Abbott Molecular, 106 AbbVie, 20, 25, 109, 113, 153 Abilify (aripiprazole), 12 accountable care organizations
(ACOs), 110, 178, 188 active ingredient-based groups, 151 Actonel (risedronate), 103
Actos (pioglitazone), 115
acute myeloid leukemia (AML), 99 Aetna, commercial insurers, 164, 174,
190, 223
affordability, 130, 141, 142, 145, 156, 169
of prescription drugs, 145 up-front of medicine, 154
Affordable Care Act (ACA), 21, 149, 167, 175, 176, 179, 189
Afrezza inhaler, 97 AG-221 (enasidenib), 99 AgaMatrix, 204
Agency for Healthcare Research and Quality (AHRQ), 131 Aggarwal, R, 129
agile supply chain, creating, 229 algorithms, 216, 225, 232
Bayesian, 222, 228 AliveCor, 8, 12, 204
alliance. see also strategic alliances acquisition continuum, 58 biotech companies, 50
Celgene, 49, 54, 55, 84, 95, 98, 99, 107, 140
contract, 62 contractual, 59
arrangement, 64
pharma companies, 50 transactions, 59 Alnylam
cash raising, 27
financing strategies, 26–28 funding, 26
RNA interference (RNAi) based therapies, 26
Alphabet, 6, 8, 12, 17, 18, 20–22, 37, 84, 85
healthcare portfolio, 18
ALS (amyotrophic lateral sclerosis), 102, 127, 129, 199
Alzheimer’s disease, 218 Amazon, 21, 215, 231
American Lung Association, 205 American Society of Clinical Oncologists’
(ASCO) Value Framework, 181 American Telemedicine Association
(ATA), 211 accreditation, 211 American Well, 210–212 AmeriDoc, 211
Amgen, 4, 46, 49, 54, 73, 103, 113, 117, 123, 164, 190, 227
amyotrophic lateral sclerosis (ALS), 102, 127, 129
analytics, 219
across biopharma value chain, 227 continuum, 224
descriptive, 224 driven organization, 226
enabled biopharma organization, 241 first organization building, 238
as strategic imperative, 239
273
Managing Biotechnology: From Science to Market in the Digital Age, First Edition. Françoise Simon and Glen Giovannetti.
© 2017 John Wiley & Sons, Inc. Published 2017 by John Wiley & Sons, Inc.
COPYRIGHTED MATERIAL
analytics (continued )
changing behaviors, 239, 240 partner forflexibility, 240, 241 role in establishing product value, 230 to rethink commercial
activities, 229–232 anaplastic lymphoma kinase, 106 Anthem, 10, 20, 207, 212 antibiotic
development, 146 inappropriate use, 131 research, 146 Apache Hadoop, 222
Apple, 6, 8–17, 22, 85, 100, 138, 140, 197, 199, 200, 202, 206, 215, 227, 229
Apple iTunes, 11 Apple ResearchKit, 140
Apple Watch, 10–14, 22, 138, 197 Aprecia Pharmaceuticals, 201 artificial intelligence (AI), 25, 222 Aspinall, Mara, 72, 91
AstraZeneca, 49, 76, 78, 84, 104, 108, 114, 123, 128, 134, 163, 164, 199, 203, 227
scheme, 164
Australian Pharmaceutical Benefits Advisory Committee (PBAC), 180
Avalere/Faster Cures’ Patient Perspective Value Framework, 181 Baris, Aris, 227
benefit/cost ratio, 156
beyond the pill, 24, 48, 70, 73, 90, 101, 122, 135, 136, 143, 185, 191, 232, 241
beta thalassemia, 140 biannual price, 149, 152 big data, 198, 215, 219, 221
Bill and Melinda Gates Foundation, 41 Biogen, 4, 49, 56, 127, 128, 199, 207, 213 biomarkers, 6, 8, 10, 13, 19, 22, 50, 70,
72, 75, 77–81, 84, 85, 90, 93, 98, 101, 102, 125, 165, 197, 227, 235
development, 80, 84
targeted medicines, 79 biometric sensor, 219 biopharma, 145
best practices for, 210 digital strategies, 197
new R & D patient-centric models, 197, 198
optimizing clinical trials, 198, 199 role of social media in trials, 199,
200 divesting, 55–56 marketing strategy, 94
organizational models, 187, 188 payer relationships, 185–187 pricing model, 147
strategic alliances, 50–51 dealing for, 56–65
mission and objectives, 57–58 monitoring results and
learning, 64–65 partner selection, 56–57 structure, 58–62 drivers and constraints, 47 innovation, 49
meeting commercial needs, 49 vs. acquisition, 53–54 structure considerations, 55 to become analytics experts, multiple
forces, 217 trust-breakers, 186 value chain, 226
biopharma-payer engagement, 188–192 chronic, high-cost diseases
and outcomes-based deals, 191 data co-creation with payers leads
to, 192 integrated payers, 191 scaling-up new partnership
models, 189–191 biopharmaceutical
companies (see biopharma) products, 145
biosensors, 6, 8, 9, 14, 101, 102, 112, 122, 128, 135, 197, 204, 207 biosimilars
challenge of, 117 developing, 49
from Lilly and Boehringer Ingelheim, 203
value proposition associated with, 159 biotech company/entrepreneurs, 35
business model, 34 commercialization, 44
funding sources, and representative amounts, 25 (see alsofinancing sources)
fundraising, geographic considerations, 33 mergers and acquisitions, 43–44 patient engagement capabilities, 44 R & D support payments, 43 biotechnology
industry applications, 5–6 megatrends impact, 6
Biotechnology Innovation Organization (BIO), 228
Blin, Emmanuel, 138 blue biotech, 5
Blue Gene supercomputer, 3 Boniva (ibandronate), 97 Bosley, Katrine, 29, 31
BrainBaseline cognitive software, 140 brand
generics, 116–117 loyalty, 100 medicines, 168 models, 94, 103, 104 Brilinta (ticagrelor), 114
Bristol-Myers Squibb, 21, 55, 77, 109, 138, 139, 161
employee engagement initiative, 138–140 goals, 139
program development, 139 globally relevant, 139 internally focused, 139 program launch, 140 storytelling, power of, 139 strategy, 139
budget
constraints, 182 discipline, 169
bundled payment, 167, 168 experiments, selected, 168
models, 165, 167 business
development, 31 models, 50, 69, 218
business-to-business (B to B) model, 176 Bydureon (XR exenatide), 203
Calico, 6, 12, 17, 18, 20, 22 campaigns, 109, 110 CancerCare, 132 cancer treatment, 75
chemotherapy, 75 radiation therapy, 75
capital preservation, adaptations to, 32–33 capital raising
biotech management teams, 32 strategic decisions, 30–33
pricing and dilution, 31–32 capital, sources, 28
corporate venture capital, 28 crossover investment funds, 28 governments and disease foundations
grants, 28
larger pharmaceutical/biotechnology companies, collaborations with, 28
public investors, 28 seed stage investors, 28 venture capitalfirms, 28 cardiometabolic syndrome, 12 CDC (Centers for Disease Control and
Prevention), 18, 198
Celgene, 49, 54, 55, 84, 95, 98, 99, 107, 140
Centers for Medicare and Medicaid Services (CMS), 131, 167, 189 Cerezyme (imiglucerase), 117
Charpentier, Emanuelle, 3 chemotherapy, 71, 74, 106 Chick, Stephen, 129 chief data officer (CDO), 239 chief information officer (CIO), 239 chief patient officer, 138, 143, 204 chronic myeloid leukemia, 4, 70, 95 Cigna, 164, 190, 211
Cleveland Clinic, 12, 16, 17, 105, 133 Clinical Commissioning Groups, 180
Clinical Educator Programs, 137 clinical trial, 6, 10, 13, 14, 19–21, 27, 32,
42, 48, 50, 60, 62, 63, 77–79, 84, 104, 108, 120–122, 125, 127, 133, 144, 155, 160, 186, 190, 197, 198, 200, 205, 206, 208, 216, 218–220, 223, 228, 229, 231, 232, 235, 238, 240 consent forms, 120
enhancement, 127 improving, 228, 229
patient-to-patient communications, 122 trial optimization, 126
cloud computing, 16, 221 cloud-based storage, 216 collaborations, 192 combination products, 115 commercial insurers, 174, 177 commercial risks, 157 commercialization costs, 147
companion diagnostics, 70, 77, 105–107 competition, 146, 148, 150, 153, 158, 165,
167, 175, 176, 186, 212, 229, 242
competitors, 105, 128, 146 consumer
attitudes, 102
centered trends, 206–210 decision journey, 100
social media, 208–210 wearables, 206–208 empowerment, 69
Web-driven, 197 engagement, 110
focused healthcare loans, 170 information gaps, 123 pricing, 94
research, 96
content marketing, 110–111
continuous glucose monitoring (CGM) device, 12
cost control, 73–74 cost-effectiveness, 180
analyses, 182
cost-of-illness modeling, 170 cost-sharing, 217
Coulter, Martin, 129
Crestor (rosuvastatin), 103, 104 Crick, Francis, 3, 4
CRISPR/Cas9 technology, 3, 20
cross-functional teams, 107, 118, 143, 204 cultural barriers, 238
curative therapies, 170 custom-built IBM apps, 16 cutting-edge technologies, 51 CVS Health Corp., 164
cysticfibrosis, 42, 70, 91, 96, 145 drugs, 145
Cystic Fibrosis Foundation, 122, 128, 209 Dana-Farber Cancer Institute, 170, 206 data
analysis, 217 analytics, 226, 232
and analytics challenges, 233 capabilities, 225
driven solutions, 218 generation, 218
integration, 226, 233–235 lifestyle, 220
mining process, 136 privacy, 86, 236, 237 quality, 233, 235, 236 sharing, 81, 88 data breaches, 207 Davidovic, David, 221, 222 debt-financing instruments, 170 decision-making power, 152 democratization, 217
department-specific key performance indicators (KPIs), 138 Dexcom, 6, 12, 19
diabetes, 6, 12, 16, 18, 19, 75, 104, 105, 110, 114, 123, 124, 134, 141, 167, 168, 190, 191, 200, 203, 204, 207, 224, 231
diagnostics developers, 88 drug developers, deal making
between, 89 digital biomarkers, 72 digital communications
metrics for, 141
digital engagement tools, 133 digital ethnography tools, 97
digital health, 146 landscape, 9 opportunities, 6–7 strategies, 197
digital impact on supply chain management, 200, 201 digital markers, in diabetes, 75 digital medicines, 90
digital precision medicine, 74–75 digital services, scope of, 211, 212 digital technologies, 69
digital tools, 146
digital transformation, of commercial activities, 201–204 direct-to-consumer (DTC)
advertising, 107 promotion, 134 disease
management services, 112 outcomes, 75
prevention accelerator, 123 DNAnexus, 220, 221 doctor on demand, 211
virtual services, 133 Doudna, Jennifer, 3, 4 3D printing, 201 drug costs, 168 drug developers, 84, 88
drug development, 47, 51, 73, 90, 146 efforts, 24
product approval, 24 drug discovery, 47, 226 drug facts box, 107 drug formulations, 115 drug interactions, 117 drug manufacturers, 168 drug marketing, 106 drug pricing, 55
challenges, 48 decisions, 145 economics of, 146–153 inflation, 109
drug recalls, 96 drug regulators, 90 drug resistance, 76 drug spending, 94 drug therapy, 69
drug-diagnostic combinations, 71 pairs, 79
drug-resistant microbial infections, 146 drugs targeting, 71
dRx Capital, 6, 200, 213
DTC/direct to consumer spending, 109 dual branding models, 103
dynamic
data market, 233
financing environment, 33 e-consultations, 133 e-patients, 126 economic drivers, 146 economic models, 146
EGFR. see epidermal growth factor receptor (EGFR)
EHRs. see electronic, health records (EHRs) electronic
consent process, 121
health records (EHRs), 6, 74, 122, 220 data, 220
employee incentives, 61 employers, 179
ACA strengthened federal government’s role, 179
drive harder bargain with, 179 variance, 179
vertical integration, 180 Entresto (sacubitril/valsartan), 164 enzyme replacement therapies, 60 epacadostat, 107
eParticipants, 129
epidermal growth factor receptor (EGFR), 76
expression, 165 inhibitor, 76, 79 epigenome, 72
epinephrine injection, 145 EpiWatch app, 14 equity capital, 33
ertugliflozin(insulin glargine), 203 European Medicines Agency (EMA), 121,
147
European Patients Academy on Therapeutic Innovation (EUPATI), 123
European Patients Forum, 123 European payers, 180 European Union, 150
payers characteristics, 181 high-level unity, low-level
fragmentation, 180, 181 portfolio of pricing controls, 150, 151 RareConnect site, 208
variations to manage drug prices/
costs, 150
EURORDIS (European Organization for Rare Diseases), 208
Everyday Health, 130, 211 evidence-based marketing, 104 evidence-based medicine, 222 evidence-based strategy, 96 evolocumab, 73
experimental pricing strategies, 165 indication-specific pricing, 165 external reference pricing, 151 exubera, 97
Fabrazyme (agalsidase alfa), 117 Facebook, 18, 145, 199, 204, 205, 208,
209, 215, 232
Farxiga (dapagliflozin), 115, 203 Federal Trade Commission (FTC), 208 financial-based agreements (FBAs), 163 financing and capital allocation
strategy, 31 financing sources, 33–43
asset-basedfinancing, 43 corporate venture capital, 37 crossover investors, 38 government grants and disease
foundations, 41–42
mergers and acquisitions, role of, 44 private philanthropic sources, 41 public investors
IPO process, 38–41 seed capital, 33–35
angel investors, 33–34 crowdfunding, 34 government grant, 34–35 strategic alliances, 43 venture capital, 35–37 venture debt, 37–38
Fitbit, 6, 10, 11, 14, 138, 197, 199, 202, 206, 207, 213
wristband sensor, 10 Fit2Me website, 134 fixed price, 168
fluorescent reporter protein, 5
Food and Drug Administration (FDA), 4, 10, 12, 46, 73, 78, 81, 84, 91, 121, 124, 128, 134, 147, 155, 197, 200, 207, 208, 209, 237 alliance with the PatientsLikeMe online
community, 121 approval of Keytruda, 78, 106 approved Amgen and Sanofi/
Regeneron’s PCSK9 inhibitors, 43
approved medical devices, 8, 9 approved Neupogen indications, 117 development and validation of
PROs, 124, 127 digital health unit, 75 encouraging biomarker-driven
approaches, 84 Foschini, Luca, 231 Fouse, Jacqualyn, 98 franchise building, 113–114 Franklin, Rosalind, 3, 4 Frazier, Kenneth, 145 future investors, 32
Galvani Bioelectronics, 18, 19 Garfield, Susan, 162
gene
mutations, 101
sequencing optimization, 3 sequencing technologies, 70
Genentech, 4, 5, 17, 46, 60, 61, 69, 101, 115, 128, 199, 205, 222 generic drugs, 94, 168
genetic data, 219, 220, 227 genetic markers, 98 genome sequencing, 79, 226 genomic screening, 102 genomic technologies, 226 genotype-specific oral therapy, 4 Genzyme, 4, 60, 62, 103, 120, 125, 126,
209
Gilead, 54, 94, 105, 109, 153, 161, 163, 164
Gleevec (imatinib), 4, 70, 76 Gleevec(from Novartis), 70 globalfinancial crisis 2008, 30 global organization, 108 global price
band, 159 regulation, 147 GlucoSuccess, 13 GNS Healthcare, 222 golden rice, 5
Google, 8, 11, 12, 17–22, 101, 198, 199, 206, 236
Google Flu Trends (GFT), 18, 236 Google Health, 17, 21
Google mobile apps, 18 Google Play, 11
government-funded systems, 180 Grail, 21, 25, 28, 91
green biotech, 5
group purchasing organizations (GPOs), 162
GSK (Glaxo SmithKline), 18–20, 52, 56, 58, 114, 227
Hack Epilepsy, 51, 124
Harvard Pilgrim’s outcomes-based deal, 169
Harvard Pilgrim Health Care, 190 Harvoni (ledipasvir/sofosbuvir), 94, 105,
109
HCAHPS (Hospital Consumer Assessment of Healthcare Providers and Systems) survey, 131–133, 135 health apps, 8
health data, 189, 207, 215, 216, 218, 219, 221, 236
collected on Apple devices, 13 consumer-generated, 6 easily quantified through, 135 volume, variety, and velocity of, 215 health ecosystem, 221
health exchanges, 176 health expenditure, 174 Healthgrades, 131, 208 health insurance, 174
health plans, 174, 176 online shopping for, 176
health technology assessment (HTA), 83 criteria, 147
healthcare, 218
big infotech strategies, 12–21
Alphabet healthcare portfolio, 17–18 Apple and IBM in Japan, 16 Apple, role of, 12–14 Calico partnerships, 20 Galvani bioelectronics, 19–20 Google investments, 20–21 Google search and mayo
clinic, 18–19 IBM, role of, 15–16 Medtech and pharma alliances, 16–17 Qualcomm, role of, 14–15 Verily partnerships, 19 Watson partnerships, 17 business model variance, 10 costs, 147
data vectors, 218 data, explosion of, 219 digitization of, 90
infotech, disruption risk from, 8–11 infotechnology initiatives, 8 reimbursement, 218 technology strategies, 11–12
new entrants, 12
wearables, rise and limitations of, 11 transforming forces, 7
value chain, impact of digital solutions, 7
HealthKit software, 6 HealthUnlocked, 128
hepatitis C, 12, 94, 105, 108, 109, 129, 134, 145, 153, 156, 158, 159, 163, 164, 167
medicine, 153, 158–159, 163
Herceptin (trastuzumab), 4, 5, 69, 76, 77, 101, 103, 114
Heywood, Ben and Jamie, 127 high drug prices, 147
high-level unity, 180
high-price/high-value product, 156 high-price/low-value product, 156
high-value data, 225 Hill, Colin, 222
HIPAA (Health Insurance Portability and Accountability Act), 86, 200, 207, 208
hospitalizations, 170 hospital rankings, 131
human epidermal growth factor receptor 2 (HER2), 70
Human Genome Project, 3 Humira (adalimumab), 113
hypertension management program, 137 IBM, 3, 6, 8, 9, 12, 13, 15–17, 21, 22, 49,
85, 197, 199, 227, 233 developed apps, 16
IBM Watson, 9, 49, 197, 233 Icahn Institute for Genomics and
Multiscale Biology, 218 ICarbonX, 128
Illumina, 21, 84, 91, 227 Immunotherapeutics, 52 incentives, 88
Incivek (telaprevir), 129 Incyte, 107
indication-specific pricing, 166 information technology (IT), 85 infotech companies
customer/end user, 10 economics, 10 product or service, 10 technology, 10 validation, 10 inhaler, 97
initial public offerings (IPOs), 29 US and European biotechnology, 29 innovation, 145, 147, 152, 159, 186,
197, 218 partnership, 200
Institute for Clinical and Economic Review (ICER), 165, 166
insulin autoinjector, 120 insulin pump, 12
intellectual property, 51, 63 intelligent supply chain, 202
InterMountain Healthcare, 73, 74, 178, 206
Internet of Things (IoT) devices, 219 investment, 29, 34, 146, 201, 209, 225,
234, 237. see also potential investor
bankers, 40 Google, 20 in Aurora, 209 in digital health, 22 joint, 19
minority, 58 return on, 234 rising, 95
traditional model, 35 venture, 14
Invokana (canagliflozin), 115 IPO
investors types, 38
dedicated specialist investment funds, 38
generalist investors, 38 journey, 39
shares offered, 38
Iressa (gefitinib), 76–79, 163, 164 Jakafi (ruxolitinib), 126
Janssen, 52, 88, 118, 123, 128, 187 Janumet (sita-metformin), 203 Januvia (sitagliptin), 110, 111, 203 Japan
direct and indirect price controls, 151, 152
generic reimbursement prices, 151 Jardiance (empagliflozin)
Johns Hopkins, 14, 15, 21, 54, 212
Johnson & Johnson, 8, 19, 51, 115 joint steering committee (JSC),
63
joint ventures (JVs), 58 JSC. see joint steering committee
(JSC)
Jumpstart Our Business Startups (JOBS) Act, 40–41
Juntado, Ryan, 86
Juvenile Diabetes Research Foundation (JDRF), 124
JVs. see joint ventures (JVs)
Kaiser Permanente, 74, 174, 178, 206 Kalydeco (ivacaftor), 42, 70, 96, 104,
128, 209
key account managers (KAMs), 111 key opinion leaders (KOLs), 102 Keytruda (pembrolizumab), 77, 78, 106, 107 killer app, 13
Lantus (insulin glargine), 203 launch strategies, 95, 105, 112, 113 Leukemia and Lymphoma
Foundation, 199 leverage, 174
big data, 225
data’s four vectors, enabling tools to, 220
Lilly, 4, 88, 105, 109, 114, 115, 190, 203 Lipitor (atorvastatin), 104, 105
liquid biopsy, 91 loan products, 37 Longman, Roger, 175
low-density lipoprotein(LDL), 104 low-level fragmentation, 180 low-price/high-value product, 156 low-price/low-value product, 156, 157 machine learning, 216, 222
causal, 223
management teams, 219 Managing the 360, 133 Maraganore, John, 26 market intelligence, 100 market research, 100 Massachusetts Institute of
Technology, 170 Mathers, Ed, 24
Mayo Clinic, 6, 13, 18, 22, 73, 110, 131, 134, 197, 206, 211
Mazzei, Chris, 239 MDLive, 202, 211 Medicaid, 152
prices, 161 programs, 152 medical
apps, 75 benefits, 173 needs, 24
medical science liaisons (MSLs), 102, 111 Medicare, 131, 152, 162, 173, 189 Medidata Solutions, 221
Medscape Professional Network, 210 Medtronic, 6, 12, 15, 16, 18, 204 Memorial Sloan Kettering’s Drug
Abacus, 181
Merck, 4, 12, 21, 49, 56, 77, 78, 96, 104, 106, 107, 110, 111, 114, 123, 127, 128, 138, 145, 165, 191, 203, 209, 222
Merck Manual, 111, 203, 209 metabolome, 72
Mevacor (lovastatin), 104 M2Gen, 128, 199 microbiome, 83 Microsoft’s Cloud, 21 Miller, Steve, 164 Miller, Tom, 91 mining metadata, 232 mobile
appointment scheduling, 133
electrocardiogram (ECG) device, 8, 12 medical device, 12
technologies, 215
mobile health (mHealth), 8, 90, 200 evolution of, 11
Mount Sinai, 17, 54, 199, 200, 218 MSLs. see medical science liaisons (MSLs) multi-omics analysis, 83–84
multichannel communications, 109–110 multidisciplinary practice units, 167 multiple forces, 217
multiple myeloma, 85, 95, 99, 181 multiple sclerosis, 75, 98, 128, 199,
207
myelodysplastic syndrome (MDS), 99, 140
Myozyme (alglucosidase alfa), 103 Nadauld, Lincoln, 74
National Academy of Medicine (NAM), 123 National Cancer Institute, 206 National Comprehensive Cancer
Network’s Evidence Blocks, 181
National Health Insurance (NHI), 152 National Health Service (NHS), 85, 118,
131, 164, 180, 206
National Health Technology Assessment (HTA) agencies, 180 National Institutes of Health (NIH)
research, 3, 84, 124
National Quality Forum (NQF), 128, 199 natural language, 216
Netflix, 231
Neupogen (filgrastim), 113 new drug
and data analytics, 226 and price-sensitive therapy, 191 applicationfiling, 12
distribution platforms, 229 positioning, 114
prelaunch sales expectations, 230 time to discover and develop, 48 new market, 33, 114
and payer power, 175
force increase payer power, 175 science, 98, 99
next-generation sequencing, 74 nondilutive capital, 31
nongovernment US payers, 174
non-small cell lung cancer (NSCLC), 106, 107
Novartis, 4, 6, 14, 19, 22, 49, 56, 60, 70, 83, 109, 114, 116, 128, 164, 181, 190, 200, 213, 227 Novo Nordisk, 105, 110, 111, 120, 124,
203, 213
nurse-staffed call centers, 137
observational studies, 97, 102, 124, 129 off-label therapy, 73
Omada Health, 14, 136, 200 Omnitrope, 116, 117 OnePath program, 134 online discussions sites, 129 online healthcare information, 8 online patient communities, 127
PatientsLikeMe, 9, 102, 121, 127, 207, 208, 213, 215
Online shopping, for health plans, 176
Opdivo (nivolumab), 77, 78, 106, 107, 109
open innovation strategies, 51 alliances with academia, 51 alliances with external venture
groups, 51 crowdsourcing, 51 innovation hubs, 51 open-label studies, 96
optimized customer penetration, 115–116 O’Reilly, Michael, 13
Organisation for Economic Co-operation and Development, 147 organizational barriers, 238 organizational structures, 219 Orkambi (lumacaftor/ivacaftor), 42, 70 Orphan Drug Act, 96, 125
Orphan Medicinal Products Committee, 121 Oscar Health, 212
OSE Immunotherapeutics, 52 Otsuka, 12
outcome-based
partnerships, 170, 171 pricing experiments, 170 reimbursement, 164 over-the-counter drugs, 108 Parkinson’s disease, 49, 199 Partners Healthcare, 128, 212 patent protection, 116–117 patient
adherence, 110 centric ecosystem, 135 engagement, 73
initiatives, 130 metrics, 140–142
engagement tools, impact of, 133 friendly delivery systems, 120 identification, 125
insight research, 124–125 management, 80 registries, 127 satisfaction, 131
ratings, 131–132 value pathway
diabetes case, 141
Patient Access and Engagement Report, 132
patient advocacy, 41, 60, 87, 112, 120, 122, 134, 138, 143, 155 patient centricity, 49, 70, 73, 120–122,
129, 138, 141–143, 167, 189, 204, 217
crowdsourcing initiatives, 124 drivers and barriers, 121–122 forces impacting, 122 framework, 121
organization models, 142–143 structure options, 143 organizing for, 138–140
points of care, connecting, 130–135 engagement, biopharma initiatives
for, 134–135
patient satisfaction ratings, 131–132 physician/patient dynamics,
132–134
strategies in R & D, 129–130 patient engagement, 21, 44, 73, 90, 120,
123, 124, 130, 132, 133, 138, 140, 144
metrics, 140, 144
Patient Focused Medicines Development Initiative (PFMD), 123 patient journey, 135–138
adherence strategies, 137–138 behavior, drivers and barriers
of, 136–137 follow-up surveillance, 135
patient key opinion leaders (KOLs), 120 patient-friendly clinical trials
designing, 125–130
patient-reported outcomes (PRO), 90, 97, 122, 124, 126–130, 135, 143, 144, 205, 213
rare disease trials, 125 trial recruitment and
retention, 125–126
patient-reported outcomes (PROs), 90, 124
comparative drug effectiveness, 126 PatientsLikeMe, 9, 102, 121, 127–129,
207, 208, 213, 215 alliance strategy, 128
business model evolution, 128 challenges, 129
competitive dynamics, 128 disease-specific registries, 127 focus vs. scale, 129
grants, 128
health data platform, as, 127 partnerships, 128
patient forum to personalized health platform, 127–129
patient-centric care decisions, 127 trial optimization, role in, 127 virtual trials, 127
PatientsLikeMe website, 9
pay-for-performance arrangement, 161 payer engagement strategies, 182–185
geospatial mapping tools, 183 information technology (IT)
infrastructure, 183
mapping payers based on behaviors and preferences, 184
strategic payer segmentation and engagement, 184 payer segmentation, 176
payer-driven patient segmentation, 87 Pehl, Michael, 98
performance-based agreements (PBAs), 164 personalized care manager, 134
Pfizer, 51, 58, 83, 97, 104, 106, 108, 109, 114, 117, 118, 123, 166, 199, 201, 205, 209
pharma-biotech alliance, 59 pharmacodynamic marker, 99 Pharmacovigilance Risk Assessment
Committee, 121
pharmacy benefit managers (PBMs), 162, 174, 179
drug costs consideration in isolation, 179 problem, 179 profitability, 177 Physician Compare, 208 physician digital trends, 210 physician ratings, 208 platinum glucose sensor, 12 Plavix (clopidogrel), 114
P-medicine, 72 point-of-care tools, 137 policymakers, 87, 169 pollution-eating bacteria, 5 Pompe disease, 103
portfolio diversification, 113–114 postmarketing surveillance, 6, 128 potential investor
factors affecting, 37 fund age, 37 future rounds, 37 investment philosophy, 37 relevant experience, 37 Pothier, Kristin, 87
Praluent (alirocumab), 61, 73, 153, 190 pre-digital age, 216
precision genomics, for cancer, 74–75 precision marketing, 94, 101
strategy, 95
precision medicine, 6, 69, 70, 94 alliances in, 50
biopharma’s engagement, 89 commercial challenges, 81–83 data collection, 86
diagnostic-driven, 79 focused start-ups, 91 forces driving, 76 genomics-driven, 74 in practice, 75–78
infrastructure challenges, 80–81 molecular-driven, 71, 90 regulatory challenges, 81
scientific and clinical challenges, 80 stakeholder expansion in, 86–87 targeted prevention strategies, 91 Precision Medicine Initiative, 71, 124 predictive biomarkers, 80
preferred stock, 35–36 Premera Blue Cross, 207 premiums, 152, 176, 179, 224 prescription drugs, 94, 108
DTC advertising of, 109 spending, in US, 94 prescriptive analytics, 224 price
concessions, 152 control, 152
flexibility
attributes determining, 158 product factors determine, 158, 159 models, 160, 161
regulations, in major markets, 149 sensitivity, 152
strategy, 157
new, deployment of, 163, 164 setting, 157
private insurance, 146 product
attributes stakeholders prioritize, 154 development, 98, 215
efficacy and safety, 153 pricingflexibility, 150 pricing strategy, 153, 165 safety, 217
unproven real-world value at launch, 153, 156 professional lifestyle coaches, 136 progression-free survival (PFS), 106 Prolia (denosumab), 103
promotion, 59, 107, 109, 110, 114, 134, 135, 154, 203
PROs. see patient-reported outcomes (PROs)
Proscar (finasteride), 114 protein coding regions, 226 proteome, 72, 83
Proteus Digital Health, 12, 197 providers, 178
financial accountability, 178 financially at-risk, 178 vertical integration, 178 public
financing market, 29 health crisis, 146 health systems, 146 private partnership, 84 Qualcomm
life ecosystem, 15
three lines of business, 14–15 licensing and acquisitions, 15 platforms, 14
venture investments, 14 Qualcomm Ventures, 14, 15, 200
quality of life, 97, 115, 124, 126, 138, 153, 154, 203, 217
quantified self, 122, 235 QuintilesIMS, 233
radio frequency identification, 200 RAND team, 170
rare diseases, 27, 32, 95, 102, 111, 116, 120, 122, 124–126, 128, 129, 140, 144, 169, 170, 198, 199, 208, 209, 213, 227
R & D programs, 216 real-time data, 215, 216 real-time intelligence, 21 real-world data, 218, 219, 230 real-world evidence, 7, 102, 107, 112,
117, 118, 122, 126, 130, 146, 162, 199, 203, 217, 227, 230, 231, 233, 234
real-world value, 218 red biotech, 5
redistributing reimbursement risk, 163 reference pricing decisions, 151 Regeneron Genetics Center (RGC),
227
regional health systems, 174 regional price bands, 159 Reid, Jeffery, 221 reimbursement, 153
delays, 165
Remicade (infliximab), 52, 118 remote monitoring, 132
patient portals, role of, 132 Repatha (evolocumab), 73, 153, 164 ResearchKit software, 6
Revlimid (lenalidomide), 95, 107 rheumatoid arthritis (RA), 222 risk-sharing agreements, 90, 161 Rituxan (rituximab), 4, 5, 76, 103 RNA interference (RNAi)
technologies, 26 Robert Wood Johnson Foundation
(RWJF), 128
Roche, 4, 49, 60–62, 72, 76, 77, 79, 88, 97, 103, 107, 114, 222 Roche-Genentech relationship,
61, 62
Sage Bionetworks, 140 salesforce strategies, 111–113 Samsung, 12, 15, 49, 197, 206 Sandoz, 113, 116, 117
Sanofi, 6, 18, 19, 49, 60–62, 84, 97, 103, 105, 114, 117, 120, 125, 126, 138, 143, 166, 190, 203, 204, 209
Sanofi digital portfolio in diabetes, 204 Sanofi Genzyme, 103
Sanofi-Regeneron alliance, 61–62 Sarbanes-Oxley Act, 41
Schadt, Eric, 218 Schleifer, Len, 160 scientific innovation, 145 scientific wellness, 11
segmentation (consumer, physician segments), 102
semiautonomous payer committees, 180 sequencing technology, 227
shared decision making (SDM), 132 shareholders, 27, 31, 35, 39, 44, 60 sharing data, 226
Shaywitz, David, 220 Sherman, Michael, 190 Shire, 124, 125, 134, 209
single payment access (SPA) scheme, 164 Small Business Innovation Research
(SBIR) program, 34 small data, 219
smartphones, 206
social content risk assessment, 209 social media, 8, 9, 14, 18, 96, 97, 112, 128,
129, 131, 135, 142, 145, 146, 197, 199, 200, 205, 206, 208, 209, 215, 217, 219, 220, 228, 232 from insight mining to co-creating,
208–210 interactions, 219 sites, 145
Sovaldi (sofosbuvir), 94, 105, 153, 161, 163, 164, 169
specialty drugs, 95 high-priced, 94, 175 report, spending on, 95 specialty pharma companies, 47 specialty therapies, 95
Spence, Pamela, 225 Spritam (levetiracetam), 201 Sprycel (dasatinib), 76 stakeholders, 216, 225
start-up biotech companies, 34, 37 statistical analysis, 170
step-therapy, 73 strategic alliances, 50–51
deal terms, 62 exclusivity, 63 financial terms, 63 governance and dispute
resolution, 63 intellectual property, 63 responsibilities, 62 scope, 62
term and termination, 63 dealing for, 56–65
mission and objectives, 57–58 monitoring results and
learning, 64–65 partner selection, 56–57 structure, 58–62
in US and Europe, 2006–2016, 59 process, 56
strategic payer engagement, 192, 193 structured data, 219
super-consumers, 217
supply chain, 7, 49, 198, 200, 201, 227, 229, 241
surgery, insurer offering medical coverage, 174 sustainability strategies, 113–118
addressing challenge of biosimilars, 117, 118 building the market, 115 franchise building, 113, 114 mature strategy, 115
portfolio diversification, 113, 114 renewal strategies, 116, 117 Tagrisso (osimertinib), 70, 76
vs. Iressa (gefitinib), 78 Tarceva (erlotinib), 76, 77
target product profiles (TPPs), 98, 112 targeted
cancer drugs, 69, 103
medicines, 69–71, 77 therapies, 70, 74, 76, 107 taxpayer-funded national, 174 Tecentriq (atezolizumab), 107 technographics, 95, 102, 103, 118 Teladoc, 210, 212
telehealth, 210
scope of services, 211
telemedicine, effectiveness of, 212 therapeutic innovations, 98 time-based bundled payment
approach, 169 tools
analytics, 239, 240 Cloud-based analytics, 9 digital, 73, 112, 146, 172, 231 for outcomes-data capture, 170, 171 geospatial mapping, 183
interactive, 106 predictive, 102 visual, 224
Toujeo (high-concentration glargine), 203
TPPs. see target product profiles (TPPs)
traditional
financing model, 32
pharma pricing practices under pressure, 148 Tradjenta (linagliptin), 115
transatlantic disparity, drug pricing, 146 transcriptome, 72, 83
transportation industries, 215 true non-dilutive capital, 31 tumor biomarkers, 80 tumor characterization, 75 Twitter, 208, 215, 232 tyrosine kinase
Bcr-Abl gene coding, 4 inhibitor aflibercept, 166 UCB, 51, 123, 124, 128, 138 UK’s National Institute for Health and
Care Excellence (NICE), 180 undisclosed discounts, 147
unit-based pricing methodology, 147 United Healthcare, 20, 86, 212
United States
adopt European-style cost-effectiveness hurdles, 181, 182
Affordable Care Act (ACA), creating new kinds of payers,
176–178 employers, 179, 180 providers, 178
and European biotechnology capital raised, 30
initial public offerings (IPOs), 29 drug pricing, 162, 163
drug valuation tools, 155 financing market, 33
Food and Drug Administration (FDA), 147
payer
Europe, governments, 174 key types, 174
nongovernment, 174
offer prescription drug coverage to, 173
operate in isolation, 174
prescription drug coverage to citizens qualify for Medicare, 173 prioritizing value to control drug
costs, 152, 153 reimbursement, 152 UnitedHealthcare
bundled payment program in oncology, 169 commercial insurers, 174 University of Washington, 160 unstructured data, 219, 221, 224
health, 216
urgency in payer community, 167 Value Labs, 165
value leakages, 141 value-added services, 112 value-based contracts, 164
work, 164
value-based healthcare, 73 value-based reimbursement, 165 value-creating event, 32
value-focused price not enough for Entresto, 182
Venter, J. Craig, 3, 84
venture capital-backed companies, 30 venture capitalists, 36, 60
venture philanthropy
Cystic Fibrosis Foundation (CFF), 42, 122, 128, 209
model, 209
Vertex pharmaceuticals, 42 Verb Surgical, 19
Verily, 6, 17–20, 22, 49, 84, 85, 125 Vertex, 25, 42, 70, 96, 122, 128, 129,
209
Veterans Health Administration (VA), 212
Viagra (sildenafil), 114, 166 Victoza (liraglutide), 110, 111, 203 Vioxx (rofecoxib), 96
virgin territory, 166 voting equity securities, 31 Walgreens, 202, 211 Wang, Jun, 128 Watson, James, 3, 17
wearables, 11, 14, 76, 83, 84, 90, 122, 128, 130, 133, 135, 137, 144, 199, 206
biosensors, 122 sensors, 72, 74
WebMD, 130, 202, 208, 211 Welldoc, 8, 12
white biotechnology, 5 whole genome sequencing, 86 Wilkins, Maurice, 3, 4 Witty, Andrew, 186
World Health Organization (WHO), 8 Xalkori (crizotinib), 106
X-ray crystallography, 3 YouTube, 101, 210, 228, 232 Zarxio, 113, 117
Zocor (simvastatin), 104
