Early access authorization, compassionate access authorization and compassionate access framework to medicinal products
What is the early access program in France (previously ATU program) ?
Early access programs (EAP) are known as expanded access program (or managed access programs) in the United States. They give certain patients early access to investigational drug before they are authorized for marketing.
Indeed, for certain rare, serious debilitating disease, life threatening disease or disabling pathologies, the exceptional use of investigational drugs before obtaining their Marketing Authorization (MA) or their coverage under common law may be authorized by the competent authorities.
France have very specific local regulations for such programs and notably published a new reform in July 2021 with several objectives such as:
- Faster access to innovative treatments for patients with unmet medical need
- Attractive to pharmaceutical companies
- Financially sustainable for the french healthcare systems and pharma companies
BlueReg can advise and assist you in setting up these french expanded access programs known as early access authorizations, compassionate access authorizations and compassionate prescription frameworks.
Learn more about early access authorization and our consulting services in France below by contacting our experts.
Early Access Programs in France was originally initiated in 1992 to enable the sale of certain medicinal drugs offering solutions to serious or rare diseases ahead of any reimbursement of pricing negotiations; particularly new therapies for human immunodeficiency virus (HIV).
Since then, it has expanded to cover all areas of research and services.
Later early access programs, also called ATU (Autorisation Temporaire d’Utilisation ) have been extended in 2010 creating two systems (cohort ATU and nominative ATU also called named patient programs or ATU nominative ANSM).
The system has been further developed ending with six interlinked schemes, with different perimeters, eligibility criteria and reimbursements.
The system was simplified in July 2021, you can learn more about this new system here.
Early Access Programs in France compared to MAA from FDA & EMA regulatory approval:
French regulatory authorities (ANSM) recently published a retrospective study on access time for anticancer drugs, comparing FDA, EMA and the French ATU program (cohort or named patient program) scheme over 13 years:
- In France, the average time between Marketing Authorization and time when patient gain access to reimbursable drugs is 530 days, which has led to numerous criticisms about delays in access to therapeutic innovation compared to other European countries or those worldwide such as the USA.
- However, these criticisms do not take into account the early availability of medicines, well before MA and reimbursement, via ATU, which makes it possible to use, on an exceptional basis and over a limited but potentially renewable period, a drug product that does not have an MA and is not available to patients through a clinical trial.
- The purpose of this study is to analyze the average time for access to antineoplastic treatments, taking into account ATU, in relation to the date of US FDA and EMA approval
* FDA: Food and Drug Administration / EMA: European Medicines Agency
Why pharma companies should consider initiating early access programs in France even during Phase 3 clinical trial?
Between 1st January 2007 and 31st December 2019, ANSM evaluated and granted early access program in oncology to 36 antineoplastic drugs.
Thanks to early access program granted, almost 70% (25 out of 36) of investigational drugs were made available in France before FDA approval.
Thanks to early access program, drugs are on average available in France 200 days before their first regulatory approval (FDA or EMA).
We are supporting today more and more companies with French early access programs set-up. For instance, PharmaBlue (our French affiliate) as French “exploitant” company can provide operational, pharmacovigilance and regulatory support in the management of early access programs in France.
Reminder: French regulatory authorities require having an « Exploitant » authorized and established in France to manage an early access program.
Indeed, Pharma Blue today partners with a lot of US companies looking to outsource early access program in France and Exploitant responsibilities in the field of rare or serious disease & unmet medical need.
Benefits of early access program in France for pharma companies:
- Allowing continuous access to the investigational drug for patients
- Building the company image before commercial launch
- Generate turnover before marketing approval by regulatory agencies
- Setting the scene for future market access of commercial product
French Early Access Program management for a US pharmaceutical company
An American pharmaceutical company starting to build its European affiliates entrusted PharmaBlue with the responsibility of submitting, obtaining, and implementing...Learn more
More information about the new early access program regulation
Two new Medicare access and coverage arrangements went into effect on July 1, 2021:
Pre-marketing authorization (formerly cohort Temporary Authorization for Use – ATUc) and post-marketing authorization (formerly Temporary Funding Scheme – PECT) and the early access or “accès précoce”, which targets drugs that meet an unmet therapeutic need, are likely to be innovative and for which the laboratory undertakes to file an MA or an application for reimbursement.
Compassionate use authorizations (AAC, formerly ATUn) and compassionate prescription schemes (formerly RTU) – the compassionate access or “accès compassionnel”, which is aimed at medicinal products that are not necessarily innovative, that are not initially intended to obtain marketing authorization but that satisfactorily meet an unmet therapeutic need.
BlueReg have specific expertise for market access and reimbursement process in France which allows patients access and the possibility of reimbursement according to established rules. We provide full support, or we can adapt our services according to the support requested.
Promoting rapid, broad and controlled access to innovation – Patients access
At BlueReg, we advise our clients on access and reimbursement schemes, on the impact and effects on clinical trial programs, and on specific marketing considerations, such as product labeling.
Accelerated review pathways in some countries (e.g. PRIME in Europe or Fast Track in the United States (US)) are also covered by our regulatory consulting services.
Early Access – “Accès précoce”
Early access targets medical needs that can be met by investigational therapies for which the pharmaceutical company has a commercial focus.
Early Access Programs eligibility criteria:
The following five eligibility requirements must be met:
- Efficacy and safety are strongly presumed based on the results of clinical trials when the drug does not have a marketing authorization in the indication considered
- The drug must be intended to treat serious, rare or disabling diseases
- There is no suitable treatment options available
- The implementation of the treatment cannot be deferred
- The medicinal product is presumed to be innovative, particularly with regard to a possible clinically relevant comparator
These authorizations may concern the indication:
- of a drug prior to obtaining any marketing authorization. In this case, the ANSM (French national health authority) must issue an opinion on its efficacy and safety based on the results of clinical trials before the HAS (Haute Autorité de Santé) can make a decision
- a drug that already has a marketing authorization for the indication in question, before it is covered by the health insurance system
Compassionate access covers two distinct cases that share the fact that they concern a drug that treats eligible patients suffering from illnesses without appropriate treatment, in a given therapeutic indication without being intended to obtain a marketing authorization in France.
Compassionate access targets medical needs that can be met by drugs for which the laboratory does not have a commercial strategy.
This compassionate access route is requested for an unauthorized drug not available in France by a hospital prescriber (treating physician request) for a named patient, provided that the ANSM is able to assume a favorable benefit/risk ratio for a serious, rare or incapacitating disease: this is a named compassionate access authorization (“autorisation d’accès compassionnel nominative ”).
Alternatively, at the request of the ANSM, with a view to prescribing a drug available in France without a MA for the indication but with a marketing authorization for other well established indications this is a known as compassionate prescription framework.
An exemption to compassionate access has been provided for, in order to allow nominated access to drugs in development: this is “pre-early” or “very early” compassionate access.
BlueReg Support for pharmaceutical companies looking to run French expanded access programs
As we have seen, there are some situations where patients have a life threatening or orphan disease, and their physicians have exhausted all available treatment options or there is simply no treatment. Some country-specific mechanisms exist across the globe to enable access to pre-approved or unlicensed medicines outside of clinical trials.
These mechanisms support and an earlier access to more and more innovative medicines for patients.
- PharmaBlue is composed of experts in preparation and submission of early access dossiers in compliance with the current regulations and also through the new pilot schemes.
- PharmaBlue has the skills and expertise to implement early access programs and patient follow-up in line with the current regulations and with pharmacovigilance (PV) requirements.
- The PharmaBlue team worked in close collaboration internally and with the client.
- Preparation and writing of early access dossier including the letter of intent, the application form, justification part and the Protocol for Therapeutic Use and information collection (PUT-RD)
Operational activities and project management
- PharmaBlue has set up an early access task force in order to ensure the validation of patient inclusion, drug orders, patient follow-up in the program.
- Patients’ data collected by PharmaBlue were analyzed for early access report writing.
- Advice for early access renewal dossier, MA dossier.
- Advice and warning on the specificities of the French regulations, particularly with regards to the different early access schemes and follow-up activities once early access ended.
- Management of PV cases (analysis of PV cases and submission to the French authorities).
- Management of medical information requests.
- Management and follow-up of treatment batches.
- Management of quality complaints.
Benefits for our clients
Peace of mind operations on French Market + Immediate activation of French affiliate
+ Access to rare skills and experienced early access teams in France
Drug Development Consulting & Strategy
Currently developing a new medicinal product ? BlueReg can provide an end-to-end project plan to guide you from the development of your medicinal product right through to authorization and beyond. We can provide advice and recommendations to ensure the shortest time to market by minimising any possible barriers during the development process and beyond.
Advanced Therapy Medicinal Product (ATMP)
Looking for an Advanced Therapy Medicinal Product (ATMP) classification? BlueReg can guide you to submit a classification application to Health Authorities to determine if your product meets the requirements to be defined as an ATMP.
Scientific Advice & Interaction with Health authorities
BlueReg has extensive experience in scientific advice with the European Medicines Agency (EMA) and many national competent authorities. BlueReg can assist with all competent authority meetings.
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Looking for support with your EMA / HTA bodies Parallel Consultation? BlueReg can provide a team of highly experienced consultants specialised in regulatory affairs, market access and scientific writing. Our consultants can advise you on all steps of the procedure and author the required documents required for this important process.