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Drug Information Journal
, Vol. 45, pp. 541–550, 2011 • 0092-8615/2011Printed in the USA. All rights reserved. Copyright © 2011 Drug Information Association, Inc.
Correspondence Address
Peter J. Pitts, Center for Medicine in the PublicInterest (email: ppitts@cmpi.org).
Peter J. Pitts
Center for Medicine in thePublic Interest
Werner Cautreels
Selecta Biosciences
Mary Baker, MBE
President, EuropeanFederation of Neurological Associations; Vice President,European Brain Council,United Kingdom
In the last 50 years the average life span hasincreased by 10 years, largely due to advance-ments in pharmaceutical drug development, primarily within the developed world. What willdrive this over the next 50 years?Improved patient access to drugs is a critical goal for all stakeholders in drug discovery, de-velopment, and delivery. However, drug accesslevels for patients continue to decrease, drivenby, for example, stricter regulatory policies,lower R&D output, stronger payer cost pres-sures, and a complex legal environment. Giventhe importance of health and well-being, itis critical for stakeholders to better under-stand these hurdles and develop high-impactsolutions that signicantly increase patient ac-cess to new medicines. While pharmaceuticalcompanies have had some limited success inovercoming R&D hurdles, a multistakeholder holistic approach is necessary to develop para-digm-changing solutions. A meeting of 12 distinguished health care ex- perts was convened on January 26–27, 2010,in London, UK, to address this issue. The par-ticipants included government regulators,health care policy experts, industry leaders,health economists, health care attorneys, pa-tient advocates, and academics. The primaryobjectives of the meeting were to further under-stand the hurdles to patient access and to de-velop high-impact global solutions. Topics dis-cussed included the following:• The nature of the problem• Initiatives currently underway• Insights on the challenges and barriers thatinhibit patient access to new and innovativemedicines• Prioritizing ideas to facilitate meaningfulstrategies for expediting patient access tonew medicines• A potential path forward for implementing the priority ideas from the meeting Five of the ideas that were discussed are as fol-lows:1. A nonprot, independent, patient-drivendrug assessment agency to provide an ap- praisal of risks versus benets of new drugsto both individuals and the society as awhole, unbiased by nonclinical measure-ments such as the quality adjusted life year (QALY)2. An academic institute of regulatory scienceto drive research into the discipline of drug regulation3. A new Asia-Pacic panregional regulatoryagency to provide centralized regulatorysupport4. Better sharing of data within the pharma-ceutical industry (eg, safety databases, ge-nomic and small-molecule libraries, negativeoutcomes) directly, or potentially through government bodies5. Enhanced use of private-public partnershipsin new product development building onlearning from organizations such as the Billand Melinda Gates Foundation, the FDACritical Path Initiative, the Innovative Med-icines Initiatives, and the Cardiac SafetyResearch Consortium.
The column reviews important discussion and in-sight emerging at the meeting, and is intended asa call to action highlighting the importance of improved global patient access to new medicines.The rst two of the ideas just described wereviewed as top priority based on feasibility and potential impact.
Expediting Patient Access to New Medicines:A Call to Action, London, January 26–27, 2010
meeting reports
541
introduction
 
Innovation is at the heart o humankind’s well-being and a driving orce behind impressive im-provements in the standard o living. Over thepast 50 years the advent o new medicines hasbeen a key driver o signifcant increases in lieexpectancy and quality. While oten invented inclose collaboration between academic and in-dustrial scientists, these drugs were mostly de-veloped and commercialized by the pharma-
 
542
meeting reports
 Pitts, Cautreels, Baker
ceutical industry. However, despite an ever-increasing global burden o chronic diseases,the number o applications or new medicinescontinues to decline and time lines to deliverdrugs to patients continue to increase despitemassive expansion o investments into R&D andenormous advances in understanding biologi-cal systems.While patients, the most important stakehold-ers o all, have become more educated aboutdisease, their share o voice in the beneft-riskand beneft-cost debates has not increased com-mensurately. Furthermore, new demographicshave exacerbated other needs in terms o howwe measure therapeutic impact including mor-tality and morbidity, ability to unction, andquality o lie. Addressing these issues requiresnovel approaches regarding the need and bene-fts o incremental versus breakthrough innova-tion. Furthermore, the voice o patients in thedeveloping world will continue to be an impor-tant actor in this debate, one that must be con-sidered or any solution to be complete.An aging population in the developed world,as well as the growing lie expectancy o the de-veloping world, stands to urther exacerbate theproblem. For example, by 2030, individuals age65 and over will comprise approximately one-fth o the US population (1). While we are liv-ing longer,
1
we are also experiencing unprece-dented rates o cancer, America’s second lead-ing cause o death (2). At the beginning o 2007,in the United States approximately 12 millionmen and women were alive who had a history o cancer o all sites (3), and as our populationages, we can expect greater numbers o us willbe living with and seeking treatment or thisdisease.These challenges will persist and perhaps in-tensiy as the global population grows and thedeveloping world continues to present increas-es in liestyle diseases such as coronary arterydisease, stroke, obesity, and type 2 diabetesmellitus. In the past, liestyle diseases were dis-eases o the auent, and uncommon in the de-veloping world. It is now predicted that by2020, these diseases will be causing 7 out 10deaths in developing countries (4). The devel-oping world is now aced with a dual dilemma o having both liestyle diseases and communica-ble diseases (5). Access to critical lie-savingdrugs will be an imperative or these nations,particularly in the midst o severe economicconstraints. Given the importance o this issueand the many stakeholders involved, a holisticlook is warranted to create a new paradigm ordeveloping and delivering innovative medicinesto patients. This is both a challenge and a moralduty or all stakeholders involved.This report describes discussions at a meet-ing aimed at a holistic analysis o critical actorslimiting early patient access to medicines, andcreating a process toward the resolution o these issues. The path orward must include allrelevant stakeholders, such as the biopharma-ceutical and medical device industry, payers,providers, regulators, and health policy makers.Pivotal to the success o any initiative will beregulators and policy makers who can help torefne the beneft-risk equation underlying pa-tient access to new medicines.
overview of the natureof the problem
The main challenge impeding access to innova-tive medicine lies in the optimal assessment o beneft versus risk or new therapies rom devel-opment, regulatory, and delivery perspectives.There are fve key elements to the problem, de-scribed below.
Decining R&D PRDuctivity nDtheR inDutRy FctR
The traditional development and approval cycleo R&D in the pharmaceutical realm has seendecreasing success and increasing timelines orregulatory review, with an added impact on in-tellectual property (IP) protection. The result isreduced patient access to novel medicines anddisincentivization o innovation in pharmaceu-ticals.Likewise, the industry’s perceived lack o transparency has contributed to a poor percep-tion by the public, urther complicating intro-duction and marketing o new products. For ex-ample, there is a need or greater transparency
 
Expediting Patient Access to New Medicines
meeting reports
543
Drug Information Journal
o several actors including total spending ondrug development (particularly those that ailto reach market), physician payments by phar-maceuticals, and total expenditure on lobbyistsand relationships with policymakers.
itigtin enviRnment
Excessive litigation protection issues havecaused physicians and manuacturers to bemore risk averse in their choice o therapies topursue. Tort considerations have been particu-larly important to the pharmaceutical industryin limiting the risks o high R&D investment.Highly regulated and closely watched, pharma-ceutical companies have recently experiencedsignifcant setbacks. A poor public image andrising fnancial costs resulting rom litigationaround saety-related issues have become alltoo common. The recent
Wyeth v. Levine
case,
2
 or example, showed that a drug with seriouspotential adverse eects may trigger a winninglawsuit, despite clear saety warnings on its la-bel. Similarly, physicians suer rom the rapidgrowth in malpractice lawsuits. The resultingrisk aversion drives pharmaceutical companiesand physicians to orgo potentially attractivedrugs due to potential saety concerns, therebydepriving access or the subset o patients orwhom the drug could be sae and efcacious.
inteectu PRPeRty PRtectin
IP protection timelines have changed little inthe past 15 years, resulting in shorter time onthe market or most drugs due to the combinedeect o longer time to approval and increasedability o generic pharmaceutical manuacturersto roll out new products. The requirements orpatent protection as part o the developmentprocess urther disincentivize development o drugs, especially in diseases with low commer-cial viability. In addition, the rise in “pay to de-lay” (6)—where a branded drug owner pays apotential generic competitor to delay genericintroduction—only highlights the continuousdeensive measures companies are taking to pro-tect their asset prior to loss o exclusivity.There is a great need or IP reorm that allowsowners o branded pharmaceuticals and gener-ic manuacturers to have greater certainty in IPprotection. In addition, alternatives to the cur-rent period o exclusivity are worth consideringto encourage branded manuacturers to investin technologies that may be more expensive toproduce but enable improved beneft-risk pro-fles or the patient subpopulations with great-est need.The combination o IP and tort system reormwould have signifcant impact on medicine in-novation and would provide much-needed im-petus to biopharmaceutical companies to investin innovation. Such reorm would involve com-pelling changes and landmark legislature butrepresent a critical step toward expediting pa-tient access to new medicines.
RegutRy Picie
Enhanced ocus on drug saety by both regula-tors and consumers highlights the importanceo identiying patient subpopulations or whoma particular drug’s benefts would outweigh therisks. As a result, drug saety standards and datademands are now more stringent.For example, the risk evaluation and mitiga-tion strategy program, which calls or manuac-turers to collect and analyze additional inorma-tion on product saety, has been legallyenorceable in the United States since 2007 ora broad and expanding range o drugs. Thepharmaceutical industry is slowly adapting tothe demands o the evolving regulatory environ-ment, but it has not ully internalized behavioralchanges called or by more rigorous regulations.In addition, as European regulators (eg, UK,Germany) are getting increasingly concernedabout cost eectiveness o approved drugs, pa-tient access to certain new drugs is becominglimited. In 2009, a prominent case saw Roche/Genentech deciding not to seek approval o Avastin (a cancer drug) in the UK rather thancomply with local regulators’ request or datarequired to complete a cost-eectiveness evalu-ation, stressing the need or closer cooperation.
PyeR ct PReuRe
Increasing overall health care costs have re-sulted in cost control eorts rom both gov-
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