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Value-based services and Orphan Drugs: Italy says yes

By Andrea Bernardini, Senior Analyst

Email: [email protected]

AIFA has approved reimbursement of the orphan drug Evrysdi (risdiplam) by Roche, the first at-home oral treatment for Spinal Muscular Atrophy (SMA) type 1, 2 and 3. From this decision, we can spot significant trends and draw some key learnings on what to expect from 2022 and beyond.

We should firstly note that Evrysdi reimbursement decision “only” took ~6 months, which is ~1.5 month less than the median time to reimbursement (TTR). In fact, while in June 2021 Evrysdi was placed in class C(nn) (not yet evaluated for reimbursement), the reimbursement decision came in January with the drug placed in H class (full reimbursement and distributed by hospitals). Usually, only status of full innovativeness can reduce TTR of up to 1 month, so what did Evrysdi do to achieve such good outcome?

Evrysdi has already been available in Italy since 2020, thanks to a compassionate use scheme for patients with SMA type 1 and type 2. This shows how early access programmes (EAP) are favourable to a positive reimbursement decision for an OD in Italy, where this type of market entry is well established. EAP have often resulted in positive outcomes, not only for patient access but also for manufacturers. In fact, AIFA welcomes the collection of evidence in the in the Italian clinical setting that can be acquired during monitoring of such schemes.

Secondly, Roche not only offered a novel therapy but a whole innovative service that actively limits the need for hospital visits for vulnerable patients as the therapy can be independently managed by the patient itself and/or the caregiver. In fact, Roche also offered an app solution to the patient, through which clinicians can remotely monitor and manage therapy aspects like daily adherence and other factors to understand their impact on the product profile and optimise the therapeutic plan.

In fact, after the lesson learnt during the COVID19 pandemic, Italy wants to transform its healthcare system to a more decentralised and territorial-based one that facilitates patient access. An example of this renewed focus on therapy proximity can be seen in the National Resilience Plan, where enhancement of the territorial assistance is sought through the development of “community hospitals” or “intermediate care facilities”.

Evrysdi reimbursement decision shows how the opportunities offered by this transformation process can be seized by pharma manufacturers. In fact, significant importance is now being given by payers not only to the product itself but also to the additional services that can offer alternative therapeutic pathways with continuous remote monitoring and management in the safety of patients’ home. This decision is solid proof that digital tools that enable telemedicine, home-based therapies and remote disease management of rare disease are now appreciated by Italian payers. Here at P4A we already highlighted these trends last year, and early birds like Roche are catching the worms .

Sources:

https://www.gazzettaufficiale.it/atto/serie_generale/caricaDettaglioAtto/originario?atto.dataPubblicazioneGazzetta=2021-06-19&atto.codiceRedazionale=21A03644

https://www.gazzettaufficiale.it/atto/serie_generale/caricaDettaglioAtto/originario?atto.dataPubblicazioneGazzetta=2022-02-07&atto.codiceRedazionale=22A00781

https://www.aboutpharma.com/blog/2022/02/10/atrofia-muscolare-spinale-sma-aifa-approva-rimborsabilita-di-risdiplam/

https://journals.aboutscience.eu/index.php/aboutopen/article/view/2184/2221:~:text=Results%3A%20Of%20213%20new%20active,7.6%20months%20(228%20days)

Links to our previous opinions:

https://www.linkedin.com/posts/partners4access_sintomi-di-atrofia-muscolare-spinale-sma-activity-6814205275348729856-_vSS/

https://www.linkedin.com/posts/partners4access_in-italia-60-studi-clinici-%C3%A8-digitale-o-activity-6868566685650063360-K5p_/

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