AdaptSmart

Beneficial treatments

MAPPs is not a rush to expose patients to untested treatments. Clinical data must show that the benefits will very likely outweigh the risks in the defined patient group.


Key questions addressed by ADAPT SMART:
 

  • How can sufficient evidence be gathered in a MAPPs pathway to demonstrate a positive benefit-risk balance?
  • How can Real World Data (RWD) be used to bridge the efficacy to effectiveness gap?
  • What are patient's and health care providers’ (HCPs) views on MAPPs?
  • How is the collection of evidence for quality, safety and efficacy evaluations governed within a MAPPs approach?

How can sufficient evidence be gathered in a MAPPs pathway to demonstrate a positive benefit-risk balance?

Lessons from ADAPT SMART

 

  • A prerequisite of MAPPs is stakeholder agreement on a prospective plan about evidence generation throughout the medines life cycle.
     
  • A core principle of the MAPPs pathway is the use of Real World Data (RWD) to supplement randomized clinical trial data.
     
  • Robust tools to appropriately collect, analyse and interpret RWD, the use of (conditional) marketing authorisation based on single-arm studies and improved methods to integrate network meta-analysis in regulatory decision-making will be a key facilitator of the MAPPs approach. 
     

Download links

ADAPT SMART report: Gathering evidence through real-life use to supplement clinical trial data - Executive summary only (pending publication)
 

ADAPT SMART report: Indirect comparisons (network meta-analysis) and single arm studies - Executive summary only (document under review)

How can Real World Data (RWD) be used to bridge the efficacy to effectiveness gap?

Lessons from ADAPT SMART

 

  • The use of RWD (e.g. registries, electronic health records etc.) to supplement data from randomised clinical trials can speed up the decision-making process.
     
  • Integrating evidence from different sources is key to satisfying different stakeholder needs which need to be considered over the entire life cycle of a product.
     
  • The infrastructure to study disease epidemiology and relative effectiveness of medicines needs to be further developed.

 


Download links

ADAPT SMART workshop report: Select methods, toolset and datasets to perform scenario studies

What are patient's and health care providers’ (HCPs) views on MAPPs?

Lessons from ADAPT SMART

 

  • Patients pointed out the need for a close consideration of the trade-off between benefits and risks in MAPPs.
     
  • A preliminary study showed that HCPs are willing to provide patients with the appropriate information, reasons and advantages of joining a disease registry, when considering a MAPPs product.
     
  • HCPs expressed an unanimous willingness to be part of the iterative decision-making process when developing, licensing and considering the financial coverage of a MAPPs product.  

 


Download links

ADAPT SMART report: Inventory and Analysis of Increased Uncertainties of Health Care Providers, Patients and other Stakeholders - Executive summary only (document under review)
 

ADAPT SMART workshop report: Patient uncertainties and exploring the ethical and legal considerations of MAPPs

How is the collection of evidence for quality, safety and efficacy evaluations governed within a MAPPs approach?

Lessons from ADAPT SMART

 

  • The requirement to demonstrate a positive benefit-risk balance through a MAPPs approach will not be different from medicinal products that obtain a market authorisation via a standard procedure.
     
  • No legal hurdles have been identified in a MAPPs approach with respect to the framework governing the approval of medicinal products and market access rules.
     
  • To what extent some uncertainties may remain with regard to non-legislative provisions need to be further assessed.

 


Download links

ADAPT SMART report: Legal constraints with regard to MAPPs
 

ADAPT SMART report: Impact of adaptive licencing and adaptive access on IP and regulatory exclusivity right periods

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Right patient groups

Under MAPPs, a product is initially “fast-tracked” exclusively for patients who will benefit the most. It is therefore important that in the beginning only those patients will get the new medicine.


Key questions addressed by ADAPT SMART:
 

  • How can iterative developments in MAPPs support the managed introduction of new treatments for the right patient groups?
  • What are the main uncertainties for patients and how can they be mitigated?
  • How can we ensure that medicinal products approved under MAPPs are appropriately used?
  • Could restricting prescribing of medicinal products to a defined patient group raise new ethical or legal considerations?

How can iterative developments in MAPPs support the managed introduction of new treatments for the right patient groups?

Lessons from ADAPT SMART

 

  • In the MAPPs context, an iterative development means approval in stages, starting with a restricted patient population and then expanding to wider patient groups.
     
  • Iterative development will rely on well-informed decisions about what the target patient population that receives initial approval should be and which patient populations are subject of possible additional indications.
     
  • New scientific developments related to the growing stratification of treatment-eligible populations and methods to support decisions based on smaller samples are currently under development.

 


Download links

ADAPT SMART report: Approval in stages - Iterative development in MAPPs - Executive summary only (pending publication)

What are the main uncertainties for patients and how can they be mitigated?

Lessons from ADAPT SMART

 

  • Patient perspectives are full of questions but one certainty: there is no compromise on safety.
     
  • Mitigation of patient uncertainties requires continuous engagement of all stakeholders.
     
  • There is a key role of patient organisations and patient advocates as information providers.

 


Download links

ADAPT SMART report: Inventory and Analysis of Increased Uncertainties of Health Care Providers, Patients and other Stakeholders - Executive summary only (document under review)
 

ADAPT SMART workshop report: Patient uncertainties and exploring the ethical and legal considerations of MAPPs

How can we ensure that medicinal products approved under MAPPs are appropriately used?

Lessons from ADAPT SMART

 

  • A variety of different tools and systems are in place in EU Member States to guide the appropriate use of medicinal products, including treatment guidelines, diagnostic tools, restrictions by prescriber speciality and registries.
     
  • Different enforcement mechanisms exist in EU Member States for managing non-compliance to tools/systems that guide appropriate use.
     
  • More alignment is needed between EU Member States with regard to the implementation of tools and systems for guiding appropriate use in the MAPPs context.

 


Download links

ADAPT SMART report: An overview of tools and systems to guide appropriate use of medicinal products in twelve EU Member States
 

ADAPT SMART workshop report: Tools and systems used for prescription control & the ethical and legal aspects of adaptive decision making

Could restricting prescribing of medicinal products to a defined patient group raise new ethical or legal considerations?

Lessons from ADAPT SMART

 

  • Any prescribing tools imposed should not impact on healthcare professionals’ freedom to prescribe under the duty of care for the patient.
     
  • The use of disease registries to support data collection is preferable, but not available or practical in all EU Member States, as well as practical options to meet the data needs of all stakeholders will need extra consideration.
     
  • Governing and recording prescribing behaviour(s) may be harder in some healthcare systems with fragmented systems, such as those with parallel private and public healthcare systems.

 


Download links

ADAPT SMART report: Ethical And Legal Aspects Of Prescribing And Use By Target Populations
 

ADAPT SMART workshop report: Tools and systems used for prescription control & the ethical and legal aspects of adaptive decision

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Sustainable fashion

High drug prices have become a topic of debate that could block early access. The MAPPs concept seeks to find solutions for this emerging financial constraint by ensuring the sustainability of innovation and healthcare systems.


Key questions addressed by ADAPT SMART:
 

  • How can flexible pricing and reimbursement schemes contribute to a MAPPs approach in a sustainable healthcare system?
  • How could stakeholders effectively sustain resource mobilisation over the lifetime of a medicinal product?
  • How can the impact of a decision to disengage from MAPPs be managed?
  • Can the legal and ethical aspects of guiding the appropriate use of medicinal products be dealt with in a sustainable fashion?
  • How do the current exclusivity rights work for a product developed under MAPPs?

How can flexible pricing and reimbursement schemes contribute to a MAPPs approach in a sustainable healthcare system?

Lessons from ADAPT SMART

 

  • Early dialogue with HTA bodies and national payers is critical to understand how (cost-) effectiveness could be assessed under MAPPs in order to agree on pricing and reimbursement schemes.
     
  • Making better use of post-marketing evidence generation to facilitate outcomes-based arrangement can support the managed introduction of a MAPPs product.
     
  • A flexible pricing model with variable discounts to manage an evolving evidence base might be acceptable for both payers and manufacturers.

 


Download links

ADAPT SMART report: Addressing uncertainty - managed entry agreements for pharmaceuticals in the context of adaptive pathways in Europe - Executive summary only (pending publication)
 

ADAPT SMART workshop report: Managed entry in the context of adaptive pathways

How could stakeholders effectively sustain resource mobilisation over the lifetime of a medicinal product?

Lessons from ADAPT SMART

 

  • Political and financial support for MAPPs concept is needed for all stakeholders to engage effectively and sustainably.
     
  • Identify and pilot methods to evaluate how engagement criteria can be sustained over multi-year engagement approach.
     
  • Utilizing existing platforms that already involve processes such as early dialogues and multiple stakeholder collaboration could help streamline resource requests.

 


Download links

ADAPT SMART report: Implementing adaptive pathways - decision points and resource implications
 

ADAPT SMART report: Seamless Process and Decision Points of an Adaptive Pathway
 

ADAPT SMART workshop report: The seamless pathway and decision making

How can the impact of a decision to disengage from MAPPs be managed?

Lessons from ADAPT SMART

 

  • Stakeholder remits and decision making remain unchanged under MAPPs.
     
  • Multi-stakeholder dialogues create an interdependency and a moral obligation to inform other stakeholders of a decision and to consider the impact to other stakeholders.
     
  • Principles and process for stakeholder engagement and disengagement should be explicit and documented.

 


Download links

ADAPT SMART report: Enablers of decision making in an adaptive environment: managing uncertainties and disengagement strategies - Executive summary only (document under review)

Can the legal and ethical aspects of guiding the appropriate use of medicinal products be dealt with in a sustainable fashion?

Lessons from ADAPT SMART

 

  • Managed access agreements can help guide healthcare professional and patient dialogues as they detail items such as; patient’s eligibility to receive a medicine, additional data collection needs and alternative treatment options should access change.
     
  • Greater understanding of the legal and ethical frameworks, and societal expectations of prescribing behaviour across Member States would help support appropriate prescribing.
     
  • There remains a clear case for further empirical research of the legal tools available with which to enact prescription control, the ethical frameworks and related socioeconomic variables and expectations across EU Member States.

 


Download links

ADAPT SMART report: Ethical And Legal Aspects Of Prescribing And Use By Target Populations
 

ADAPT SMART workshop report: Tools and systems used for prescription control & the ethical and legal aspects of adaptive decision making

How do the current exclusivity rights work for a product developed under MAPPs?

Lessons from ADAPT SMART

 

  • MAPPs can work well within a predictable way within the current incentive and exclusivity regime – no legal blocks exist.
     
  • Early and prospective dialogue and considerations of all tools available is important to be able to clearly define the market value for medicine developers.
     
  • Any change in the current legal framework of incentives or rewards will impact medicinal products; it is therefore vital that the MAPPs concept is considered adequately alongside other existing instruments.

 


Download links

ADAPT SMART report: Impact of adaptive licencing and adaptive access on IP and regulatory exclusivity right periods
 

ADAPT SMART workshop report: The Impact of MAPPs and adaptive access on IP and regulatory exclusivity rights

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Earliest appropriate time

Once it is determined there is a patient group with an unmet medical need and a medicine in development is very likely to offer help, the goal of MAPPs is to bring the treatment to market at the earliest appropriate time in the product life-span.


Key questions addressed by ADAPT SMART:
 

  • How can licensing and reimbursement at the earliest appropriate time be achieved?
  • How can early access to novel treatments through a MAPPs approach be achieved within the current regulatory framework
  • What resource considerations are needed by stakeholders to permit early and iterative multi-stakeholder dialogues?
  • Are there legal or ethical considerations when prescribing a product approved under MAPPs?

How can licensing and reimbursement at the earliest appropriate time be achieved?

Lessons from ADAPT SMART

 

  • MAPPs will facilitate the utility of both pre- and post-marketing authorisation (MA) generated data, allowing regulators to make earlier, well-informed decisions and re-evaluate decisions as more information becomes available.
     
  • Early interactions with payers and greater shared experiences of pricing and reimbursement authorities could expedite local pricing and reimbursement decisions and assist in early access to novel treatments from a MAPPs approach.
     
  • Outcomes-based arrangements can support the managed introduction of a MAPPs product where additional data needs to be collected.

 


Download links

ADAPT SMART report: Seamless Process and Decision Points of an Adaptive Pathway
 

ADAPT SMART workshop report: The seamless pathway and decision making
 

ADAPT SMART report: Addressing uncertainty - managed entry agreements for pharmaceuticals in the context of adaptive pathways in Europe - Executive summary only (pending publication)

How can early access to novel treatments through a MAPPs approach be achieved within the current regulatory framework

Lessons from ADAPT SMART

 

  • In order to achieve timely access for patients to beneficial treatments, the MAPPs concept foresees utilizing existing regulatory approval pathways without changing the current regulatory standards for evaluation.
     
  • Conditional marketing authorisations (CMAs), approvals under exceptional circumstances and "PRIME" are tools and procedures that are considered key to obtaining earlier authorisation for medicinal products addressing an unmet medical need.
     
  • No legal hurdles with regard to market access were identified relating to products that were approved on a conditional basis or under exceptional circumstances.

 


Download links

ADAPT SMART report: Legal constraints with regard to MAPPs
 

ADAPT SMART report: Impact of adaptive licencing and adaptive access on Intellectual property and regulatory exclusivity right periods

What resource considerations are needed by stakeholders to permit early and iterative multi-stakeholder dialogues?

Lessons from ADAPT SMART

 

  • Pricing and reimbursement authorities, patients, and healthcare providers have the greatest resource needs to engage with MAPPs.
     
  • New knowledge of disease science, regulatory processes (such as conditional marketing authorisation), and scientific methods of trial design and data collection are needed.
     
  • Appropriate representativeness and accountability of individual expert advice compared to those from umbrella organisations requires careful prospective planning.

 


Download links

ADAPT SMART report: Implementing adaptive pathways - decision points and resource implications
 

ADAPT SMART workshop report: The seamless pathway and decision making

Are there legal or ethical considerations when prescribing a product approved under MAPPs?

Lessons from ADAPT SMART

 

  • Products from a MAPPs pathway are approved by the European Medicines Agency by demonstrating a positive benefit-risk balance, for which the provision of additional data, such as long-term safety, may be a condition of the approval.
     
  • Prescribers need additional education on the concept of a conditional approval, ongoing data collection needs, and post authorisation commitments.
     
  • A mechanism is needed to better facilitate the cycle of communication between patients and healthcare professionals as to the expected risk and benefits, data collection needs, and what may happen if access changes at a later stage.

 


Download links

ADAPT SMART report: Ethical and Legal Aspects of Adaptive Decision Making
 

ADAPT SMART workshop report: Patient uncertainties and exploring the ethical and legal considerations of MAPPs

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High unmet medical needs

MAPPs focuses on products that offer a credible promise to address a patients’ unmet medical need in a meaningful way. A patients’ suffering is not enough reason to allow products on the market, reimbursed, that will do more harm than good.


Key questions addressed by ADAPT SMART:
 

  • What constitutes a high unmet medical need in the context of MAPPs?
  • How can stakeholders agree on what constitutes a high unmet medical need and how this justifies a MAPPs approach?
  • Can we stratify target populations with regard to the degree of unmet medical need?
  • What is needed to help patients take a role in ensuring early access to medicines?

What constitutes a high unmet medical need in the context of MAPPs?

Lessons from ADAPT SMART

 

  • An important building block of MAPPs is a clear focus on the promise to address a high unmet medical need.
     
  • In the MAPPs context, high unmet medical need refers to life threatening or severely debilitating conditions for which no treatment or no satisfactory treatment exist.
     
  • Early stakeholder dialogue should facilitates stakeholder agreement about defining a high unmet medical need in patient population to be considered for a MAPPs approach.

 


Download links

ADAPT SMART report: Seamless Process and Decision Points of an Adaptive Pathway
 

ADAPT SMART workshop report: The seamless pathway and decision making
 

ADAPT SMART report: Medicine Adaptive Pathways to Patients: why, when and how to engage?

How can stakeholders agree on what constitutes a high unmet medical need and how this justifies a MAPPs approach?

Lessons from ADAPT SMART

 

  • A key challenge in enabling MAPPs will be to reach an agreement about the meaning of high unmet medical need that satisfies each stakeholders involved in the MAPPs approach.
     
  • Payers in Europe have expressed a desire to reach a shared understanding of priority medicines that qualify for innovative access schemes such as adaptive pathways, which can support the discussion about unmet medical need.
     
  • Patient input is desirable when identifying areas of high unmet medical need and what outcomes are meaningful.

 


Download links

ADAPT SMART report: Shaping adaptive pathways: multi-stakeholder collaboration and unmet need - Executive summary only (pending publication)
 

ADAPT SMART workshop report: Scenario studies in order to facilitate stakeholder’s engagement  - unmet medical need

Can we stratify target populations with regard to the degree of unmet medical need?

Lessons from ADAPT SMART

 

  • The rationale behind an earlier access approach for patients with high unmet medical need is that a higher uncertainty relating to both safety and efficacy could be acceptable justifying shorter and/or smaller trials to assess treatment effects.
     
  • The involvement of health care providers and patients in discussions about selecting patients that could benefit most from the medicine is key to a successful MAPPs pathway.
     
  • Because different (sub-) populations face different degrees of unmet medical need and will benefit differently from a given treatment, on-market utilisation should be in line with the target population agreed by the stakeholders.

 


Download links

ADAPT SMART report: Medicine Adaptive Pathways to Patients: why, when and how to engage?
 

ADAPT SMART report: Tools and systems to guide appropriate use of medicinal products in twelve EU Member States

What is needed to help patients take a role in ensuring early access to medicines?

Lessons from ADAPT SMART

 

  • Patients need to take part in the discussion about perceptions of unmet medical need and added therapeutic value
     
  • Patient education could be facilitated through patient led education and joint efforts with other stakeholders, on the tools and methods of early access and MAPPs.
     
  • Prioritise stages where patient engagement across the lifecycle of medicine development is lacking or needs improving, such as; research priority setting, early dialogues and clinical trial design.

 


Download links

ADAPT SMART report: Inventory and Analysis of Increased Uncertainties of Health Care Providers, Patients and other Stakeholders - Executive summary only (document under review)
 

ADAPT SMART workshop report: Patient uncertainties and exploring the ethical and legal considerations of MAPPs

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Stakeholder collaboration

MAPPs emphasises the collaboration between stakeholders throughout the product’s life-span. In the regulatory pathway of MAPPs, stakeholders work in parallel, rather than in sequence as is currently the case. 


Key questions addressed by ADAPT SMART:
 

  • Why, when and how to engage in MAPPs?
  • How can universal engagement criteria inform the decision to select novel treatments for a MAPPs pathway?
  • How can we ensure a seamless pathway in a MAPPs approach?
  • What is the role of payers in timely access to new treatments through MAPPs in an affordable healthcare systems?
  • Can we learn from European multi-stakeholder initiatives in discussions about early stakeholder engagement in MAPPs?

Why, when and how to engage in MAPPs?

Lessons from ADAPT SMART

 

  • MAPPs is a scientific concept based on coordinated and repeated multi-stakeholder dialogue throughout the entire product life-span.
     
  • The nature of the early informal, and later formal multi-stakeholder interactions, will require a delicate balance between maintaining appropriate levels of transparency versus confidentiality.
     
  • Many aspects still need to be addressed before MAPPs can become a reality, such as how each stakeholder can allocate sufficient resources to ensure a collaborations throughout the products life-span.

 


Download links

ADAPT SMART report: Medicine Adaptive Pathways to Patients: why, when and how to engage? - Executive summary only (pending publication)

How can universal engagement criteria inform the decision to select novel treatments for a MAPPs pathway?

Lessons from ADAPT SMART

 

  • A number of MAPPs engagement criteria need to be addressed and agreed on by stakeholders when considering the MAPPs pathway for a given medicinal product.
     
  • The decision to select or de-select a specific product will depend on circumstances and can only be made on a case-by-case basis, requiring consensus among all stakeholders.
     
  • A successful MAPPs approach depends on the willingness and ability of all involved stakeholders to participate in the lifecycle of the MAPPs pathway; through appropriate and constructive discussions.

 


Download links

ADAPT SMART report: Discussion paper on Engagement Criteria for MAPPs
 

ADAPT SMART workshop report: Selection Criteria for MAPPs - Exploring and aligning stakeholder needs
 

ADAPT SMART Blog: Reflections on the workshop’s outcomes

How can we ensure a seamless pathway in a MAPPs approach?

Lessons from ADAPT SMART

 

  • Pre-defined core moments of multi-stakeholder engagement and assessment can be used to map out a conceptual MAPPs approach within the current European and national legal framework.
     
  • An iterative development plan can be the vehicle for multi-stakeholder engagement at critical development moments in the post-authorisation setting of a MAPPs pathway.
     
  • Further research needs to be done to define appropriate disengagement strategies if certain stakeholder needs are not met.

 


Download links

ADAPT SMART report: Seamless Process and Decision Points of an Adaptive Pathway
 

ADAPT SMART workshop report: The seamless pathway and decision making

What is the role of payers in timely access to new treatments through MAPPs in an affordable healthcare systems?

Lessons from ADAPT SMART

 

  • Payers have a crucial role in facilitating the early access of new treatments for patients.
     
  • The early involvement of payers in the MAPPs approach is essential to address the data needs from payers early on in the process.
     
  • Learnings and achievements from current payer initiatives and projects from different EU regions can inform next steps on payer involvement in MAPPs.

 


Download links

ADAPT SMART report: Payer requirements - The crucial role of payers in facilitating timely access to new treatments - Executive summary only (document under review)

Can we learn from European multi-stakeholder initiatives in discussions about early stakeholder engagement in MAPPs?

Lessons from ADAPT SMART

 

  • Multi-stakeholder collaboration and dialogue is a key condition for the success of MAPPs in the future.
     
  • We can learn from several existing platforms and other initiatives in Europe that have been built around stakeholder collaboration, involving payers and HTA.
     
  • Progress in currently on-going projects around patient involvement might contribute to exploring the MAPPs concept further through the better involvement of patients in parallel scientific advice procedures.

 


Download links

ADAPT SMART report: Early stakeholder involvement in discussions on a medicine’s development - Executive summary only (pending publication)

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