Regulatory utilization of Flexible Regulatory Pathways to meet unmet medical need

Transcription

1 Median approval time (days) Regulatory utilization of Flexible Regulatory Pathways to meet unmet medical need Session: UNMET (MEDICAL) NEED: SHOULD STAKEHOLDERS ALIGN ON A DEFINITION? Neil McAuslane Director Centre for Innovation in Regulatory Science ISPOR Presentation Glasgow New active substance (NAS) median approval time for six regulatory authorities in EMA FDA PMDA Health Canada Swissmedic TGA CIRS, R&D Briefing Approval year Approval time is calculated from the date of submission to the date of approval by the agency. This time includes agency and company time. EMA approval time includes the EU Commission time. 1

2 Facilitated regulatory Pathways Median approval time (days) NAS median approval time by review type for six regulatory authorities in - Expedited Page 3: Figure 4 CIRS, R&D Briefing EMA** FDA PMDA Health Canada Swissmedic TGA Approval year Expedited review refers to EMA Accelerated Assessment and FDA/PMDA/Health Canada/ Swissmedic Priority Review. TGA has introduced an expedited (priority) review programme in Approval time is calculated from the date of submission to the date of approval by the agency. This time includes agency and company time. EMA approval time includes the EU Commission time. Page 4: Figure 6 Proportion of NASs approved by each agency in that benefitted from at least one Facilitated Regulatory Pathway (FRP) NASs with at least one FRP NASs without FRP CIRS, R&D Briefing EMA FDA PMDA Health Canada Swissmedic TGA Accelerated (referred to in this briefing as Conditional Exceptional Prime Adaptive Pathway Priority (referred to in this briefing as Accelerated Breakthrough Designation Fast Track Priority (referred to in this briefing as Sakigake Priority (referred to in this briefing as Conditional (NOC/c) Priority (referred to in this briefing as Prior notification (VmVA) TGA has introduced an expedited (priority) review programme in

3 Facilitated Pathways -FDA 5 Facilitated Pathways -EMA 6 Prime UMN concept borrowed from CMA and applied to foster accelerated assessment Support scheme with early and enhanced scientific dialogue 3

4 US and Europe - Criteria USA Fast Track Filling an unmet medical need is defined as providing a therapy where none exists or providing a therapy which may be potentially better than available therapy. If there are available therapies - must show some advantage over available therapy, such as: Showing superior effectiveness, effect on serious outcomes or improved effect on serious outcomes Improving the diagnosis of a serious condition where early diagnosis results in an improved outcome Decreasing a clinical significant toxicity of an available therapy that is common and causes discontinuation of treatment Ability to address emerging or anticipated public health need Europe- Conditional Medicine addresses an unmet medical need and targets: a seriously debilitating life-threatening disease, a rare disease use in emergency situations in response to a public health threat In addition, the benefit to public health must outweigh the risk 7 IMPACT OF FACILITATED REGULATORY PATHWAYS ON ACCESS - IMPROVING PREDICTABILITY Facilitated regulatory pathways (FRPs) refer to regulatory pathways designed to accelerate submission, review and approval of medicines where there is an unmet medical need by providing alternatives to standard regulatory review routes. In this ongoing metric study, CIRS sought to understand the impact of those regulatory models on market access. The survey results show that facilitated regulatory pathways have shortened time to HTA recommendation/coverage. Facilitated regulatory pathway EMA Accelerated Assessment Conditional Authorisation Exceptional Circumstances Englandroll-out timelines: median time (days) for regulatory and HTA review Facilitated regulatory pathway Health Canada Priority review Conditional (Notice of Compliance with conditions; NOC/c) Canadaroll-out timelines: median time (days) for regulatory and HTA review Facilitated regulatory pathway FDA Priority Review Accelerated Approval Breakthrough Therapy Fast Track United States roll-out timelines: median time (days) for regulatory review and coverage EMA review days NICE review 265 days Health Canada time 356 days CADTH review 209 days FDA review 303 days United coverage 106 days FRP EMA review days NICE review 221 days FRP Health Canada time 250days CADTH review 240 days FRP FDA review days United coverage 3 dats

5 Industry view divergences observed 9 For what type of products do you see there is biggest divergences between regulatory and HTA in terms of evidentiary requirements? Accelerated regulatory approval Conditional regulatory approval Oncology drug Orphan drug High cost drug (high budget impact) Small incremental innovation Crowded class Source: Tina Wang, CIRS Survey September 2017 Disconnect between FRP and HTA recommendations 10 ID Statement Agree Disagree Use of the FRP pathway should result in a higher proportion of positive HTA recommendations because of high unmet medical need N=44 Source Poll at CIRS Workshop September 2017 Flexible Regulatory/Access Pathways Are we there yet 5

6 Health technology assessment outcomes for conditional marketing authorization versus standard marketing authorization for all jurisdictions included in the study 11 The outcomes of HTA decision making for the 25 oncology products approved in the period 2007-, i.e. 8 CMA compared to 17 SMA, by the 6 European HTA bodies are summarized. Little to no differences between recommendations of HTA bodies by pathway. None of these differences were statistically significant. HTA bodies that came to more positive decisions on products approved through a SMA pathway, did the same on CMA products. This was also the case for negative decision making or proportions of products where HTA review was still pending. CMA = Conditional Marketing Authorisation; SMA= MA Iga Lipska, Jarno Hoekman, Neil McAuslane, Hubert G.M. Leufkens, Anke Hövels - Published Clin Pharmacol Ther. Nov;98(5): Summary - Points 12 Regulatory Agencies have a number of pathways to ensure flexibility in process and time for expediting medicines development and approval There is an increasing use of these pathways to ensure medicines of unmet medical need are made available There is however it seems a disconnect between regulatory approval status and HTA decisions More cooperation between regulatory and HTA bodies is particularly needed to ensure: alignment on which medicines can be considered to fulfill high unmet medical need the clinical evidence requirements for CMA both pre and in post-marketing obligations which will be sufficient for both regulatory approval and HTA recommendation. To create common understanding of unmet medical need, early dialogue and scientific advice is needed with participation from all stakeholders including regulators, HTA bodies, patients, payers and industry. 6

7 Regulatory utilization of Flexible Regulatory Pathways to meet unmet medical need Thank You Neil McAuslane Contact details ISPOR Presentation Glasgow