survey, which may prevent a generalization of these findings to the broader population of Parkinson’s patients.

“Physicians should work with their patients to assess the need for an on-demand treatment for “OFF” episodes that fits their patients’ lifestyles and circumstances,” the researchers wrote.