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Research & Reports

Searching in Biopharma (1575)

How Will Payers and Physicians Respond to Novel Agents in Late-Stage Clinical Development for Pulmonary Hypertension?

Pulmonary hypertension (PH) constitutes a group of rare diseases, yet the commercial potential for novel drugs approved for it is considerable, driven by the premium prices that may be charged for drugs in these indications. The late-stage pipeline for PH includes Bayer’s first-in-class soluble guanylate cyclase (SGc) stimulator riociguat, Actelion’s selexipag (oral non-prostanoid prostacyclin analogue), and Actelion’s Opsumit (macitentan; novel endothelin receptor antagonist [ERA]).

At the annual CHEST meeting in October 2012, Actelion and Bayer presented pivotal clinical trial results for their respective investigational oral PH therapies, Opsumit and riociguat. Both therapies met their Phase III primary end points, with Opsumit reducing the risk of a mortality and/or morbidity event in pulmonary arterial hypertension (PAH) patients compared with placebo, and riociguat demonstrating improvement in exercise capacity in two separate trials conducted in PAH patients and in patients with chronic thromboembolic pulmonary hypertension (CTEPH).  

The lack of head-to-head trial data for current PH therapies, either against one another or against agents in development, and a lack of data in patient populations other than PAH, create an increasingly complex treatment landscape. Pulmonologists face the challenge of considering each new therapy’s unique balance of benefits, drawbacks, risks, and rewards when making treatment decisions. Furthermore, the evolving reimbursement landscape and loss of market exclusivity for the first oral PH therapies in the United States (sildenafil [Pfizer’s Revatio] in 2012 and bosentan [Actelion’s Tracleer] in 2015), coupled with the burgeoning number of available treatment options, will result in increased market access challenges for PH therapies.

The entrance of these therapies will lead to the development of a more complex marketplace for severe, refractory disease, leading physicians and payers alike to make key decisions about which treatments to prescribe or reimburse over others. This report examines the dynamics that will limit or promote market access for new market entrants, employing the results from our survey of 100 pulmonologists and 30 managed care organization (MCO) pharmacy or medical directors.

Questions Answered in This Report:

  • Understand pulmonologists’ use of currently available PH therapies. How do pulmonologists and payers differentiate between the different oral, inhalable, and infused PH therapies? What are the key considerations for physicians when selecting PH agents, and how does treatment differ for different subpopulations of PH patients (e.g., PAH, CTEPH, or PH due to left ventricular dysfunction [LVD])? What restrictions (e.g., step therapy) do payers place on the use of current PH agents?

  • Explore pulmonologists’ and payers’ attitudes towards treating PH as new therapies become available. How do surveyed pulmonologists expect to incorporate Opsumit, riociguat, and selexipag into clinical practice? Where do they expect these agents to sit in the changing treatment algorithm, and which current PH therapies are most at risk of losing share?  What factors will most influence formulary inclusion of these emerging agents? How will MCO reimbursement constraints, including tiering and formulary restrictions, impact the potential patient share of these emerging drugs? Are data demonstrating a benefit to morbidity/mortality an advantage in the eyes of payers for Opsumit, and, conversely, is the absence of such data a hindrance for riociguat?

  • Examine pulmonologists’ and MCO PDs’ receptivity to and expectations for generic versions of older PH drugs such as sildenafil and epoprostenol. How do pulmonologists expect to incorporate such agents into clinical practice should they reach the market? How will this impact the branded agents? What, if any, will be the impact on other branded PH drugs? How do MCO PDs expect to position generic sildenafil and generic epoprostenol on their formularies, what restrictions will they impose, and how will branded agents be affected?


This U.S. Physician & Payer Forum investigates payer and physician dynamics that affect prescribing practices for therapies for PH in the United States. The report is based on a survey of 100 pulmonologists and 30 pharmacy/medical directors at MCOs that offer commercial health insurance. We analyze current physician and payer insights and practices; perceptions of biologics and Spiriva in the pipeline; uptake of emerging therapies for severe, refractory asthmatics; and formulary decision making for current and emerging agents, including Actelion’s Tracleer, Gilead’s Letairis, Pfizer’s Revatio, United Therapeutics’ Adcirca, GlaxoSmithKline’s Flolan, Actelion’s Veletri, United Therapeutics’ Remodulin, Actelion’s Ventavis, and United Therapeutics’ Tyvaso.

Markets covered: United States

Primary research: Online survey of 100 pulmonologists and 30 MCO pharmacy or medical directors

Population segments: Group 1 - 5 PH