Pulmonary hypertension (PH) constitutes a group of rare diseases, including pulmonary arterial hypertension (PAH), yet the commercial potential for novel drugs approved for PH indications is considerable, driven by the premium prices that may be charged for drugs in these indications. Several novel therapies for the treatment of PAH have launched in the last five years, including Actelion’s Opsumit (macitentan), Bayer Healthcare’s Adempas (riociguat), United Therapeutics’ Orenitram (oral treprostinil), and Actelion’s Uptravi (selexipag). The launches of these agents, along with the increasing use of combination therapy following the positive results of the Phase III AMBITION trial, have added to the variety of treatments available for PAH. They have also contributed to the increasing complexity of the PAH treatment algorithm. For example, the soluble guanylate cyclase stimulator Adempas represents a novel drug class for the treatment of PAH, is the first therapy to be approved for more than one WHO PH group and is the first therapy to be indicated for the treatment of chronic thromboembolic pulmonary hypertension (CTEPH). The use of off-label therapies is another feature of the PH treatment landscape. Despite launches of new agents, off-label use of therapies, and the poor prognosis for patients with PH, the reimbursement environment for PH therapies is challenging, and the healthcare costs associated with PH are substantial. Consequently, the market opportunity for an efficacious, affordable, well-tolerated agent is considerable, although drug developers need to be aware of the unique dynamics of the PH market to ensure access, secure reimbursement, maximize uptake, and realize commercial potential.
Markets covered: United States.
Primary research: Online survey with 54 pulmonologists, 52 cardiologists, and 31 MCO pharmacy and medical directors.
Commercial context: Epidemiology tables, drug-treatment algorithms, and managed care background information.
Therapies covered: Drug classes used for the treatment of PH and PAH: endothelin receptor antagonists, PDE-5 inhibitors, prostacyclin analogues, and a soluble guanylate cyclase stimulator.