From adaptive licensing to adaptive pathways: Delivering a flexible life‐span approach to bring new drugs to patients

Eichler, H. G.; Baird, LG; Barker, Richard; Bloechl-Daum, Brigitte; Kristensen, Finn Børlum; Brown, Jeffrey S.; Chua, Rachel Choon-Rong; Signore, Susanna Del; Dugan, Ute; Ferguson, John; Garner, Sarah; Goettsch, Wim; Haigh, J. R. M.; Honig, Peter K.; Hoos, Anton; Huckle, P; Kondo, Tatsuya; Cam, Yann Le; Leufkens, Hubert G. M.; Lim, Robyn; Longson, Carole; Lumpkin, Murray; Maraganore, John M.; O’Rourke, Brian; Oye, Kenneth A.; Pezalla, Edmund J.; Pignatti, Francesco; Raine, June; Rasi, Guido; Salmonson, Tomas; Samaha, Dima; Schneeweiß, Sebastian; Siviero, PD; Skinner, Matthew; Teagarden, J. Russell; Tominaga, Toshiyoshi; Trusheim,; Tunis, Sean; Unger, T. F.; Vamvakas, Spiros; Hirsch, Gigi

doi:10.1002/cpt.59

Cited by 169 publications

(149 citation statements)

References 35 publications

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“…The latest EMA tendency is to fast-track drugs to marketing [11], so as to make new drugs available to patients as soon as possible. In practice, once preliminary efficacy and safety have been assessed, evaluations of comparative efficacy and cost-effectiveness are devolved to national authorities.…”

Section: Registrationmentioning

confidence: 99%

Competition in pharmaceuticals: more product- than price-oriented?

Garattini

Padula

2017

Eur J Health Econ

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Section: Registrationmentioning

confidence: 99%

Competition in pharmaceuticals: more product- than price-oriented?

Garattini

Padula

2017

Eur J Health Econ

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“…For example, a treatment on a 'lower' matrix position must provide stronger evidence to obtain the same E-score as a treatment placed in a higher square. Whether such trade-offs are adequate or necessary is a matter of debate in the field [13]. These considerations are very complex, and often there may not be a 'correct' answer but different answers based on cultural, personal and other contextual differences.…”

Section: • Limitations Of Our Methodsmentioning

confidence: 99%

Dynamic value assessments in oncology supported by the PACE Continuous Innovation Indicators

Paddock¹,

Goodman

Shortenhaus

et al. 2017

Future Oncol.

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Aim: Several recently developed frameworks aim to assess the value of cancer treatments, but the most appropriate metrics remain uncertain. Methods: We use data from the Patient Access to Cancer care Excellence Continuous Innovation Indicators to examine the relationship between hazard ratios (HRs) from clinical trials and dynamic therapeutic value accumulating over time. Results: Our analysis shows that HRs from initial clinical trials poorly predict the eventual therapeutic value of cancer treatments. Conclusion: Relying strongly on HRs from registration trials to predict the long-term success of treatments leaves a lot of the variance unexplained. The Continuous Innovation Indicators offer a complementing, dynamic method to track the therapeutic value of cancer treatments and continuously update value assessments as additional evidence accumulates. Concerns about increased healthcare costs and the ability to pay for meaningful health improvements have fueled discussions about value in healthcare. Various organizations have responded by developing value frameworks to assess the relative benefits of therapeutic interventions. These frameworks typically assess new treatments at the time of launch based on data from initial registration trials and other available information. Some of these frameworks rely mainly on hazard ratios (HRs) of primary end points from registration trials, because these measures often provide the only available estimate of efficacy. However, the extent to which HRs from initial trials predict future value of new therapies is unclear. Moreover, while current value frameworks provide a good initial basis for conceptualizing value, they are not designed to consider value across the lifecycle of a treatment, and they inadequately account for patient preferences.Several examples of published case studies attempt to quantify value dynamically. Among these is the application of Garrison and Veenstra's dynamic lifecycle model to trastuzumab to calculate annual incremental cost-effectiveness ratios (ICERs) for two indications: metastatic breast cancer and adjuvant treatment for early breast cancer [1]. That model demonstrated a decrease over time in ICERs for the combined indications. Similarly, Lu et al. conducted an analysis of the dynamic cost-effectiveness of docetaxel and paclitaxel as new indications arose and patents expired [2]. They concluded, "One direction for future research might be to create a predictive tool that estimates the predicted long-term dynamic ICER for a specific drug at product launch. … All these sources of information can be used together to predict the probability of different events over a drug's life cycle." KEYWORDShazard ratio • health economics • value assessment

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“…EMA's Senior Medical Officer Eichler has preferred the term 'adaptive pathways', indicating a less legalistic and softer, provisional (conceptually hybrid, see Table 1) approach to flexible gatekeeping that arguably lessens the financial risk for producers (Eichler, 2015). EMA's intended adaptive pathways initiative amounts to an umbrella policy approach that would integrate a number of elements, especially related to non-conventional data requirements, such as adaptive clinical trial design, patient centric benefit/risk assessments and the continuous evaluation of a therapy as new evidence 'including real world evidence' (meaning from patient registries or patient cohort studies without control groups) becomes available.…”

Section: Gatekeeping the Marketplacementioning

confidence: 99%

Special Treatment? Flexibilities in the Politics of Regenerative Medicine’s Gatekeeping Regimes in the UK

Faulkner

2017

Science as Culture

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Emerging flexibilities are apparent in gatekeeping regimes applicable to regenerative medicine products, raising issues about the extent to which and forms in which such flexibilities might promote emerging products as a sector warranting special treatment, in the context of recent policy developments in the UK and wider European Union. Concepts of 'gatekeeping', 'gatekeeping regimes' and 'gateways' can point to the ways in which regulatory institutions, health technology assessment organisations, and national planners and purchasers of health services together define and control entry to the medical product marketplace and the adoption of products into the public healthcare system. Flexibilities in existing regimes and new gateways are a way of maintaining 'connection' between gatekeeping regimes and technoscientific innovation in order to steer innovation pathways. The gateways concept has affinity with that of Callon's 'obligatory passage points'. A wide set of recent policy documents show that the measures promoted exhibit a range of alternative gateways that are being constructed around central, legal, restrictive gatekeeping regimes. However, it would be easy to overestimate the significance of these developments as relaxations that would favour innovative producers and their products on a large scale with wide public health impacts. The concepts of gatekeeping regimes and gateways enable understanding of hybrid developments of exceptions and exemptions to dominant regimes which bridge across the arenas of market regulation, health technology assessment and healthcare system planning. These arenas are being drawn closer together as a means of politically managing stakeholders' aims in the UK, EU and other innovating biomedical health systems globally.

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From adaptive licensing to adaptive pathways: Delivering a flexible life‐span approach to bring new drugs to patients

Cited by 169 publications

References 35 publications

Competition in pharmaceuticals: more product- than price-oriented?

Competition in pharmaceuticals: more product- than price-oriented?

Dynamic value assessments in oncology supported by the PACE Continuous Innovation Indicators

Special Treatment? Flexibilities in the Politics of Regenerative Medicine’s Gatekeeping Regimes in the UK

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