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Since 2015, innovative gene and cell therapies (Alofisel®, Kymriah®, Holoclar®, Imlygic®, Spherox®, Strimvelis®, Yescarta ®, Zalmoxis®, Luxturna® and Zynteglo®) have been granted marketing authorization by the European Commission. The aim is to compare pathways to market access for such therapies.

There are several mechanisms other than Health Technology Appraisal (HTA) approvals and Pricing & Reimbursement (P&R) decisions by which patients can access orphan drugs pre-launch within a local healthcare system.

Early access programs (EAPs) for orphan drugs differ among the EU member states due to their national regulation based on which the EAPs are implemented.

The objective of this analysis is to examine similarities and differences of EAPs in the EU-5 countries (Germany, France, Italy, Spain and UK).

The healthcare system in Europe seems simple at first glance. The European Medicines Agency (EMA) centrally regulates the marketing authorization of new medicines in Europe. However, the health systems are not part of the Lisbon Treaty and therefore in the national accountability of the individual European Union (EU) member states. This means 28 different ways of market access and reimbursement.

For orphan drugs, the opportunities of market access begin even before the drug approval through EMA. To show the possibilities, the market access, pricing and reimbursement processes of the big five European countries (EU5) - France, Germany, Italy, Spain and the United Kingdom (UK) - were analysed for similarities and differences.

Visual impairments (VI) have a huge impact on patient’s quality of life. They restrict the work ability and daily life of patients and if untreated VI could cause blindness.The WHO counts worldwide 36 million blind people and 217 million people with visual impairment. Around 75% of these might be avoidable with (early) treatment. [1]

In the last 10 years, various medications for visual impairments have been assessed by HTA authorities in the EU5. This study provides a comparison of benefit assessments for reimbursement by EU-5 HTA agencies using the Prismaccess®/Evalumade® database. Within that it uses its three-colored scale and shows the availability of therapies for patients.

Although the orphan drug designation and marketing authorization (MA) are managed and granted at the European level by the European Medicines Agency (EMA), the pricing and reimbursement process is defined on a national level, is often driven by HTA outcomes and is strongly influenced by the external price referencing.

In Italy͕ the price setting of medicines reimbursed by the NHS is regulated at the central level by the Italian Medicines Agency (AIFA).

In Italy, access to treatment for patients suffering from a rare disease is guaranteed through various legislative instruments.

In Italy, access to treatment for patients suffering from a rare disease is guaranteed through various legislative instruments.

In July 2006 the Italian Medicines Agency (AIFA) signed the first managed entry agreement (MEA). MEAs are arrangements between manufacturers and payers that enable access to health technologies subject to certain conditions.

In France, the Temporary Authorisation for Use (ATU) (early access process) allows patients to be treated by drugs that may not have received a marketing authorisation. These ATUs are provided in the occurrence of unmet medical needs for serious or orphan diseases in the absence of alternative treatments. During this specific early access program, some data may be recorded in order to reinforce the evaluation conducted by the HAS and may impact the P&R delay.

For each indication of a drug that has a positive reimbursement decision (sufficient medical benefit), the Transparency Committee (TC) of the HAS gives an opinion on the “Added Medical benefit” (ASMR) also called “actual clinical benefit”. The added medical benefit measures the drug’s added clinical value compared to existing therapies already reimbursed and is used to determine the price. In France, the Temporary Authorization for Use (ATU) (early access process) allows patients to be treated by drugs that may not have received a marketing authorization.

For each indication of a medicine, the Transparency Committee (HAS) gives an opinion on its “Medical benefit” (SMR). This opinion determines whether the medicine is reimbursed (three ratings: important, moderate, low) or not (insufficient medical benefit) and the rate of its reimbursement by the French national health insurance (important: 65%; moderate: 30%; low:15%). In France, the Temporary Authorization for Use (ATU) (early access process) allows patients to be treated by drugs that may not have received a marketing authorization. These ATUs are provided in the occurrence of unmet medical needs for serious or orphan diseases in the absence of alternative treatments. During an ATU period, some data may be recorded and may impact the evaluation conducted by the HAS.