Managed Access Programs (MAPs) provide patients with anticipated access to drugs before their full-fledged commercialization. As often seen in Europe, the situation is complex: on one hand, MAPs can refer to several different schemes within a country, on the other hand MAPs are regulated at country level, with almost as many situations as there are countries. Yet, MAPs are an integral part of a successful market entry strategy for Europe. How can you maximize your chances to implement a successful MAP that will help the patients in need and speed up your adoption by the medical community?
What are MAPs and why are they necessary?
While market authorization can now be granted at a European Level, “Pricing and Reimbursement” are still the prerogative of each Member State: in some countries, a drug can fall into the paradoxical state where it is approved and yet cannot be prescribed, for lack of reimbursement. To mitigate this problem, Managed Access Programs have emerged in most European countries.
MAPs are programs designed to accelerate patient access to new, efficacious drugs, in an ethical, compliant, and controlled way until they become fully marketed (approved, with price and reimbursement schemes in place). MAPs bridge the gaps that exist between clinical trials and commercially available drugs: they are usually set up once phase III results are known, and the marketing authorization has been filed, i.e. even before the marketing authorization is granted.
For the sake of clarity, MAPs in this article will be considered to be mostly Compassionate Use Programs, but be aware that the term “MAP” sometimes has a broader definition and could be used interchangeably with – or include – programs such as Early Access Programs, conditional approval, PriMe (Priority Medicine scheme) …
Even within a given country, MAPs could either concern individual patients or groups of patients. The first case is referred to as NPP for Named Patient Program, the second as Cohort Programs.
Who will benefit from a MAP?
Getting access to effective medicine when no other option is available: the benefit to the patient is obvious.
One way for patients to receive treatment early is to be included into clinical trials (e.g. Open label extension after the randomized phase of the pivotal trial), but not all patients are eligible to participate in the trials, which are protocol-driven and have stringent inclusion and exclusion criteria. Thus, MAPs are often the only option for patients who cannot wait for the drug to be commercialized and cannot enter clinical trials.
Furthermore, MAPs are validated by local authorities and the drug can only be distributed through well defined channels, reducing the risk for counterfeit drugs.
For physicians, MAPs are extremely useful as well, because on top of the direct benefit it provides to their patients, it also allows them to better understand the drug and have their first hands on experience with a new drug.
It can also please the early adopters within the physician population, who, once convinced of the benefits the drugs bring to patients, can become the best brand advocates, whether to recommend to their colleagues or to intervene as experts during pricing and reimbursement negotiations.
Most compassionate use programs require the drug to be provided free of charge by the market authorization holder (MAH). Some named patient programs and the Autorisation Temporaire d’Utilisation in France (ATU, ‘Temporary Authorization for Use’) are the exception to the rule: the healthcare system would pay for the drug in this case.
The MAH still takes care of the pharmacovigilance (PV): this provides safety data from a larger population sample in a more “real-life” setting, which helps better assess the safety, tolerability, and Quality of Life aspect of the reimbursement and pricing negotiations.
The market authorization holder
Despite having to provide the drug for free in most cases and assuming the costs of PV, some associated benefits have been noticed as MAPs became more common:
- Patient-centric company image: MAPs show the goodwill of the company towards patient needs, which helps strengthen the ties between the MAH, the medical community (both KOL and community prescribers) and the patient advocacy groups. Furthermore, it increases the likelihood that patients try the treatment offered through the MAP instead of others.
- Spontaneous awareness: More physicians and patients will experience the benefits associated with the treatment, which will likely fuel discussion among patient groups. This increased spontaneous (i.e. non-promoted) awareness will more likely speed up the widespread medical adoption once the drug is commercialized. It is likely it has an impact on the 1st year market share[i] (a gain close to 1/3 in terms of market share was loosely associated with the implementation of a NPP in a small retrospective study).
However, one cannot stress enough that the primary goal of a compliant MAP should be to provide efficacious treatments to patients with unmet needs.
Diversity of schemes across Europe
The diversity of programs available in France (FR), Germany (DE), Italy (IT), Spain (ES) & the United Kingdom (UK) illustrates the complexity of setting up a European MAP for a newly approved drug:
Absence of global EU framework: Within the scope of Article 83 of Regulation (EC) No 726/2004, the EMA provides recommendation through the Committee for Medicinal Products for Human Use (CHMP), but does not create a single legal framework.
There are 5 different authorities to coordinate:
- Agence nationale de sécurité du médicament et des produits de santé (ANSM, FR)
- Bundesinstitut für Arzneimittel und Medizinprodukte (BfArM, DE) & Paul-Ehrlich-Institute (PEI, DE)
- Agenzia Italiana del Farmaco (AIFA, IT)
- Agencia Española de Medicamentos y Productos Sanitarios (AEMPS, ES)
- Medicines and Healthcare Products Regulatory Agency (MHRA, UK)
Diversity in program types: Cohort programs only (DE), Named Patient Program only (UK) or both cohort and NPP available (FR, ES, IT).
European MAPs are more complex than their US counterpart, with no centralized approach (yet). However, and despite their non-negligible cost, they provide benefits for all stakeholders within the healthcare ecosystem, including the market authorization holder. Thus, MAPs are an integral part of strategy to foster fast adoption by the medical community but require careful planning to reach their goals while keeping expenditure under control.
Cepton Strategies will be attending the JP Morgan conference in January. We would be delighted to discuss these points with you in detail and address your concerns for the European market. Please click here for more information and to get in touch.
[i] A.K. Bates, Journal of Medical Marketing (2008) 8, 319 – 324