Generating Evidence to Drive Patient Access
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The document discusses the challenges of achieving patient access to new treatments in a healthcare environment that demands real-world evidence of value beyond regulatory approval. It outlines the necessity for innovative contracting and real-world studies to ensure that therapies are used effectively, addressing concerns of efficacy, safety, and economic impact. Additionally, it emphasizes the importance of capturing the patient experience and leveraging data to inform decision-making for both regulators and payers.
Generating Evidence to Drive Patient Access
- 1. © 2017 PAREXEL INTERNATIONAL CORP. GENERATING EVIDENCE TO DRIVE PATIENT ACCESS 11 October 2017
- 2. © 2017 PAREXEL INTERNATIONAL CORP. / 2 THE NEW REALITY In the current healthcare environment, regulatory approval gets a sponsor to a new “starting line.” The marketplace is demanding demonstration of real-world value before fully adopting or supporting a new treatment alternative.
- 3. © 2017 PAREXEL INTERNATIONAL CORP. / 3 A PRODUCT MUST CLEAR FOUR HURDLES TO ACHIEVE PATIENT ACCESS Quality1 Safety2 Efficacy3 Value4 Regulators Payers Key considerations of… Focus of… Hurdles to Patient Access Regulatory Approval is Traditionally Seen as the Key Barrier
- 4. © 2017 PAREXEL INTERNATIONAL CORP. / 4 THE MOLECULAR BASIS OF UNCERTAINTY: A SUBCELLULAR HYPOTHESIS - SEAN TUNIS Decision Makers Low affinity receptors for evidence. > Knowledge Translation #2 Health Technology Assessment Knowledge Translation #3 Identify Gaps in Evidence Decision-making Defective Transport Knowledge Translation #1 Slow Diffusion Clinical Research Enterprise Published Evidence Intellectual Curiosity Scientific Evidence•
- 5. © 2017 PAREXEL INTERNATIONAL CORP. / 5 5 Clinical Benefit - Magnitude More accepting of shorter-term surrogate endpoints (eg, PFS, ORR, HbA1C, LDL) More demanding of benefits in patient relevant “final” endpoints (eg, morbidity, mortality, QoL) Endpoints Study Design More accepting of benefits from a highly controlled clinical study setting Want to know that study benefits will be reflected in the real- world population to be treated Regulators Payers £ $ Payers Want to Ensure that Endpoints from RCTS Translate Into Real Patient Benefits in Clinical Practice HEALTH, WELL-BEING HEALTH ECONOMICS
- 6. © 2017 PAREXEL INTERNATIONAL CORP. / 6 UNCERTAINTY AROUND DRUG EFFECTIVENESS IS DRIVING THE USE OF INNOVATIVE CONTRACTING AGREEMENTS • How can uncertainty at the time of launch about a new drug's real value be overcome? • What can be done to ensure that a medicine is used by the right patients in the real world and improved patients’ QoL? • How can spending on the drug be contained for individuals and the patient population as a whole? Payers are likely to delay or refuse reimbursement if one or more of these questions remain unanswered, especially if a drug is expected to have a substantial budget impact. THE TAKEAWAY
- 7. © 2017 PAREXEL INTERNATIONAL CORP. / 7 REAL-WORLD CHALLENGES 7
- 8. © 2017 PAREXEL INTERNATIONAL CORP. / 8 8 REAL-WORLD EXAMPLES: MARKET-ACCESS ISSUES Drug Disease Issue Consequences & mitigation strategies Lipoprotein Lipase Deficiency (ultra orphan indication) nmDMD (ultra-orphan indication) 2L+ NSCLC Hypercholesterolemia Chronic Psoriasis and PA Pancreatic cancer 1st EC approved gene therapy Small patient population, no comparator, surrogate endpoint Conditional EMA approval on Phase IIb data – only subgroup data positive (6 min walk test) Trial terminated early Lack LT follow-up Uncertainty over DOT Regulatory approval on clinical trials using surrogate endpoint (LDL-c) Seen as innovative by NICE but not as value for money. Not seen as cost-effective by IQWiG Significant increase in OS but only 1.8 months and no QOL data collected Delayed launch (EMA approved in 2012) to collect 6-yr follow up data >€1 million per patient price tag. DE launched in Q4 2014, UK launch in July 2016. In May ‘16 only 1 reported sale in Europe Rejected by SMC but has been approved by NICE under a Managed Access Agreement, whereby the drug is made temporarily available at a discount until more mature data is available Despite transformational LT survival benefit claims, have struggled to gain positive appraisals in some obligate cost/QALY markets (incl NICE & PBAC) in NSCLC. Economic stopping rule used in NICE ACD. Data sufficient for regulatory approval but not for reimbursement (not recommended by SMC and TLV, no added benefit-IQWiG, ASMR IV -HAS). Ongoing trials to measure CV outcomes Strong efficacy and safety data but limited cost-effectiveness data. Approved in August 2016 by NICE based on a confidential discount and use in only severe patients. HAS – deemed ASMR IV versus generic comparator, led to exclusion from the liste en sus and being only reimbursed under a DRG – i.e. low prices and limited access
- 9. © 2017 PAREXEL INTERNATIONAL CORP. / 9 NEW CURATIVE AND HIGHLY EFFICACIOUS END-OF- LIFE THERAPIES CREATE UNIQUE CHALLENGES e.g. @Hepatitis C • “The sky-high cost of Glybera ($1.4 million)..shows single curative therapies to fix faulty genes may upend the conventional pharmaceutical model” • What was the benefit to the patient and the health system as a whole?
- 10. © 2017 PAREXEL INTERNATIONAL CORP. / 10 HARD ENDPOINTS ARE MORE SIGNIFICANT IN THE REAL WORLD THAN SURROGATE ENDPOINTS • Landmark Outcomes Study Shows That Repatha® (Evolocumab) Decreases LDL-C To Unprecedented Low Levels And Reduces Risk Of Cardiovascular Events With No New Safety Issues • Repatha Significantly Reduces Risk of Hard Major Adverse Cardiovascular Events by 20 Percent • Risks of Heart Attack, Stroke and Coronary Revascularization were nominally reduced by 27 Percent, 21 Percent and 22 Percent, respectively • Patients in Study had History of Heart Attack, Stroke or Symptomatic Peripheral Arterial Disease and Were Treated With Optimized Statin Therapy • Amgen agreed to Offer Innovative Refund Contracts, including a refund of the cost of Repatha for all of their eligible patients who have a heart attack or stroke • Detailed Results Simultaneously Published in the New England Journal of Medicine and Presented at the American College of Cardiology 66th Annual Scientific Session
- 11. © 2017 PAREXEL INTERNATIONAL CORP. / 11 CO-CREATION IS A WAY OF ENSURING EARLY BUY-IN FROM THE MARKET Novartis's deal with French healthcare system Xolair (omalizumab) Government agreed to make Xolair available at requested price, if evidence of the drug’s real-world effectiveness was collected, according to a protocol designed in conjunction with the payer The study aimed to determine whether Xolair decreased the risk of hospitalization due to exacerbations among severe asthma patients. The parties agreed that Xolair’s price would be maintained if the evidence was positive, and would fall if not. The results from the trial, which involved over 750 patients and was funded entirely by Novartis, showed that the drug did decrease hospitalization risk. The relative rate of severe asthmatic episodes fell by 40–65% among those taking Xolair (Grimaldi-Bensouda et al. 2013) PROS Risk-shares can allow pharma to maintain a premium price while generating the kind of peer- reviewed real-world evidence (RWE) that will increasingly be required for uptake in all major markets.
- 12. © 2017 PAREXEL INTERNATIONAL CORP. / 12 PUTTING YOUR MONEY WHERE YOUR MOUTH IS The industry must convince payers that we are willing to back our product, with innovative contracts supported by strong RWE
- 13. © 2017 PAREXEL INTERNATIONAL CORP. / 13 WHY IS THE PATIENT EXPERIENCE SO IMPORTANT IN SUCCESSFUL PRODUCT REIMBURSEMENT Rare Diseases / complex LT conditions e.g. haemophilia / conditions with high unmet needs e.g. chronic migraine, atopic dermatitis Highly active patient associations & growing online forums discussing current and future treatment options, clinical trials and patient experiences Capturing and communicating the patient experience is key to reimbursement because Payers budgets are influenced by the political environment Conditions such as Cancer, Diabetes and CVD are likely to always be prioritised over other conditions Other therapy areas have to fight for their share of a limited funding pot
- 14. © 2017 PAREXEL INTERNATIONAL CORP. / 14 PATIENT ADVOCACY SUCCESS STORIES A regional politician in Wales stated that chronic migraine for their constituents was not being properly managed due to a lack of funding – Payers from LHBs were put under pressure to find the appropriate funding for the condition or receive bad press; as a result the condition soon took priority over other conditions, all fighting for a share of the budget (at least whilst it was in the newspapers) Similarly, patient share-of-voice can be instrumental in lobbying Government for additional funding for rare or chronic conditions. In the UK, Astellas successfully lobbied Parliament for a greater allocation of funding for patients with Incontinence and the company was invited to speak at Parliament, alongside patient group representatives. This also moved the therapy area further up the priority list in a number of CCGs across England In Ophthalmology, in Germany and the UK, patients with RVO in rural areas were going untreated or left with long waiting times before they could be seen by a Consultant. In some cases, patients were losing their sight, due to these long delays. In Germany, Consultants were incentivised to increase the throughput of patients by being paid a supplement for treating patients with the condition within a certain timeframe. In the UK, a separate pot of money was agreed with the NHS purely for the treatment of these patients In Haemophilia A and B, patient groups are highly active and often change their treatment options, depending on the latest available therapies. Patients with inhibitors are especially prone to treatment failure and are always looking for new and better options for managing their condition. Capturing the patient experience is key to convincing Patients, Clinicians and Payers to endorse new therapeutic options in these complex disease area
- 15. © 2017 PAREXEL INTERNATIONAL CORP. / 15 BERLIN, GERMANY CAPTURING THE PATIENT EXPERIENCE REAL-WORLD EVIDENCE STRATEGY:
- 16. © 2017 PAREXEL INTERNATIONAL CORP. / 16 WHY DO WE NEED REAL-WORLD STUDIES? Of 20,388 US Medicare patients ≥ 65 years of age, only 1 in 5 discharged from an acute care hospital with a diagnosis of congestive heart failure (CHF) met the criteria for enrollment in 3 landmark trials that guide treatment of all CHF patients. Masoudi FA, Havranek EP, Wolfe P, et al. Am Heart J. Aug 2003;146(2):250-257. Most RCTs use narrow inclusion and exclusion criteria to select the participants most likely to benefit from an intervention and least likely to experience harm. ?
- 17. © 2017 PAREXEL INTERNATIONAL CORP. / 17 Randomized Clinical Trials (RCTs) don’t reflect actual medical practice. • Tight inclusion/exclusion criteria • Mandated procedures and treatment • Randomization, blinding, placebo, etc. • Short in duration • Homogeneous sites and patients We need to know how a product is used and how it “performs” under real-world conditions. • Safety • Clinical outcomes and effectiveness • Economic value • Humanistic value BECAUSE… Homogeneity Heterogeneity
- 18. © 2017 PAREXEL INTERNATIONAL CORP. / 18 EXACTLY WHAT ARE REAL-WORLD STUDIES? Clinical research that observes clinical practice, rather than defines it. Patients are “real,” with varying co-morbidities, compliance, and preferences. The Physician prescribes an approved drug or treatment for their patient, with no protocol mandate The Physician treats a patient per usual care – the study doesn’t alter their care in any way. They generate details about patient outcomes, patient-selection criteria, product usage, product safety, effectiveness and overall value in the “real world.” They are scientifically driven and clinically relevant.
- 19. © 2017 PAREXEL INTERNATIONAL CORP. / 19 REAL-WORLD RESEARCH MODELS PRAGMATIC ‘SPECTRUM’ Primary Interventional Studies Retrospective Chart Reviews Secondary Database Analyses EMR Data Analysis Primary NIS / Observational Studies / Registries Hybrid Studies Interventional Observational Minimally Interventional Studies
- 20. © 2017 PAREXEL INTERNATIONAL CORP. / 20 UNIQUE VALUE OF REAL-WORLD DATA • Outcomes • Incidence and prevalence • Standard of care and treatment patterns • Diagnosis distribution • Distribution of care • Quality of life • Demographics (age, gender, geography) • Risk-sharing and value agreements • Cost-effectiveness • Budget impact • Health care costs • Burden of illness • Socioeconomics (income, education, sick-leave) • Productivity loss • Patient-selection criteria • Prescription patterns • Safety & effectiveness • Persistance • Compliance • Dosage • Referrals • Time to treatment • Supplement trial data UNDERSTAND DISEASE CLARIFY ECONOMICS CHARACTERIZE TREATMENTS
- 21. © 2017 PAREXEL INTERNATIONAL CORP. / 21 In actual use… • Will the product be safe and effective? • Will the product be cost-effective? • Will quality-of-life be enhanced? • Are there important hypotheses worthy of further evaluation? • Are there subsets of patients in which the product seems to work best? • Where are the patients, and who are the treating physicians? • For whom – and how - will physicians prescribe the product? • How will the market be affected by the entry of the product? • How will competitors be affected? WHAT QUESTIONS CAN YOU ANSWER?
- 22. © 2017 PAREXEL INTERNATIONAL CORP. / 22 = Good outcome = Intermed outcome = Bad outcome MEAN TREATMENT DIFFERENCE MEAN TREATMENT DIFFERENCE RISK STRATIFICATION WHY ARE SUBGROUPS SO IMPORTANT?
- 23. © 2017 PAREXEL INTERNATIONAL CORP. / 23 LINKING DATA ASSETS OFFERS THE MOST COMPREHENSIVE VIEW OF THE PATIENT EXPERIENCE The Patient Experience EHR Clinical Data Healthcare Utilization & Cost Data from Claims Other Secondary Data Physician- Reported and Patient- Reported Measures
- 24. © 2017 PAREXEL INTERNATIONAL CORP. / 24 DIRECT-TO-PATIENT OPPORTUNITIES ARE EMERGING PHARMA MUST DECIDE HOW BEST TO LEVERAGE THEM DTP Innovations Impacting RWE Generation The delivery of direct-to-patient studies includes ‘siteless constructs’ which leverage e-consent & screening technologies, social media, central labs & home-health professionals, wearables/devices for collecting patient reported outcomes in real-life, as well as EMR and secondary data analysis
- 25. © 2017 PAREXEL INTERNATIONAL CORP. / 25 SIMPLIFYING THE JOURNEY - KEY BENEFITS FOR MOBILE HEALTH/SENSORS The Sponsor • Increased frequency of data assessment • Novel data sources and outcomes assessment • Improved protocol compliance • Lowered costs through fewer visits • Expansion in participation The Site • Better insights into patient’s profile and health beyond routine visits • Reduced burden and cost • Earlier indications of warning signs • Better compliance • Eliminates recall bias • Improved communications The Patient • Increased engagement, motivation, and compliance • Fewer in-clinic assessments, less travel • Increased frequency of endpoint assessment • Safety: real time user feedback • Ease of use The magnitude (or even existence) of benefits is critically linked to appropriate protocol design, sensor choice and operational model
- 26. © 2017 PAREXEL INTERNATIONAL CORP. / 26© 2017 PAREXEL INTERNATIONAL CORP. / 26 THANK YOU