Summary of Expensive Drugs for Rare Diseases (EDRD) Working Group Webinar
Stakeholder consultation is underway on a supplemental process for complex/specialized drugs that builds upon the existing national HTA and reimbursement process to make responsive funding decisions with a focus on non-oncology therapies. The supplemental process is a significant development in EDRD policy and it looks to streamline the evaluation and reimbursement process of eligible drugs incorporating additional RWE requirements (when applicable) and the use of managed access agreements. Continued reimbursement would be contingent on the evaluation of RWE considering the pre-determined targets (cost-effectiveness, outcomes, etc.). The supplemental process is not intended to circumvent high quality evidence generation.
Suggested eligibility criteria are broader than a specific incidence/prevalence definition of “rare” which has been previously debated in the absence of a federal DRD regulatory policy in Canada. Typically, assessing the value of drugs for treatment of rare diseases is challenging because of uncertainty of evidence, low prevalence, and poorly explored epidemiology with many trials using unvalidated biochemical markers and “biological plausibility” that trajectory of disease will be improved by the drug under consideration. A number of practical implementation questions remain on cost-effectiveness thresholds, whether a national DRD registry is needed, how RWE targets will be determined/ collected/assessed, informed patient consent and other legal considerations, who will be managing the process, and what resource supports will be put in place to support this process. Given the number of large national initiatives such as PMPRB modernization reforms, national pharmacare, and pre-existing jurisdictional DRD reimbursement policies/plans, it will be interesting to see how all these initiatives align.
A key development is the proposed creation of a national panel of experts to assess individual patient cases for public funding eligibility once a drug is listed for funding, with funding decisions remaining at the jurisdictional level. The co-ordination of clinical experts may assist in equity and consistency of drug funding decisions, help address implementation issues at the jurisdictional level, and assist in the creation of centres of excellence.
Suggested timelines for implementation of the supplemental process is Spring/Summer of 2019.
On November 8, 2018 the Expensive Drugs for Rare Diseases (EDRD) working group, co-led by Ontario, Alberta and British Columbia, held their first of two identical webinars to present the proposed supplemental process for highly specialized/complex drugs and to receive stakeholder feedback on this proposal.
The EDRD working group was established in 2014 to explore management of rare disease drug therapies with evidence-based approaches. This newest proposed initiative is meant to build upon existing review processes and serve as a proactive, consistent, fair and transparent process to assess complex/specialized drugs for the purposes of making responsive funding decisions. Accounting for consultations, the high-level target of implementing this process is Spring 2019.
The webinar was led by Chad Mitchell (Executive Director, Pharmaceutical and Health Benefits, Alberta Health) and Heather Logan (Vice President of Pharmaceutical Reviews (acting)), and a summary of the presentation and subsequent discussion with industry can be found below.
The proposed process builds upon strengths of the current system while accounting for the changing landscape and aligns with other significant efforts to improve overall management of pharmaceuticals in Canada (i.e. Health Canada regulatory reforms, CADTH, PMPRB modernization and the pCPA).
The process provides accommodation to existing processes to address challenges for complex/specialized drugs for severe diseases with high unmet need, limited evidence of drug efficacy/effectiveness, poor cost-effectiveness and/or significant budget impact. This includes five key processes:
- Early Screening and Identification of Potentially Eligible Drugs
- Based on the Health Canada acceptance for review through an expedited pathway and other potential criteria.
- Concurrent Submission Process
- Parallel regulatory/HTA review and submissions to PMPRB and pCPA would also be at this time.
- HTA Review Process
- Additional RWE requirements, start/stop criteria and cost-effectiveness targets would be identified.
- pCPA Negotiations & Implementation
- Managed access agreements may be sought taking into consideration RWE requirements and pre-determined cost-effectiveness thresholds. These would be encoded into the PLA.
- Collection & Re-assessment of RWE
- At pre-determined time points RWE would be evaluated and assessed against pre-negotiated targets.
- After the re-assessment, changes to the listing criteria, price or delisting would occur according to the terms outlined in the PLA at the outset.
Additional components include:
- Individual Patient Access: centralized panel of experts to assess individual patient cases for public funding eligibility (e.g. starting criteria).
- Communications: important throughout the supplemental process and will conduct multiple consultations with stakeholders.
The potential benefits of these supplemental processes are:
- More timely funding recommendations & decisions;
- Improved use of RWE to inform evidence-based evaluations;
- Ability to enter conditional managed access schemes with pre-set expectations;
- Improved negotiation/coordination to ensure value/affordability;
- Increased consistency of funding implementation.
It is important to recognize that meeting eligibility criteria does not guarantee public funding nor does it intend to allow drugs to bypass regular processes or circumvent high-quality evidence generation.
The presenters highlighted that industry feedback on this process, particularly the seven questions posed in the Appendix, is crucial. Written feedback is also appreciated however it has been requested that the responses be collated where possible through the respective industry associations. After the second webinar has concluded a survey will also be circulated to industry representatives registered for the webinar to elicit further feedback on these questions including:
- What do you perceive to be the current challenges and barriers facing expensive drugs for rare diseases?
- From your perspective does the proposed supplemental process address some or all of the current challenges encountered with complex/specialized drugs, including drugs for rare diseases? Why or why not?
- What role could you or your organization play in working with others to achieve the stated objective of the proposed supplemental process?
- Please provide your perspective on real world evidence (RWE) and how it could be incorporated into the current processes.
- What challenges and/or opportunities do you see in obtaining and using RWE?
- What is your perspective on having a national review panel to review patient cases? How do you believe this will impact access to EDRDs?
- In considering the proposed process, have we missed anything?
During the winter and spring of 2019, the P/Ts will review the recommendations and make any required changes to the processes with a high-level target of implementing this process in Spring 2019.
The presenters then invited comments and questions from industry representatives on either the process on the seven questions outlined above. Of note:
- INESSS has been consulted;
- CADTH will be facilitating and coordinating the consultation and will consider revisions to its own processes in light of what comes out of the consultation;
- Oncology is not currently included in this process;
- The RWE component will not apply for all products, only where appropriate, and it will be important to establish validated data collection and outcomes in consultation with industry;
- One aim of the supplemental process is to improve the timeliness of reimbursement; however this may not be true for all drugs as it depends on numerous factors;
- Other partners in the system have provided feedback on the process and this type of work requires alignment between regulatory, HTA, and payer bodies;
- Clinical panels would be launched once a drug is successfully listed and patient case assessment will depend on the drug in question as adjudicating criteria may look different;
- It will be important to consider which aspects of the review process need to be conducted sequentially (i.e. product labelling) if regulatory and HTA reviews are being conducted in parallel; and
- The Canadian Fabry Disease Initiative was recommended as an example of success in this space.
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