ADAPT SMART – PUBLIC SURVEY ON EMA PILOT ON ADAPTIVE PATHWAYS
Background
MAPPs* (Medicines Adaptive Pathways to Patients) seeks to foster access to beneficial treatments for the right patient groups at the earliest appropriate time in the product life-span in a sustainable fashion
In March 2014, EMA launched a pilot on Adaptive Pathways in order to explore how the adaptive pathways approach might work in the existing regulatory framework with real medicines in development (http://www.ema.europa.eu/docs/en_GB/document_library/Other/2014/03/WC500163409.pdf).
As this concept was a new one considering the proposed features (i.e. iterative development of the label, use of real world data, involvement of Health Technology Assessment bodies) , it called the interest of a number of stakeholders, primarily pharmaceutical industry but also hospitals or research organisations. Up to now EMA received 58 applications and 19 of them have been successfully progressed through the pilot .
Reference is made to the EMA Adaptive Pathways webpage and submission guidance therein.
http://www.ema.europa.eu/ema/index.jsp?curl=pages/regulation/general/general_content_000601.jsp&mid=WC0b01ac05807d58ce
On July 1st 2015, the IMI coordinated support action on ADAPT SMART kicked off (http://adaptsmart.eu/), the objective is to facilitate and accelerate availability of MAPPs through the establishment of an enabling platform for the coordination of MAPPs related activities within IMI2 and engaging dialogue with relevant stakeholders.
One of the workstream has been tasked to review lessons of EMA pilot on Adaptive Pathways. A survey has been developed to capture the experience of the industry and other organisations with the EMA pilot on Adaptive Pathways.
The survey will consist of two parts:
- One EMA questionnaire for those companies which actually submitted an application to the pilot. EMA will share it with participants directly.
- One public questionnaire for those organisations which considered applying but did NOT submit an application in the end.
The attached survey will aim at collecting feedback from those organisations which did some preparatory work in the view of applying with an asset to the EMA pilot but which give up in the end and did NOT progress with a submission.
The responses to the survey will be treated confidentially and the results will be anonymised. These results will be used for the preparation of a report on the learning s of EMA pilot on Adaptive Pathways as part of the Work Package 2 of the IMI ADAPT SMART. The report will providing a mapping of knowledge gaps and recommendations for areas for further work to be addressed by the other workstreams.
The report will be made available on the IMI ADAPT SMART website in Q2 2016.
This questionnaire will be posted on the ADAPT SMART website and shared with EFPIA and EuropaBio membership.
For any questions on the content of the survey please contact Isabelle Clamou (), Tel: +327752786
ADAPT SMART – PUBLIC SURVEY ON EMA PILOT ON ADAPTIVE PATHWAYS
The Objectives of the public questionnaire are to:
· Capture the perceived hurdles for applicants to participate in the pilot
· Capture the perceived benefits for applicants to participate in the pilot
· Get a sense of why certain assets have not been submitted
· Get feedback on the MAPPs concept
Please respond to this survey by March 25th, 2016. Completed survey should be sent to : .
For responding to questions, please refer to the glossary of terms in the Annex to this document.
Name of the organization:
If your organisation has been considering submitting an Adaptive Pathways Pilot request please answer the next series of questions:
1.1 What were the perceived potential benefits (‘wins’) for your organisation with the Adaptive Pathway concept/pilot? (Please provide your answer by putting ‘X’ on the dots. Feel free to select more than one answer)
… A) Anticipated earlier approval,
… B) Enhanced access to in depth discussion on development programme,
… C) Inclusion of stakeholders (e.g. patients, HTA bodies)
… D) Gain experience with an early dialogue in a non committal way
… E) Early discussion with payers on data to support perceived value of the asset
… F) Other: Please complete with free text: …
1.2 What were the perceived potential hurdles for your organisation with the Adaptive Pathway (AP) concept/pilot? (Please provide your answer by putting ‘X’ on the dots. Feel free to select more than one answer)
… A) Resource constraint/internal development timelines
… B) Value of the different early dialogue schemes available and their benefits and risks
… C) No guarantee of accelerated approval/faster development time
… D) Concern that pricing structure will be unfavourable
… E) Early investment in manufacturing of products
… F) Preference to use a tried-and-tested approach to data collection and not to use Real World Data as substitute to clinical trials data
… G) CMC (Chemical Manufacturing & Control) /Quality aspects may limit acceleration of conduct of studies quality data is under development.
… H) Concern over the ability to contain the prescription to the initially licensed population
… I) Uncertainty on the acceptability of the use of RWD for the iterative development of the label
… J) Impact on intellectual property incentives and overall return on investment
… K) Other: Please complete with free text:
1.3 Which grounds did you finally consider to decide NOT to participate to the EMA AP pilot? (Please provide your answer by putting ‘X’ on the dots. Feel free to select more than one answer)
… A) Asset discontinued or challenges to identify the right asset
… B) Timing (i.e. no data available in the timeframe of the pilot or too late in the development process)
… C) Portfolio prioritization
… D) Resource constraint
… E) Investment decision
… F) Other: Please complete with free text:
Questions about support of the MAPPs concept in your organization
2.1 How supportive is your organization of the adaptive pathways concept? (Please provide your answer by putting ‘X’ on the dots. Feel free to select more than one answer)
… A) Very supportive
… B) Supportive
… C) Neutral
… D) Unsupportive
… E) Very unsupportive
2.2 Please detail the main concerns of your organisation with the AP concept, as it is understood. You will find below a list of examples of some of the current perceptions (Please provide your answer by putting ‘X’ on the dots. Feel free to select more than one answer)
… A) US FDA not supportive of the AP approach
… B) Lack of global support/alignment on AP approach.
… C) AP products will never be commercially viable
… D) AP is suitable only for SMEs
… E) AP may be used retrospectively by regulators to limit the label (similar concerns with the FDA Special Medical Use proposal).
… F) Impact on Intellectual Property
… G) Stakeholders will not accept real word evidence in practice
… H) AP requires too much change to existing business model
… I) AP products are too niched and difficult to identify early in development.
… J) Concerns with the potential increased cost of post approval studies
… K) Concerns that later clinical data will not support the initial approval (need to withdraw) makes it too high risk.
… L) AP concept is too complicated!
… M) Others: Please complete with free text
2.3 What, if anything, could/would need to change to make your organisation more supportive?
Please complete with free text:
Annex Glossary of key terms
Adaptive pathways - A prospective planned approach of development involving all stakeholders to support patient access to medicinal products answering an unmet medical need. It foresees an initial marketing authorisation and reimbursement of a medicinal product in a well-defined patient subgroup and subsequent widening of the indication to a larger patient population based on additional evidence gathered and/or a conditional marketing authorisation and conditional reimbursement where initial data are confirmed through the collection of post-authorisation data on the medicinal product’s use.
Benefit - The positive therapeutic effects of a medicinal product.
Benefit-risk balance -An evaluation of the positive therapeutic effects of the medicinal product in relation to the risks.
Clinical study - Any investigation in relation to humans intended: (a) to discover or verify the clinical, pharmacological or other pharmacodynamic effects of one or more medicinal products; (b) to identify any adverse reactions to one or more medicinal products; or (c) to study the absorption, distribution, metabolism and excretion of one or more medicinal products; with the objective of ascertaining the safety and/or efficacy of those medicinal products.
Clinical trial - A clinical study which fulfils any of the following conditions: (a) the assignment of the subject to a particular therapeutic strategy is decided in advance and does not fall within normal clinical practice of the Member State concerned; (b) the decision to prescribe the investigational medicinal products is taken together with the decision to include the subject in the clinical study; or (c) diagnostic or monitoring procedures in addition to normal clinical practice are applied to the subjects.
Health Technology Assessment (HTA) - The systematic evaluation of the properties and effects of a health technology, addressing the direct intended effects of this technology, as well as its indirect unintended consequences, and aimed mainly at informing decision-making regarding health technologies.
Life span (medicinal product) - All the stages of pre- and post-marketing until the product is withdrawn from the market.
Marketing authorisation - A licence given by a regulatory authority to an applicant allowing for the marketing of a specific product within the jurisdiction of the regulatory agency. The decision for granting a marketing authorisation is based primarily on the quality, safety and efficacy of the new medicinal product and is based on the evidence of a positive benefit/risk ratio at the time of approval, i.e. the expected benefits outweigh the anticipated risks in a defined population, at a defined dosing and dose regimen, with defined conditions of use.
Medicines Adaptive Pathways to Patients (MAPPs) - An EU initiative that seeks to provide timely access to medicines with potential to address significant unmet medical needs in a sustainable way. The MAPPs’ scope covers regulatory approval, health technology assessment (HTA), pricing, reimbursement, and health care delivery. The general principle is that approval and reimbursement decisions are made using a framework which supports the launch of the therapy based on initial evidence. Additional data generated post-launch supports progressive reduction of uncertainty about benefits and risks and may lead to adjustments in utilisation and price in response to the emerging information.
Medicinal product - Any substance or combination of substances which may be administered to human beings and are presented as having properties for treating or preventing disease and/or restoring, correcting or modifying physiological functions and/or making a medical diagnosis.
Payers - Regional or national organisations responsible for managing healthcare budgets and/or responsible for providing payment/reimbursement for treatments/medical care.
Real world data (RWD) - An umbrella term for data regarding the effects of health interventions (e.g. benefit, risk, resource use, etc.) that are collected both prospectively and retrospectively from observations of routine clinical practice. RWD can be obtained from many sources including patient registries, electronic medical records, and observational studies.
Real world study (RWS) - Studies that are conducted to evaluate a health intervention in clinical practice.
Stakeholder - An individual, organisation or initiative that participates in, is involved with, influences the outcomes of, or is impacted by the outcomes of, or implications, of a certain activity.
Treatment - An intervention e.g. using a medicinal product, a surgical procedure and/or medical device to improve health or well-being.
Uncertainty - A situation where it is perceived by a stakeholder that additional information would be required to further qualify the benefits and risks and value of a medicinal product.
Unmet medical need - A condition for which there exists no satisfactory method of diagnosis, prevention or treatment authorised in the Community or, even if such a method exists, in relation to which the medicinal product concerned will be of major therapeutic advantage to those affected.