Innovative Medicines Fund Update
Innovative Medicines Fund (IMF) recap
The IMF was launched in June 2022 to ensure patient access to promising non-cancer medicines when further data are necessary to resolve outstanding uncertainty. The IMF managed access fund provides £680 million of NHS funding to enable faster patient access for innovative medicines whilst data are collected. NICE supports all stages of the managed access process and evaluates whether a product is suitable for recommendation for routine use in the NHS.(1)
Until recently, only four products had received interim funding through the IMF and there were no formal NICE recommendations for managed access under the IMF. With the information available, it was unclear what the specific decision-making criteria were for the fund. However, on 24th July 2024, NICE published the final draft guidance for Hemgenix (etranacogene dezaparvovec), which was ‘recommended with managed access as an option for treating moderately severe or severe haemophilia B (congenital factor IX [FIX] deficiency) in adults without anti‑FIX antibodies’. In this article, we gather learnings from the first formal recommendation for managed access through the IMF and explore what this means for companies with medicines that may be candidates for this managed access fund.
Understanding the process for managed access through the IMF
Companies interested in being involved with the IMF with managed access should contact NICE early in development and highlight their technology meets the following criteria:(2)
- The technology has the potential to address a high unmet need.
- The technology has the potential to provide significant clinical benefits to patients; or
- The technology represents a step-change in medicine for patients and clinicians; and
- The new evidence to be generated is considered meaningful and could sufficiently reduce uncertainty.
Companies are required to cover expenses for database management, production, analysis reports, data linking to other sources and any other identified costs. The requirements for data collection will vary depending on the uncertainty detected. NICE will consult with physicians, patient groups, academics, and data custodians to ensure Data Collection Agreements (DCAs) include different stakeholders' perspectives. Regarding evidence of cost-effectiveness, manufactures should aim to set a reasonable price for the duration of managed access, along with the final set price.3
Etranacogene dezaparvovec for treating moderately severe or severe haemophilia B [NICE TA989]
Haemophilia B unmet need
Haemophilia B results in spontaneous or difficult-to-control bleeding episodes which are caused by the gene mutation that results in a deficiency of FIX, a blood clotting protein. The disease is associated with a reduced quality of life and the bleeding episodes can be fatal. Although there have been advancements in therapeutic options for preventing and managing breakthrough bleeding, significant gaps remain in further enhancing clinical, humanistic, economic, and societal outcomes
Hemgenix is a gene therapy for adults with haemophilia B, designed to introduce a copy of the human Factor IX coding DNA sequence into hepatocytes to address the root cause of the Haemophilia B disease.
Recommendation
On 24th July 2024, NICE published the final draft guidance for Hemgenix, which was ‘recommended with managed access as an option for treating moderately severe or severe haemophilia B (congenital factor IX [FIX] deficiency) in adults without anti‑FIX antibodies’.4 It is only recommended by NICE if the conditions in the managed access agreement for Hemgenix are followed. The committee made this decision based on the clinical data, which support that Hemgenix gene therapy reduces bleeding episodes annually, but stated that more long-term evidence is needed. Hemgenix will be covered under the IMF managed access fund with a list price of £2.6 million and will be accessible to patients in England, for a limited 5-year period while more clinical data are collected from the phase 3 HOPE-B clinical trial, under an outcome-based model (HOPE-B trials will offer a maximum of 10 years of data). Funding is dependent on Hemgenix demonstrating long-term clinical efficacy.
This represents a recommendation reversal from last year when NICE stated there were not enough long-term data and Hemgenix was not considered cost-effective, despite demonstrating a reduction in bleeding episodes.
Submission evidence and uncertainty
The positive recommendation from NICE was supported by evidence from the HOPE-B (NCT03569891) trial, which demonstrated that a single infusion of the gene therapy resulted in considerable increases in FIX activity levels that lasted up to 3 years and were accompanied by decreased annualised bleeding rates. The majority of patients (94%) no longer required routine infusions of FIX replacement therapy, during the HOPE-B trial.5
An indirect comparison indicates that Hemgenix improves bleeding outcomes compared to FIX prophylaxis. However, these findings were considered uncertain due to variations in the study methods, as well as the definition and evaluation of bleeding outcomes. Moreover, the lack of long-term evidence has led to a high level of uncertainty around cost-effectiveness. Despite having the potential to be cost-effective compared with FIX prophylaxis, this uncertainty has contributed to Hemgenix not being recommended for routine use in the NHS. To resolve the high levels of uncertainty, more evidence will be collected through the managed access agreement.
The committee also noted a few other considerations in their evaluation of Hemgenix; for example, the HOPE-B clinical trial did not include women or people with HIV/hepatitis. In order to receive any recommendation, the committee concluded that trials should include these patients. In addition, notable benefits were not included in the economic model, such as the potential to reduce long-term joint damage and to lower mortality. This would lead to a higher QALY benefit, so the committee agreed to include the possibility of uncaptured benefit in its decision-making. However, this did not lead to any material effect due to the uncertainty in long-term durability estimates.
References:
1. NHS England (2022). The Innovative Medicines Fund Principles. [online] Available at: https://www.england.nhs.uk/wp-content/uploads/2022/06/B1686-the-innovate-medicines-fund-principles-june-2022.pdf.
2. UK Parliament, Innovative Medicines Fund, Question for Department of Health and Social Care, available at: https://questions-statements.parliament.uk/written-questions/detail/2024-02-05/HL2187 [Quote from 09/02/2024]
3. Shapiro, L. (2024). In about-face, haemophilia B therapy Hemgenix to be covered by NHS. [online] hemophilianewstoday.com. Available at: https://hemophilianewstoday.com/news/about-face-hemophilia-b-therapy-hemgenix-covered-nhs/.
4. www.nice.org.uk. (2024). Project information | Etranacogene dezaparvovec for treating moderately severe or severe haemophilia B [ID3812] | Guidance | NICE. [online] Available at: https://www.nice.org.uk/guidance/indevelopment/gid-ta10699.
5. NICE (2024). Etranacogene dezaparvovec for treating moderately severe or severe haemophilia B. [online] Available at: https://www.nice.org.uk/guidance/ta989/resources/etranacogene-dezaparvovec-for-treating-moderately-severe-or-severe-haemophilia-b-pdf-82615918798789.
6. NICE (2024b). Overview | Etranacogene dezaparvovec for treating moderately severe or severe haemophilia B | Guidance | NICE. [online] www.nice.org.uk. Available at: https://www.nice.org.uk/guidance/ta989.
7. NHS England (2022b). The Innovative Medicines Fund Principles. [online] Available at: https://www.england.nhs.uk/wp-content/uploads/2022/06/B1686-the-innovate-medicines-fund-principles-june-2022.pdf.
8. NHS England (2024). Innovative Medicines Fund. [online] www.england.nhs.uk. Available at: https://www.england.nhs.uk/medicines-2/innovative-medicines-fund/.
9. SL Behring (2024). CTG Labs - NCBI. [online] clinicaltrials.gov. Available at: https://clinicaltrials.gov/study/NCT03569891.
10. NHS England (2024b). NHS England, Provider reimbursement in managed access funds – Standard operating procedure. [online] www.england.nhs.uk. Available at: https://www.england.nhs.uk/long-read/provider-reimbursement-in-managed-access-funds-standard-operating-procedure/.
11. NICE (2024). 4 Implementation | Etranacogene dezaparvovec for treating moderately severe or severe haemophilia B | Guidance | NICE. [online] Available at: https://www.nice.org.uk/guidance/ta989/chapter/4-Implementation.
12. NHS England (2024b). NHS England, Provider reimbursement in managed access funds – Standard operating procedure. [online] www.england.nhs.uk. Available at: https://www.england.nhs.uk/long-read/provider-reimbursement-in-managed-access-funds-standard-operating-procedure/.
Written by Alex Zaloumis
Decisive Dialogue 15th August 2024
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