Patient Influence on PNH Treatment Choices: Insights and Implications

Patient requests for Ultomiris and Empaveli are reshaping PNH treatment discussions—shifting selection from biomarker-driven choice toward attributes that matter to patients.

Clinician-led selection historically dominated PNH therapy, but patients now take a more active role, reframing expectations around treatment attributes and expanding the clinical conversation. Engagement through patient advocacy channels has increased awareness and practical knowledge, driving product-specific requests in clinic encounters.

Patients prioritize convenience, reduced dosing frequency, symptom control, and tolerability when they voice preferences. Requests increasingly favor therapies with less frequent infusions or simpler logistics and emphasize symptom relief that improves daily functioning. Perceived tolerability—how patients expect to feel on therapy—often shapes willingness to switch as much as efficacy, so side-effect profiles and administration burden now enter prescribing conversations.

Access constraints—prior authorization, coverage variability, step therapy requirements, and logistical hurdles—remain the dominant barriers to meeting many requests. Payer policies and regional plan differences frequently determine whether an expressed preference becomes an available option, while operational teams focus on documentation to support medical necessity and to prepare for appeals or prior-auth work. Those access realities can blunt the effect of patient influence on final treatment selection.

Patient priorities are a sustained force in PNH care; aligning clinicians, payers, and advocacy groups will be necessary to convert requests into delivered care.

Key Takeaways:

• Patient requests now explicitly target treatment attributes—convenience, dosing frequency, and perceived tolerability—rather than only clinical efficacy.
• Patients, hematology teams, and payer review processes are implicated across real-world treatment selection and access pathways.
• Expect clinical discussions to incorporate structured questions about dosing frequency, infusion burden, and convenience while administrative teams prepare supporting documentation for coverage decisions.