Day 1: 2:45pm- 4:00pm Panel Slides (Nov 18) Access to Innovation Conference 2019
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The document discusses opportunities and challenges for personalized medicines and sickle cell disease treatments. It provides an overview of current limited treatment options for sickle cell disease and highlights several new treatment options currently under development or approval, including Novartis' crizanlizumab, Global Blood Therapeutics' voxelotor, and gene therapies. It notes obstacles to access like high drug costs, long review processes, Canada's drug pricing system, and the small market size. The presentation calls on stakeholders to work together to improve access and education.
![© Institute for Clinical and Economic Review, 2018
HTA and High Impact (Potentially Curative) Treatments
üCAR-T for B-cell Lymphoma
üZolgensma for spinal muscular atrophy
üEmicizumab for hemophilia A with inhibitors
ü[PCSK9 for high cholesterol]
Fair Price = Fair Access = Future Innovation](https://image.slidesharecdn.com/day1panel3-191122194422/85/Day-1-2-45pm-4-00pm-Panel-Slides-Nov-18-Access-to-Innovation-Conference-2019-40-320.jpg)
Day 1: 2:45pm- 4:00pm Panel Slides (Nov 18) Access to Innovation Conference 2019
- 1. Personalized Medicines: Access Opportunities & Challenges What is coming up for Sickle Cell Disease? Biba Tinga, President/ED Sickle Cell Disease Association of Canada/Association d’anémie falciforme SCDAC
- 2. Who are we?
- 3. Limited Treatment Options: for over 40 years! • Antibiotics • Pain killers and anti-inflammatory • Vaccines • L-glutamine (Endari) • Hydroxyurea • Blood Transfusion (High risks: Iron overload & reactions) • Bone marrow transplant ( only cure available but not for everyone – 10%) It is only supportive care.
- 4. Hope is materializing! New drugs are under development: 1 – Novartis: SEG 101 - Crizanlizumab is a monthly infusion under development to prevent VOCs, pain crises in patients with sickle cell disease. It is considered as a breakthrough therapy because, it has the potential to prevent SCD pain crises. On Nov. 15,2019 -- FDA approves Adakveo!!!
- 5. A Patient wants a cure! 2. Global Blood Therapeutics, GBT has developed a drug called voxelotor (GBT440). It is an oral, once-daily therapy for sickle cell disease. It increases the affinity of hemoglobin for oxygen. USA: as early as Feb. 2020 3. L-glutamine : under review by Health Canada
- 6. Gene Therapy: a universal cure? 3- Gene Therapy: availability in 2- 4 years? Most patients are hoping for a cure thanks to gene therapy. CRISPR Therapeutics - Clustered Regularly Interspaced Short palindromic repeats - a replacement of a specific region of DNA First patient in the US – 41 years old - Summer 2019
- 7. Gene Therapy as a solution On Sept. 2019, Alabama – a 29 y. old man is free of SCD after receiving gene therapy for 2 years – Bluebird Bio
- 8. What are the obstacles? • Gene Therapy: 5 years monitoring before it is classified as a cure and high cost • Long review process: it can take up to 2 years for Heath Canada to review a new drug safety. • Canada Drug pricing: cutting the cost could impact research, development, and future access (refuse to enter the Canadian Market early on) • Small number of patients do not make an attractive market!
- 9. What can we do? As patient group, SCDAC advocates for the best care for the Sickle Cell Community. But we can do it alone! Mobilize, Organize, Unify and Educate! • Educate patient population to participate in clinical trials • Push for priority review process • Engage key Partners: other rare disease groups ( CORD), Industry Partners • Make sure the government does not forget about us!
- 10. We will be ready! • SCDAC/AAFC is currently working on a National Registry for SCD Collect data on patients diagnosed with SCD in order to facilitate a wide variety of research • Determine the disease prevalence • Assess quality of life • Improve standards of care • Evaluate novel and existing therapies in real world settings.
- 11. Core Ecosystem Element Registry Researchers Industry Clinicians Health Care System Investigator Network Patient Organizations Government Patients Families Caregivers Courtesy of Dataffinity
- 12. A sickle Cell patient getting blood exchange
- 13. Thank you Merci!
- 14. Canadian Organization for Rare Disorders Access to Innovation Conference Toronto, November 18-19, 2019 Personalized Healthcare: Interactive Tools in Hemophilia David Page National Director of Health Policy Canadian Hemophilia Society
- 15. Short bio • Member, Canadian Bleeding Disorder Registry Stakeholder Advisory Group (CBDR & MyCBDR) • User, Web Accessible Population Pharmacokinetics Service (WAPPS & MyWAPPS) • Co-investigator, Patient Reported Outcomes, Burdens and Experiences Study (PROBE & MyPROBE)
- 16. An Introduction to Hemophilia • Genetic condition • Deficiency in clotting factors VIII and IX • Frequent bleeding into joints and muscles • Without treatment, severe joint damage and short life expectancy
- 17. Severity and treatment Severity / Factor level Symptoms Treatment Severe < 1% of normal clotting activity Frequent hemorrhages into joints and muscles with no trauma 2 to 3 IV infusions per week of clotting factor to prevent bleeding (prophylaxis) Moderate 1 to 5 % of normal clotting activity Less frequent hemorrhages into joints and muscles with minor trauma IV infusions of clotting factor to stop bleeding (on-demand treatment) Mild 5 to 40% of normal clotting activity Infrequent hemorrhages into joints and muscles after trauma IV infusions of clotting factor to stop bleeding (on-demand treatment)
- 18. Prophylaxis and home care • Young children learn to self- infuse at home • > 90% of clotting factor concentrates are infused outside hospital setting • Ideally, patients go to the hemophilia clinic only 1 or 2 times per year How can care be best managed?
- 22. Pharmacokinetics (PK) • Goal: > 1% of clotting activity to prevent bleeding • But every person has different pharmacokinetics (recovery, half-life) So how to determine when you are falling to the critical 1% level?
- 26. Desired medical outcomes • Long-term survival • Few or no bleeds • Maintenance of joint function But if you ask Kevin …
- 27. Patient relevant outcomes He wants to … • be free of pain • be able to play with his friends • be able to take part in school activities • avoid hospital and joint surgery • get a good education • get a good job • have a family
- 35. 3 connected apps • The MyCBDR app allows patients to report their bleeds and their infusions to their treatment centre in real time. Treatment can be personalized. • The MyWAPPS app allows patients to see their clotting factor levels in real time. Activities can be adjusted accordingly. • The MyPROBE app allows patient organizations to collect and analyze patient relevant outcomes. These can be used to advocate for improved care.
- 36. An introduction to ICER and the evolution of the US landscape for drug pricing and access (for high impact therapies) Celia Segel Director of Comparative Effectiveness Research Policy Development
- 37. © Institute for Clinical and Economic Review, 2018 Institute for Clinical and Economic Review (ICER) • Independent health technology assessment group whose reviews are funded by non-profit foundations • Develop publicly available value assessment reports on medical tests, treatments, and delivery system innovations • Use cost-effectiveness analysis to determine value-based price benchmarks • Convene regional independent appraisal committees for public hearings on each report
- 38. © Institute for Clinical and Economic Review, 2018 Sources of Funding, 2019 2% 77% 8% 13% Government grants and contracts Non-profit foundations Contributions from health plans and provider groups Manufacturer grants and contributions ICER Policy Summit and non-report activities
- 39. © Institute for Clinical and Economic Review, 2019 Valuing Cures: Key Conceptual Issues Defining Cures: Broadening from cures to all “single and short- term transformative therapies” (SSTs) 1. Uncertainty with unrecoverable costs 2. Additional domains of value 3. Time Divergence Between Costs and Benefits 4. Affordability and “Shared savings” in calculations of alternative value-based price benchmarks
- 40. © Institute for Clinical and Economic Review, 2018 HTA and High Impact (Potentially Curative) Treatments üCAR-T for B-cell Lymphoma üZolgensma for spinal muscular atrophy üEmicizumab for hemophilia A with inhibitors ü[PCSK9 for high cholesterol] Fair Price = Fair Access = Future Innovation
- 41. © Institute for Clinical and Economic Review, 2018 Patients, Caregivers and Advocates Input • Understanding the patient population and subpopulations • How to think about heterogeneity of patients with regards to treatment response/non- response? • Is there a sub-population that has particularly high severity? • Understanding the standard of care: effectiveness and safety • Understanding Outcomes: • What are outcomes that matter to patients that are not captured in the trial data? • How do patients find the outcomes captured in the trials meaningful to their quality of life? • What are the limitations in the quality of life data used in our analysis and how should we test it? • What are important other benefits and contextual considerations for understanding the treatment in our evaluation? • Process with patients: Interviews, public comment periods (draft scope and draft report), preliminary model presentation, external review, patient expertise and testimony during a public meeting of appraisal committees
- 42. More Resources: - ICER Final Methods for Cures - ICER’s Valuing Cure Technical Brief - ICER’s Patient Participation Guide
- 43. /////////// HTA Case Study: Larotrectinib for TRK Fusion Cancer CORD Conference Nov 18, 2019 Brandon Levac, Senior Manager – Patient Access & Stakeholder Relations, Oncology, Bayer Inc. MAC-VIT-CA-0001-1
- 44. I am an employee of Bayer Inc. Bayer has provided an unrestricted sponsorship to support this conference. For complete information on Bayer products, please refer to the respective product monograph: https://www.bayer.ca/omr/online/vitrakvi-pm-en.pdf Disclaimers
- 45. VITRAKVI® (larotrectinib) is the first tumour agnostic treatment approved by Health Canada 3 • Approved under NOC/c pathway on July 10, 2019 Indication VITRAKVI is indicated for the treatment of adult and pediatric patients with solid tumours that: • have a Neurotrophic Tyrosine Receptor Kinase (NTRK) gene fusion without a known acquired resistance mutation, • are metastatic or where surgical resection is likely to result in severe morbidity, and • have no satisfactory treatment options. Treatment with VITRAKVI should be initiated following confirmation of an NTRK gene fusion in a tumour specimen using a validated test. For complete prescribing information, please see the VITRAKVI Product Monograph: https://www.bayer.ca/omr/online/vitrakvi-pm-en.pdf
- 46. On Aug 29, 2019, a positive but limited initial pERC recommendation for VITRAKVI was issued Limited to Four Rare Tumour Types Source: Larotrectinib pERC Initial Recommendation, accessed on Aug 29, 2019 at: https://www.cadth.ca/larotrectinib-neurotrophic-tyrosine-receptor-kinase-ntrk-locally- advanced-or-metastatic-solid
- 47. Four rare tumour types recommended for coverage in initial pERC recommendation, all others excluded Adapted from Cocco et al. NTRK fusion-positive cancers and TRK inhibitor therapy. Nat Rev Clin Oncol. 2018 (ePub ahead of print) 5
- 48. All stakeholder input either agreed with the initial recommendation, or supported broader coverage Feedback on initial recommendation was provided by multiple patient groups, clinicians, PAG and Bayer 6 Patient Groups Clinicians PAG Bayer • Colorectal Cancer Canada • Canadian Cancer Survivor Network • Patient group not stated • Neuroblastoma Canada • Cancer Care Ontario • Cancer Care Ontario Skin DAC • Clinical Medical Oncologist (9) • Clinical Group – Lung Cancer Canada • Pediatric Oncology Group of Ontario • 2x Agree in part • 2x Disagree • 1x Agree • 3x Agree in part • Agree • Disagree Source: Larotrectinib for Neurotrophic Tyrosine Receptor Kinase (NTRK) Locally Advanced or Metastatic Solid Tumours, CADTH website, https://www.cadth.ca/larotrectinib- neurotrophic-tyrosine-receptor-kinase-ntrk-locally-advanced-or-metastatic-solid Stakeholder Input
- 49. On Oct 31, 2019, a negative final pERC recommendation for VITRAKVI was issued No coverage recommended across all tumour types Source: Larotrectinib pERC Initial Recommendation, available at: https://www.cadth.ca/sites/default/files/pcodr/Reviews2019/10159LarotrectinibNTRK%2BSolidTumours_fnRec_REDACT_31Oct201_ChairApproved_final.pdf
- 50. Reflections and Recommendations for Improvement 8 • Larotrectinib is approved as a tumour agnostic treatment. pERC agreed it should be assessed as such. • The current review process does not support the assessment of novel, complex, rare disease treatments. There is also a mismatch between evidence expectations and realities. • Recommendations: 1. Increase scientific exchange during reviews between HTA bodies / submitter / expert committees / clinicians / patient groups 2. When the HTA review identifies additional evidence needs, adopt conditional reimbursement recommendations tied to reassessments 3. Partner on outcomes-based agreements and/or real world evidence generation to address uncertainty at the HTA stage 4. Implement outcomes-based agreements at the pCPA/payer stage 5. Start now, decision makers can ask for volunteers to pilot
- 51. BRENT FRASER, VP PHARMACEUTICAL REVIEWS NOVEMBER 18, 2019 Access to Innovation Personalized Medicines: Access Opportunities and Challenges
- 52. Conflict of Interest Declaration • No conflicts of interest to declare. • CADTH receives funding from Canada’s federal, provincial, and territorial governments, with the exception of Quebec. • CADTH is an independent, not-for-profit organization responsible for providing Canada’s health care decision- makers with objective evidence to help make informed decisions about the optimal use of drugs, medical devices, diagnostics, and procedures in our health care system.
- 53. Current Status • Precision medicine (PM): customizing care based on patients’ genetic factors • Multiple examples where CADTH has provided funding recommendations to treat condition with specific genetic markers (e.g., oncology, rare dx) • Do not follow a separate health technology assessment process for PM
- 54. Challenges • Uncertainty with respect to the role of the genetic marker e.g., does its presence change the course of treatment for patients? • Trials may be for a smaller number of patients or may be a subgroup within a larger trial. • Trial designs may be “new” requiring addition support, pre-work on methodology, etc prior to an HTA review.
- 55. So What Needs to be in Place?? • Movement toward managed access agreements. • This requires (for the most part) access to data. • All actors across the system need to be aligned to support a MAA approach to funding recommendations. • Implementation of reassessments – ranging from simple to complex. • Allows for expansion/contraction of the approved patient population based on real value.
- 56. Life cycle HTA Pre-market dialogue Managed entry Post-market surveillance Managed exit Reassessment