Sickle Cell Disease Patient Charts

The Prescription Is Available. The Patient Isn’t Getting It.

Casgevy prescribing intelligence from eleven hematologist conversations

About Patient Scribe

Patient Scribe is ZoomRx’s AI-powered conversational chart audit platform. Physicians walk through patient cases in a natural, voice-first conversation — sharing clinical reasoning, access experiences, and treatment barriers in their own words. The output is structured, but the input is uncompressed. What gets captured is what actually happened — including everything that a checkbox would never ask.

Therapy in scope: Casgevy (exagamglogene autotemcel, exa-cel) · Lyfgenia (lovotibeglogene autotemcel, lovo-cel) · Standard of care (Hydroxyurea, Endari, Adakveo, chronic transfusion therapy)

What This Study Was Designed to Answer

Eleven hematologists managing Sickle Cell Disease patients were interviewed on their Casgevy experience — from patient selection through post-treatment monitoring. The study targeted four key business questions:

• What clinical and patient factors determine eligibility for Casgevy, and how are hematologists applying those criteria in practice?
• What access, referral, and logistical barriers are preventing eligible patients from receiving Casgevy — and how large is the gap between actual and ideal uptake?
• How do physicians position Casgevy versus Lyfgenia, and what drives the choice between the two gene therapies?
• What manufacturer support — commercial, medical, or operational — would most accelerate Casgevy uptake in practice?

Key Takeaways

CONVICTION IS HIGH. EXECUTION IS BLOCKED.
Across eleven conversations, physicians consistently expressed high confidence in Casgevy — and consistently reported that a fraction of eligible patients are actually receiving it. Estimates of actual uptake range from 0% to 70% across practices; estimates of ideal uptake if barriers were removed range from 10% to 90%. The gap is not clinical skepticism. It is insurance authorization delays, a limited number of authorized treatment centers, cost at $2M+, and the logistical demands of a multi-step process that few practices can fully support. A checkbox PCA records whether Casgevy was prescribed. It does not record the six-month prior authorization that preceded the prescription, or the physician who cannot get a single appointment for an eligible patient. 

COMMUNITY PHYSICIANS ARE OUTSIDE THE COMMERCIAL ECOSYSTEM 
Three of eleven physicians reported zero or minimal contact with Casgevy manufacturer representatives or MSLs. All three are community hematologists — the physicians who are the primary referral origin for most SCD patients. One physician has never gotten an appointment for an eligible patient. Another has never seen a rep or MSL from the company. A third explicitly stated that manufacturer representatives do not visit community sites. The commercial infrastructure is concentrated at academic centers, where Casgevy is already administered. The referral pipeline that feeds those centers is going largely unengaged.

COMMERCIAL IMPLICATION
Community hematologists are the referral entry point, not the treatment point. A commercial strategy that reaches only academic centers reaches only the back half of the patient journey. Proactive engagement at community practices — including clear referral pathways and logistics support — is where the pipeline starts.

PATIENT HESITANCY IS A SECOND ACCESS LAYER THE CHART NEVER CAPTURES 
In two cases, an eligible patient declined or delayed Casgevy after the physician recommendation was made. One was a 16-year-old who refused gene therapy. Another was waiting on the patient to decide before the referral was initiated. In both cases, the prescribing record would show “not prescribed” without any indication that the clinical recommendation had already occurred. Patient hesitancy is a distinct access layer that operates after the physician decision — and is structurally invisible in any chart-based data collection.

COMMERCIAL IMPLICATION
Patient decision support programs are a commercial lever, not a clinical one. The patients who are being lost at the decision stage are not clinically ineligible — they are unconvinced or uncertain. Peer patient stories, shared decision-making tools, and long-term outcome visibility are the interventions that move this.

THE CONDITIONING REGIMEN IS THE MOST WANTED CLINICAL IMPROVEMENT 
Multiple physicians cited the chemotherapy-based conditioning regimen as the clinical barrier most likely to change eligibility decisions if addressed. Fertility risk, chemotoxicity, and secondary malignancy concern from conditioning were named explicitly. One physician identified a non-chemotherapy conditioning option as the single improvement that would most benefit the treatment process. This is not a concern about Casgevy itself — it is a concern about the path required to receive it.

COMMERCIAL IMPLICATION
Clinical messaging that acknowledges and contextualizes conditioning regimen risk — including fertility preservation protocols and toxicity management experience — directly addresses the concern that is holding back otherwise eligible referrals. Any pipeline development toward less intensive conditioning would materially expand the addressable population.

The Cases Behind the Insights

P1 — 37F | MEDICAID | HBSS | HU + ENDARI → CASGEVY (RECEIVED)

37-year-old female. HBSS genotype. 2–3 VOC/year despite therapy. Adequate organ function. Insurance approved.

Insurance did approve — but required prior authorization and extensive paperwork. The decision-to-infusion timeline ran approximately six months. Post-Casgevy: hemoglobin rose from 8.2 to 12.6. No VOC since infusion. Return to part-time work. The physician estimates fewer than 1–2% of SCD patients have received Casgevy overall, and does not expect that number to exceed 2% in practice. The outcome data is compelling. The pathway to getting there is not.

“I would be really surprised to see anything over approximately 1 to 2% in practice.” 
— Hematologist, SCD patient audit (P1) — on real-world Casgevy uptake

P4 — 27M | MEDICAID | HBSS | HYDROXYUREA 15 YRS → CASGEVY (RECEIVED)

27-year-old male. HbSS. 4 VOC/year, pain 8/10. ECOG 0. No prior complications.

Referral was smooth and no barriers were encountered. The physician is very confident in recommending Casgevy — with one explicit exception: non-compliant patients. The clinical recommendation is contingent on a compliance assessment that does not appear in any prescribing field. Two patients who look identical in chart data may receive very different recommendations depending on what the physician knows about their reliability as a treatment partner. Post-Casgevy: no further VOC episodes.

P6 — [AGE] M | MEDICAID | SS | HU + ADAKVEO → CASGEVY (0% RECEIVED, STILL TRYING)

Male. SS genotype. 4 VOC/year, 3 hospitalizations. ECOG 0. Recently added Adakveo.

The physician wants to refer this patient to Casgevy. The patient has not been able to get a single appointment at an authorized treatment center. The referral process has been described as “very difficult” and “very frustrating.” Actual uptake in this practice: 0%. Ideal uptake if barriers removed: 50%. The physician has never had contact with a manufacturer representative or MSL. The gap between clinical conviction and treatment access is total in this case.

“It has been very difficult to refer the patient because the patient has not been able to get any appointment. Very frustrating.”
— Hematologist, SCD patient audit (P6) — on ATC access barriers

P8 — 16F | MEDICAID | SS | NO GENE THERAPY — PATIENT DECLINED

16-year-old female. SS genotype. 3 VOC episodes, ECOG 0, no comorbidities. Casgevy approved for age 12+. Patient discussed and eligible.

The physician raised Casgevy as an option. The patient refused gene therapy and is not currently pursuing it. The chart records no Casgevy prescription. It does not record that the recommendation was made, that the patient declined, or that the physician’s concerns included secondary malignancy risk from conditioning and the out-of-pocket cost burden for commercial insurance patients. The patient who said no is indistinguishable from the patient who was never offered the option in any structured prescribing data.

P11 — 34F | PRIVATE INSURANCE | HBSS | HU 20 YRS + ENDARI → CASGEVY (COMMUNITY REFERRAL)

34-year-old female. HBSS. 6 VOC/year, 6 hospitalizations, pain 7/10. Pulmonary hypertension and ACS history. Community hematologist.

The physician is in a community setting. The nearest authorized treatment center is two hours away. The eligibility assessment included a practical factor that will never appear in a clinical chart: “The patient has a car and is willing and able to travel.” Access was initiated through a personal contact at the academic center, not through any manufacturer-facilitated referral process. The manufacturer does not visit this community site. Post-Casgevy follow-up is being split between the community practice and the academic center, with coordination challenges that no formal protocol currently addresses. Actual uptake: 5%. Ideal if barriers removed: 75%.

“If manufacturers came to community sites to advertise the product so that we are aware of it and what the referral process is like.”
— Hematologist, SCD patient audit (P11) — community physician, on manufacturer engagement

What Better Research Finds That Prescribing Data Cannot

Eleven conversations. Eleven physicians who believe in Casgevy. A real-world uptake that runs at 5% or below in most practices — not because physicians are unconvinced, but because the infrastructure required to deliver a gene therapy at scale does not yet exist outside academic centers. The commercial opportunity in this indication is not persuasion. It is execution. And execution requires knowing exactly where the pipeline is breaking — which is not something any prescribing dataset can tell you.

A checkbox audit of the same eleven physicians would have recorded eleven Casgevy non-prescriptions — or a small number of prescriptions with no accompanying explanation for the gap. The commercial team reading that output would have no way to distinguish a physician who has never seen a rep from one who has referred three patients to an ATC that cannot get them appointments. Both look the same in the data. They require entirely different responses.

Conversational research does not just add nuance to the numbers — it identifies problems that the numbers cannot even form a question around. In Sickle Cell Disease, the access barriers are specific, named, and addressable. Getting to them requires a research format that asks physicians to explain what happened — not to select from a list of what might have.

See What Patient Scribe Would Uncover for Your Brand

ZoomRx is offering a zero-cost pilot wave for teams who want to see what Patient Scribe surfaces for their brand. Same rigor as a full PCA study, no commitment required.