Looking Ahead to 2024: Robust Outlook for Specialty Drugs, Cell and Gene Therapies

As we bid farewell to 2023, it's time to reflect on the dynamic landscape of the specialty pharmacy industry and forecast significant trends in the year ahead.

Total public and private spending on healthcare as a percentage of GDP is 11.9% in the UK and 10.1% in the EU. While medicines comprise the smallest component of the healthcare budget (18.4%), spending on prescription pharmaceuticals in the UK and EU combined now exceeds €254 billion annually. This is in part due to the increasing number of high-cost novel medicines entering the market, especially cell and gene therapy products (CGTs) for use in rare and orphan diseases. These potentially curative therapies can have list prices in excess of €2 million per treatment. Affordability remains key as payers, manufacturers and patients balance cost and access to ensure optimal patient outcomes.

The forecast for Europe is that the growth in expenditure on medicines will continue, with analysts estimating a compound annual growth rate of 4.5% for the period 2023 to 2030.

Issues, Trends and Solutions Worth Watching in 2024

Specialty Drugs and Cell & Gene Therapies for Rare Disease

CGT research is strong in the UK and EU; however, challenges exist when translating this research into advances in healthcare for its citizens. Like most other nations, the UK and EU are faced with high costs, low manufacturing yields, complex supply chains, and a lack of long-term safety and efficacy data associated with CGTs. However, the fragmented nature of the European market creates additional challenges in relation to cross-border supply chain as well as complex reimbursement systems.

Despite these challenges, the UK recently became the first country globally to give regulatory approval to a CRISPR-based technology. The Medicines and Healthcare products Regulatory Agency (MHRA) granted conditional marketing authorisation for Casgevy™ (exagamglogene autotemcel [exa-cel]) for the treatment of sickle cell disease and transfusion-dependent beta thalassemia on 9 November 2023. This is the first medicine to be licensed that uses this Nobel Prize winning technology. Exa-Cel is also under review by the European Medicines Agency (EMA).

Shift Toward Value-Based and Outcome-Based Contracts

Value-based and outcomes-based contracts, sometimes called risk-sharing agreements, are innovative payment models used by payers and biopharmaceutical innovators that link reimbursement, coverage or payment to a treatment's real-world performance. It is anticipated that these arrangements will promote greater patient access to new treatments in the future.

Unique Financial Solutions

Demand for unique financial solutions is expected to grow, especially as healthcare budgets come under increasing strain with the approval of more and more CGTs that have multi-million-euro price tags. AscellaHealth’s innovative financial solutions, such as Pharma FlexFund™, will increasingly play a significant role in offsetting the cost of these high-cost drugs and enabling patients to get access to potentially curative CGTs.

Increased Emphasis on Patient-Centric Care

While the call for patient-centric care resonates throughout the healthcare ecosystem, there is no greater need for a patient-first approach than among individuals who are living with a rare disease or complex, chronic condition. Many of these patients share the experience of a time-consuming and sometimes frustrating journey towards diagnosis, the lack of established standards of care and the search for physicians who understand the challenges of a specific rare condition.

AscellaHealth’s novel approach always puts patients at the centre of care, exemplifying a value chain that focuses upon:

• Improved access to specialty pharmaceutical therapies
• Information exchange among prescribers, patients, and specialty pharmacies that streamlines care delivery
• Personalised, high-level coordinated support from dedicated patient care coordinators vs. automated call centres to help patients manage their conditions
• Continuity of care throughout the patient’s entire treatment journey for optimal health outcomes

This holistic approach guides stakeholders throughout the specialty pharmacy ecosystem in providing disease-specific care that improves quality of life for patients as well as their caregivers.

Evolving Role of Specialty Pharmacy (SP) and HUB Models to Manage Specialty Medications

The evolving landscape of SPs and HUB pharmacy models in managing specialty medications prompts a critical examination of their multifaceted role. AscellaHealth, at the forefront of this evolution, brings its specialised expertise to navigate the complexities of specialty medications for a diverse range of stakeholders, including patients, payers, and life science companies. With the ability to implement bespoke programmes for specialty medications, AscellaHealth not only ensures cost savings, but also enhances patient outcomes.

Greater Number of Biosimilars

Simply defined as a biological medicine highly similar to an already authorised biological medicine, referred to as the reference medicine, biosimilars are approved according to the same standards of pharmaceutical quality, safety, and efficacy that apply to all biological medicines. The adoption and potential of biosimilars to manage rising drug costs is globally recognised. In Europe, biosimilars are taking an increased share of the market from their originator products, creating savings for the healthcare systems and enabling patient access to critical new therapies. Growth of the biosimilar market is expected to continue into 2024.

The Regulatory Landscape

The EU regulatory landscape is undergoing a number of changes that will impact research and reimbursement. The new EMA Clinical Trials Information System (CTIS), based on the Clinical Trials Regulation (CTR EU 536/2014), came into full effect on 31 January 2022 with the intent of providing an easier, more streamlined approach to the registration of clinical trials taking place in Europe. Reports suggest that implementation of the regulations has been difficult, and the regulatory administrative bar has not yet been lowered. Further work will be needed in 2024 to achieve the goal of facilitating pan-European clinical trials.

Planning for the new EU Health Technology Assessment (HTA) process is well underway. The intent is to streamline the assessment of medicines, as well as in-vitro diagnostics and high-risk medical devices by using a joint clinical assessment (JCA) framework. The process will be rolled out in a phased manner, beginning with ATMPs and oncology therapies in 2025, adding orphan medicinal products in 2028, followed by all centrally approved medicinal products in 2030. The upcoming year will be a year of planning and preparation for payers and manufacturers.

On 26 April 2023, a revision of the EU's pharmaceutical legislation, including legislation on medicines for rare diseases was launched. The resulting reforms will be the first major revision of the legislation in 20 years. Proposed changes include a reduction in the length of the approval pathway from 210 to 180 days, which should help to improve Europe’s competitiveness as a launch market and to facilitate access to medicines for patients with rare diseases. More details of the changes should become apparent over the course of 2024.

AscellaHealth celebrates progress made in 2023 while acknowledging challenges. AscellaHealth anticipates the promising horizons that await patients with rare diseases or complex conditions. This includes life sciences manufacturers that are introducing novel therapies and other key stakeholders in the specialty pharmaceutical ecosystem.

For further information and/or assistance, contact AscellaHealth.