News Digest: 31 Jan 2024

We summarise the latest pricing and market access news in the pharmaceutical industry ??

Overhaul of AIFA regulations marks milestone in Italian pharmaceutical landscape

A major reorganization at the Italian Medicines Agency (AIFA) took effect on January 30th, as announced in the Journal of Official Gazette. AIFA's regulatory overhaul aims to enhance efficiency, streamline operations, and expedite drug approval processes, ensuring swift access to medical innovations for the Italian population.

The restructuring involves the establishment of the "Scientific and Economic Commission for Medicines," replacing both The Technical and Scientific Committee (CTS) and the Pricing and Reimbursement Committee (CPR). Consequently, the number of members involved in drug assessment will be reduced from twenty to ten in the new commission.

The ministerial decree introduces modifications to the rules governing AIFA's structure and operation. Notably, the position of Director General is eliminated, and the president becomes the official legal representative. The Minister of Health will appoint the Technical Scientific Director and the Administrative Director, considering input from the regions. The board of directors will now consist of the president and four members appointed by various entities, including the Minister of Health and the Minister of Economy and Finance.

What does this overhaul mean for stakeholders?

The updated AIFA regulation is a significant milestone for Italy's pharmaceutical landscape. Stakeholders in the industry should anticipate potential challenges, such as the removal of the Director General role and the introduction of a new commission. The reduction in the number of members involved in drug assessment may pose obstacles to expediting the approval process. Proactive engagement with these changes will be crucial for adapting strategies and maintaining efficiency in bringing medicines to market.

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AML treatment Tibsovo earns key rating in Germany

Following the G-BA meeting on January 18, 2024, Tibsovo (Ivosidenib) has attained a major additional benefit rating for its application as a first-line treatment in conjunction with azacitidine for acute myeloid leukemia carrying the IDH1-R132 mutation. Tibsovo now stands as the third orphan drug to receive this esteemed rating, joining the ranks of blinatumomab, which achieved the same recognition in January 2022.

In Germany, since the inception of AMNOG in 2011, novel pharmaceuticals must undergo an initial benefit assessment. In this meticulous process, IQWiG assembles a comprehensive dossier, empowering the G-BA to ascertain the product's benefit rating, evaluating whether and to what extent a clinical advantage over existing therapies is demonstrated. The assessment outcomes are systematically classified into six levels:

• Major additional benefit – premium
• Considerable additional benefit – medium
• Minor additional benefit – parity
• Additional benefit (non-quantifiable) – parity
• No additional benefit
• Benefit less than comparator

The outcome of the benefit rating plays a pivotal role in determining the pricing potential of drugs in Germany. Attaining a premium compared to the standard of care is only feasible when a major or considerable additional benefit rating is granted, as a minor or non-quantifiable additional benefit rating only permits price parity.

Despite a notable surge in the number of drugs receiving major additional benefit ratings from IQWiG in 2022 (9%), in contrast to the preceding two years (5%), the overall count remains relatively low. This underscores the formidable challenge manufacturers face in securing this coveted rating.

Establishing clinical advantages for orphan drugs proves particularly arduous due to non-randomized, single-arm trials often lacking robust endpoints. While orphan drugs are exempt from the benefit assessment procedure and automatically receive a non-quantifiable added benefit upon regulatory approval, they must undergo assessment if the €30m annual revenue threshold is exceeded (€50m before the GKV Financial Stabilization Act).

In 2022, IQWiG meticulously evaluated all orphan drugs that had undergone the benefit assessment procedure since 2011 due to surpassing the €50m threshold. The evaluation focused on confirming the automatically granted 'additional benefit' rating. In over half of the cases (54%), the confirmation did not materialize, resulting in a final rating of "no additional benefit." This was primarily attributed to the absence of an "appropriate comparator therapy."

Tibsovo earned its major benefit rating by undergoing the regular IQWiG assessment, which included:

• A randomized controlled trial with the appropriate comparator (Ivosidenib + Standard of Care vs. placebo + Standard of Care)
• A significant improvement on a hard primary endpoint, namely overall survival

So what does this mean for manufacturers?

With the implementation of the right trial - a randomized controlled trial with the appropriate comparator and robust endpoints - orphan drugs have the potential to secure a high benefit rating and, consequently, command a premium compared to the standard of care.

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NICE recommends Talazoparib for advanced breast cancer

In a significant development, NICE has revised its initial draft decision and is now set to endorse talazoparib, marketed as Talzenna, for adult patients with BRCA 1 or 2 mutated HER2-negative locally advanced or metastatic breast cancer post-chemotherapy, which will benefit 300 patients following price agreement. This shift follows Pfizer's proposal of a substantial price reduction for talazoparib.? This decision is particularly notable considering the current absence of targeted treatments for this advanced breast cancer variant on the NHS, where options are notably sparse.?

Helen Knight, NICE’s Director of Medicines Evaluation, highlighted the significance of this development. She pointed out that talazoparib, a once-daily oral medication, offers greater convenience compared to the hospital visits required for intravenous chemotherapy. Despite some residual uncertainties in clinical evidence, Knight emphasized that the notable discount from Pfizer and the profound impact of advanced breast cancer on life quality and duration justify recommending talazoparib for NHS use.?

Talazoparib operates as a PARP inhibitor, targeting and decelerating the growth of specific cancer cells, making this update a noteworthy advancement in breast cancer treatment options. Clinical trial data revealed that talazoparib extends progression free survival compared to chemotherapy, although it doesn't necessarily prolong overall survival.?

Once the draft guidance becomes available next month, NICE will have consistently supported all 20 breast cancer treatment appraisals completed since 2018.??

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Financing of new orphan drugs in Spain increases in 2023?

Last year was a positive one for patients with rare diseases as for the first time in 5 years Spain has financed more than half of the orphan drugs authorised by the EMA, with the National Health System (SNS) containing 78 of the 147 orphan drugs with EMA marketing authorization.?

In total 21 new orphan drugs were approved for funding in Spain last year, an increase of 12 compared to 2022, whilst the waiting time to reach a funding decision also improved, going from 34 months in 2022 to 23 months in 2023. However, despite this, all new orphan products are subject to conditional financing and of the new orphan products with positive pricing and reimbursement decisions, none were advanced therapies. A report by the Spanish Association of Orphan and Ultra-Orphan Drug Laboratories (AELMHU) also points out that although there have been improvements compared to 2022, there is still work to do to reduce processing times back to the 14 month average in 2019.??

Although there has been a slowdown in access to orphan medicines in recent years this signals that Spain is looking to improve timelines and become a more attractive place for orphan drug access, although there are still improvements to be made, especially in relation to advanced therapies.

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