Pipeline review of innovative therapies - PowerPoint Presentation

1. Pipeline review of innovative therapiesSummary deck

2. 2With over 7,000 medicines in development, the exciting new wave of medical innovation will play a key role in addressing the challenges faced by patients and healthcare systems Source: Health Advances analysis; Adis R&D Insight Database. March 2015, compiled by PhRMA

3. How the pipeline review was done31. Pipeline review and prioritisation of nine areas of upcoming innovation2. Benefit assessment of innovation3. How health systems can prepare to realise benefits of innovationReviewed pharmaceutical pipeline and identified innovationsPrioritised disease areas by level of need for innovation and potential benefit of the innovationSelected innovations that are expected to address unmet needs, shift treatment paradigms and have impact on systemsAssessment of benefit of each area of innovation for: Patients/familyHealthcare systemsSocietyThrough: Qualitative secondary and targeted primary research (stakeholder discussions)Semi-quantitative impact assessment (in the context of the current treatment paradigm across disease areas)Identification of areas where industry can work with other stakeholdersAdapting regulatory approval processesValuing and rewarding innovationDeveloping new financing modelsBuilding integrated care delivery pathways and infrastructureDevelopment of real world data capture systems and updating data policy

4. Over the next five years many more promising medicines are expected to become available CAR-T therapies – genetically modifying T-cells to recognise and destroy tumor cellsCombination therapies – increasing quality of life and life expectancy by combining targeted cancer treatments to increase their effectiveness Gene therapy – helping to replace defective or missing genes in cells through the introduction of DNA for the treatment of genetic diseases Cell therapy – insertion of living cells into patients to replace or repair damaged tissue, in order to facilitate improved organ or tissue functionalityAntibacterial treatments – neutralising highly pathogenic bacterial surface proteins or secreted toxins and activating the immune system to directly kill the bacteria Alzheimer’s treatments – seeking to breakdown or inhibit the formation of protein plaques helping to delay the onset and progression of Alzheimer’s disease NASH treatments – reducing liver inflammation and fibrosis, to lower severity of liver damage and in some cases even reverse disease pathologyMicrobiome therapies – readdressing the gut microbiome balance, to improve the regulation of body systems or reduce the risk of recurrence of severe gut infectionsMigraine treatments – significantly lowering the number of days patients suffer from migraine, to improve quality of life and increase autonomy

5. CAR-T* cell therapies help the body fight back against blood cancer and could replace chemotherapy with treatmentScientific breakthrough Impact on health system Impact onsocieties Impact on patients Scientists use the body’s own immune system to engineer immune cells to target specific types of blood cancer CAR-Ts have the potential to save lives of people affected by blood cancer, increasing life expectancy by nearly 70 years. They may also allow patients to enjoy a better quality of life CAR-Ts may offer a one-time solution, displacing the costs of chemotherapy, and reducing expenditure on targeted therapyThe curative promise of CAR-Ts may allow people to live longer, healthier, more productive lives; treated patients can work, pay taxes and contribute to GDP600,000 people died from blood cancers in Europe in 2013CAR-Ts can reduce expenditure on targeted therapy by 55-100%*CAR - Chimeric Antigen Receptor

6. Research into gene therapies could cure lifelong, life-threatening conditions such as HaemophiliaScientific breakthrough Impact on health system Impact on patients Gene therapy repairs the direct cause of genetic disease, by introducing genetic material into cells to compensate for abnormal genes Gene therapy may offer a one-time cure and improve patient quality of life by reducing disease management burden, spontaneous haemorrhages and the long-term disabilities, specifically arthritis Gene therapy could reduce emergency hospitalisations by decreasing patients suffering serious bleeds. It could also reduce the burden on infusion clinics and cut drug expenditureGene therapy offers a potential life change for patients with haemophilia and their families; thereby reducing absenteeism and increasing economic productivityReduction in cost of current treatment which is approx. €9.6 million over a single patient’s lifetimeImpact onsocieties Around 12,000 people in Europe have Haemophilia B

7. Cell therapy can replace a lifetime of continuous insulin therapy for patients with type 1 diabetesScientific breakthrough Impact on health system Impact on patients Living cells are injected into the patient to treat the causes of their diseasesFor patients with type 1 diabetes, the insertion of insulin producing pancreatic cells will allow for better glycaemic control, prevent the development of co-morbidities, and reduce disease management burdenHospital utilisation and drug expenditure could fall due to a decline in use of insulin therapy, treatments for co-morbidities and serious hypoglycaemic eventsEconomic productivity could increase due to reduced absenteeism and early retirement. Welfare expenditure could also decline if patients no longer require social careApproximately 5 million people in Europe live with type 1 diabetesImpact onsocieties

8. Alzheimer’s treatments help to delay the onset and progression of the disease Scientific breakthrough Impact on health system Impact on patients Whilst existing Alzheimer’s therapies only treat the symptoms of the disease, new treatments have the potential to delay the onset and progression of the disease By delaying the onset or progression of Alzheimer’s, patients are able to live an independent life for longer and enjoy more time with their friends and family New therapies could delay the need for high levels of healthcare resources utilisation associated with the severe disease state, which is estimated to cost healthcare systems € ~20 billion across the EU Families will take less time off work to care for loved ones and it will reduce the amount of money spent on social care servicesMore than 10.5 million patients in Europe live with a form of dementia of which 60% – 80% present as Alzheimer’s Social care costs for Alzheimer’s in Europe is estimated to be € 18 billionImpact onsocieties

9. Combination treatments increase their power, helping people with lung cancer live longer and healthier lives Scientific breakthrough Impact on health system Impact on patients Combination therapies use multiple drugs with different modes of action to boost the chance of the patient’s cancer being kept under controlCombination therapies could extend the lives of patients, increase their quality of life and even offer potential for a cure in patients with poor prognosisImproved and durable long-term response rates would see fewer patients requiring palliative care or overnight hospital stays, reducing waiting times for hospital beds With increased long-term survival, the care burden on families and friends will be reduced and more patients will be able to return to workAround 490,000 people are diagnosed with Non-Small Cell Lung Cancer (NSCLC) each year in the EUIn NSCLC, an estimated 120,000 deaths could be avoided through improved treatment Impact onsocieties

10. Anti-bacterial monoclonal antibodies (mAbs) offer new ways of fighting antibiotic resistance and bacterial respiratory infectionsScientific breakthrough Impact on health system Impact on patients By activating the body’s immune system to directly kill the bacteria, amongst other things, anti-bacterial mAbs offer more effective ways of addressing resistant strains of bacterial infections and antibiotic resistanceAnti-bacterial mAbs can save the lives of patients infected with drug resistant bacteria, and can improve their quality of life by reducing toxicity of high antibiotic doses Anti-bacterial mAbs have the potential to reduce hospital-acquired infections and thus surgeries and length of stays, as well as drug expenditure associated with complicationsThe development of antibacterial mAbs would increase economic productivity, as well as reduce the pressure on developing further antibiotics allowing investment elsewhere300 million people worldwide are expected to die prematurely because of drug resistance over the next 35 yearsImpact onsocieties

11. PPAR/FXR* agonists will be the first available treatments NASH**, and could significantly lower need for liver transplants Scientific breakthrough Impact on health system Impact on patients PPAR and FXR agonists will be the first ever therapies for NASH and are expected to reduce liver fat build up, inflammation and fibrosisThese new products promise to reverse disease progression resulting in a significant improvement in patient prognosis and reducing the need to undergo liver transplantationThe ability to reverse disease progression would see fewer patients requiring costly liver transplants and treatment associated with liver failure complicationsThe significant reduction in debilitating complications and time spent receiving care will reduce absenteeism, improve economic productivityThe number of late stage NASH patients could be reduced by more than 120,000 in Europe per year due to entry of PPAR and FXR agonistsNASH prevalence is ~16% of the general populationImpact onsocieties * PPAR - Peroxisome Proliferator-Activated Receptors FXR - Farnesoid X Receptors ** NASH - Non-Alcoholic Steatohepatitis

12. Microbiome therapies (MBTs) will lower C. difficile recurrence rate, improving patient outcomes and reducing costsScientific breakthrough Impact on health system Impact on patients MBT aims to establish a healthy gut microbiome, restoring gut function and significantly reducing risk of C. difficile infection recurrenceReducing risk of life threatening C. difficile infection recurrence will lower the risk of death, improve long-term quality of life and reduce the need for invasive faecal transplantsMBT will reduce the long-term costs of infection care, through reducing patient readmissions and need to treat infection complicationsUse of MBTs in combination with antibiotics will lower the risk of antimicrobial resistanceThere are over 250,000 C. difficile infections annually with a mortality rate of ~26%MBT will lower C. difficile recurrence by up to 85%, protecting patients from high mortality risk and saving € 0.6 billion per yearImpact onsocieties

13. CGRP* inhibitors will provide a new hope for patients, improving quality of life and productivityScientific breakthrough Impact on health system Impact on patients CGRP inhibitors work to block a key signalling process known to be involved in migraine, to attenuate or prevent episodic or chronic migraineThese new therapies will offer a new hope for patients for whom existing medications do not work, enabling them to regain function and improve their quality of lifeHigher efficacy of CGRP inhibitors can lower the number of primary care appointments, as well as potentially reduce headache medication overuseProductivity will increase, as new therapies help to lower the number of days lost to work and school due to presenteeism and absenteeismOver 500 million days are lost to migraine in Europe every yearNew CGRP inhibitors could lower hospitalisation costs by € 1.4 billion per yearImpact onsocieties * CGRP - Calcitonin Gene-Related Peptide

14. Horizon scanning will be vital for payers and providers to ensure awareness is high and that new innovations are planned forRobust horizon scanning at country level is needed to help healthcare systems plan and prepare for new innovationHorizon scanning should not be an isolated exercise – but inform policymaking, healthcare service management and holistic budget planningGiven the wide range of issues involved, all stakeholders should be included in implications and planning discussionsIndustry is ready to help health authorities with horizon scanning to understand what is coming through the pipelineCASE STUDY: CGRP inhibitors for migraineCGRP inhibitors are anticipated to significantly increase the number of treated patients, through offering a new mechanism of action as well as improved efficacy over existing optionsThese therapies will also be initiated in secondary care setting, whereas existing treatments are generally initiated in primary careTherefore, ahead of their launch, healthcare systems have started to plan how care pathways will be structured (e.g. in the UK, plans are already in place to ensure care is delivered through specialised hubs)

15. Stakeholder collaboration across healthcare will be required to realise benefits of innovation Incentive to invest in high risk innovation for diseases with a high unmet need Provide more effective treatment options for the best patient treatment for their individual needsProductive people, reduced welfare costsProvide new ways to improve access to innovative therapies, as well as a framework for how to set proactive healthcare agendasCosts avoided by reduction in future procedures/long term treatment and better disease managementIncreased quality of life, better health outcome and more treatment optionsCliniciansSocietyPayersPatients & FamiliesPharmaPolicymakersHealthcare providersMore effective use of resourcesINNOVATION BENEFITSAddressing considerations together will allow patients to benefit from innovation now and in the future