INNOVATIVE GENE AND CELL THERAPIES – MARKET ACCESS AND REIMBURSEMENT DECISIONS IN THE EU5: AVAILABILITY OR NOT, THAT IS THE QUESTION…
COMPARISON OF EARLY ACCESS PROGRAMS FOR ORPHAN DRUGS IN THE EU-5 COUNTRIES
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).
MARKET ACCESS, PRICING AND REIMBURSEMENT PROCESSES FOR ORPHAN DRUGS IN THE EU-5 COUNTRIES: A COMPARATIVE ASSESSMENT
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.
Comparison of EU market access decisions for Visual Disorders based on the PRISMACCESS® DATABASE
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. 
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.
Hysteroscopic removal of uterine fibroids and infertility treatment: Reimbursement in Germany
- Fibroids are the most benign tumor of the female genital tract and are usually diagnosed in the reproductive age. Therefore, they represent possible complications in the implementation of an acute desire to have children. (1)
- The main therapeutic options are minimally invasive surgical procedures or drug treatment due to the desire for a subsequent pregnancy (1). This study focuses on the minimally invasive surgical procedure of hysteroscopy.
- The study objective was to examine epidemiology of fibroids in Germany, the treatment option of operative hysteroscopy, subsequent assisted reproduction technology (ART) treatments and their reimbursement within the German health care system.
Time to reimbursement for orphan drugs in EU5 in the last 3 years
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.
PMU57: ANALYSIS OF THE EVOLUTION IN THE ACCESS TO MEDICINES IN ITALY IN THE LAST 5 YEARS (2014-2018)
PRO70: Italian Law 326/2003 application in the last 6 years: approvals, rejections and economic impact of this early access scheme
PRO76: Italian 648/96 Law application between Jan 2013 and September 2019: focus on orphan drugs
PMU67: Performace-based schemes in Italy: impact of their application in the last 5 years (2013-18)
REIMBURSEMENT AND PRICING TIMES FOR DRUGS DEPENDING ON THE EARLY ACCESS STATUS IN FRANCE (WITH OR WITHOUT AN ATU)
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.
COMPARATIVE ANALYSIS OF THE TRANSPARENCY COMMITTEE OPINIONS CONCERNING THE ADDED MEDICAL BENEFIT OBTAINED BY DRUGS AVAILABLE THROUGH THE EARLY ACCESS PROGRAM VS OTHER DRUGS IN FRANCE
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.
COMPARATIVE ANALYSIS OF THE TRANSPARENCY COMMITTEE OPINIONS CONCERNING THE MEDICAL BENEFIT OBTAINED BY DRUGS AVAILABLE IN EARLY ACCESS PROGRAMS VS OTHER DRUGS IN FRANCE
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.
PRICE DIFFERENCES FOR ORPHAN DRUGS ACROSS THE EU-5 COUNTRIES: A COMPARATIVE ASSESSMENT
- Pricing and reimbursement decisions after the European marketing authorization are the responsibility of each country, which can lead to inequalities and differences in access and prices for drugs1.
- The aim of this study was to assess market access and price differences in the five European Union countries (EU5) for orphan drugs approved by the European Commission (EC)
TIME TO MARKET AND PRICE COMPARISON FOR BIOSIMILAR DRUGS IN EU5
- After a medicine receives European Medicines Agency (EMA) marketing authorization, each member state then decides how that medicine is implemented at local level. In Germany, after EMA marketing authorization the drug is available by law. The existing differences between European countries in their policies on biosimilars may explain the heterogeneity in the drug uptake of these biosimilars. As a consequence of these differences, variations in savings can also be observed1.
- The aim of this study was to compare the time elapsed from EMA authorisation to national commercialization. Additionally, price differences of biosimilar drugs in five European countries (EU5) were assessed to evaluate whether the existing biosimilar policies might drive this difference.
CANCER DRUGS IN GERMANY: HTA DECISIONS OF NEW AND INNOVATIVE ONCOLOGY DRUGS IN GERMANY – AN ANALYSIS USING THE PRISMACCESS® DATABASE
- The German Pharmaceutical Market Restructuring Act (AMNOG) enters its eighth effective year.
- The aim of the law is to disclose the added benefit of a new patented drug over existing therapies by an early assessment, which also serves as the basis for the price negotiation.
- This study is a descriptive analysis of the early benefit assessments for new oncologics in Germany.
COMPARISON OF EU5 MARKET ACCESS DECISIONS FOR CROHN’S DISEASE BASED ON THE PRISMACCESS® DATABASE
- Crohn’s disease (CD) is a long term condition in which inflammation of the digestive system leads to diarrhoea, abdominal pain, tiredness and weight loss. There is no cure for CD at the moment .
- When the CD is active, drug treatment is usually used to manage the symptoms quickly and bring on remission.
- Prevalence of CD for Germany in 2010 was 322 (95% confidence interval [CI]: 302-346) per 100000. In line with worldwide reports, the numbers suggest a considerable increase for CD prevalence in Germany since the 1980s, which need to be adapted by healthcare services and dealt with the burden associated with increasing numbers of patients. 
- New therapies have been launched in Europe over the last few years. One key question remains if these new therapies reach patients in terms of market access in various European countries and how the national HTA agencies have decided on these therapies.
- This study visualizes the heterogenous multiple decisions by EU-5 HTA agencies using the Prismaccess®/Evalumade® database and their three-colored scale. Hence, this study will shed light on those decisions in a transparent and understandable manner for industry.
THE BUDGET IMPACT OF NICOTINIC SUBSTITUTE REIMBURSEMENT
At the end of 2017, the French ministry of health decided to allow the reimbursement of nicotinic substitutes in order to reduce tobacco smoking. Before this decision, patients could only benefit from a fixed e150 per-year reimbursement basis to cover nicotinic substitute expenses (patches and gum).
THE BUDGET IMPACT OF GLOBAL TELEMEDICINE SOLUTION IN FRANCE
- The global telemedicine solution allows patients to consult a physician from a distance in the same conditions as a visit in a traditional office: the physician talks with the patient and provides clinical health care, such as monitoring medical measurement or delivering prescriptions.
HARMONISATION OF EUROPEAN HTA - A ROUND TABLE DISCUSSION
- MEDVANCE, a unique health care consultancy based on five independent local consultancies, also leads local market access system debates and has invited key opinion leaders in the field of a potential European joint HTA for a round table discussion in Barcelona on 11.11.2018.