Medical Innovations for Neglected Patients - Doctors Without Borders

Medical Innovations for Neglected Patients - Doctors Without Borders

MEDICAL INNOVATION FORNEGLECTED PATIENTSImportant Progress Over Past Ten Years, But “Fatal Imbalance” PersistsSouth Sudan 2012 © John Stanmeyer/VIIOver the past half century, there have been unprecedented improvements inhealth outcomes, spurred in part by unparalleled scientific progress in thepharmaceutical sector. Yet access to the benefits of medical progress and scientificadvancement has not been equitably shared and many innovation gaps remain.

Three fundamental problemsThere are three fundamental problems with medical innovationtoday.First, global public health needs are not in the driving seat.Regardless of how great the needs may be, where commercialpotential is weak, there is little “pull” to develop newtechnologies. The innovation cycle is broken, with few or noincentives for the development of effective, safe, quality,suitable and affordable health technologies – leading toneedless suffering and death. There are numerous illustrationsof the lack of suitable diagnostics, vaccines and medicines(see text boxes). In addition to innovation gaps for Chagasdisease, drug-resistant tuberculosis (DR TB), and vaccinepreventableillnesses, other examples include diagnosticsand treatments for leishmaniasis and human Africantrypanosomiasis (sleeping sickness); medical tools such asnew diagnostics and appropriately adapted formulations forchildren with HIV/AIDS and TB; and new antibiotics to addressthe rise in antimicrobial resistance, which has made treatingpreviously treatable diseases more difficult.Second, as a result, developing countries must often “makedo” with innovation that primarily caters to conditions indeveloped countries. Medical tools are too often developedfirst for developed countries and only rolled out in resourcelimitedsettings in a second stage. Newer vaccines againstrotavirus, for example, may have the potential to prevent lethalchildhood diarrhea in Africa, but they have been developedwith resource-rich conditions in mind. Rolling them out indeveloping countries will pose severe strains because thesevaccines are incredibly bulky and come with considerablecold chain capacity requirements. i These worrisome findingsshould come as no surprise, however. If new medical tools aredeveloped for and tested in developed countries alone, theneeds of populations in developing countries will inevitably bean afterthought.Third, even when there is enough of a profit incentive to driveinnovation – for example when diseases affect both developedand developing countries alike – the resulting products aretoo often priced out of reach. Developing countries are notthe only ones to be hit, as ever higher prices for new medicaltools strain the healthcare budgets of developed countries aswell, posing access barriers to increasing numbers of people.New drugs to treat HIV or cancer can cost hundreds of timesmore than a person’s average annual income, and the battlefor access increasingly has to be waged drug by drug, countryby country, company by company.Medical innovation must aim to change practice, for the benefitof patients. But ideas, knowledge and inventions can onlybenefit patients who have access to the fruits of innovation.What is needed, therefore, is not just innovation – but bothinnovation and access.The WHO Commission on IntellectualProperty Rights, Innovation and PublicHealth: The Innovation CycleLack of medical innovation and lack of access to medicaltools, particularly as they affect developing countries,were first acknowledged as serious problems by the WorldHealth Organization (WHO) over two decades ago. Theneed for governments to take concerted action has beenclear since the publication of the report of the Commissionon Intellectual Property Rights, Innovation and PublicHealth (CIPIH) in 2006 ii .The report conceived of research and development (R&D)as a cycle, with three major phases feeding into eachother: discovery, development and delivery.Innovation CycleDemand fornew/improvedtools andpost-marketingresearchDISCOVERY• Lead identification/optimization• Basic researchDELIVERY• Getting productsto patients“3D”INNOVATIONCYCLETranslationalresearchDEVELOPMENT• New/Improvedtools• Preclinicaland clinicaldevelopmentMarket approvaland manufactureThe report of the Commission on Intellectual Property Rights, Innovation,and Public Health (2006), WHO.The driving force behind the innovation cycle – theexpectation of a high return on investment – has beenincreasingly recognized as the source of critical failings,particularly for developing countries and neglected people.1

Defining neglected diseasesserious, disabling or life-threatening disease can beA considered neglected when treatment options areinadequate or don’t exist, and when their drug-market potentialis insufficient to readily attract a private sector response.Infectious and parasitic diseases that predominantly orexclusively affect people in developing countries are mostcommonly understood by the term “neglected diseases,” buteven within this category a distinction is often made, basedroughly on levels of R&D activity, between “neglected” diseasessuch as HIV/AIDS, malaria, TB, and dengue, for example (wherethere is more commercial or semi-commercial R&D activityand generally speaking more resources available), and “mostneglected” diseases, such as sleeping sickness, leishmaniasis,Chagas disease, Buruli ulcer, and other neglected tropicaldiseases (where there has typically been little to no R&D activityand few resources). These diseases are distinct from “globaldiseases,” such as non-communicable diseases, for which boththe public health needs and R&D activity are significant, butfor which a majority of people affected in developing countriesare not of interest for the pharmaceutical market. Innovationis therefore not necessarily adapted to their needs, andtechnologies that do exist can often be out of reach due to highprices. The shortcomings extend to areas such as antibiotics,where new medicines need to be developed. As low- and middleincomecountries now face a double burden of disease, withboth infectious diseases and, increasingly, non-communicablediseases, all areas of market and/or public policy failure – allthe instances where the existing system has failed to meetpublic health needs – must be addressed.Neglected Diseases: Primarily Affect DevelopingDeveloping Countries & Lie Outside the GlobalCountries & Lie Outside the World Market*Source: IMS HealthNeglected Diseases Primarily AffectGlobal DiseasesPharmaceutical MarketWorld pharmaceutical market$856 billion in 2010 *MostNeglectedDiseasesNeglectedDiseases*Source: IMS HealthThere is also significant R&D activity for non-medical “lifestyle”conditions – such as baldness, erectile dysfunction, cellulitereduction, and so forth – for which there are no pressing publichealth needs. This is represented in the graphic as the whitespace that falls outside the medical needs circles but within theshaded box representing the pharmaceutical market.per year for TB, malaria, sleeping sickness, and leishmaniasiscombined. iii Findings from a survey of the top pharmaceuticalcompanies showed little private sector investment or activity inthe field of neglected diseases. In the five years prior to 2001,no company had brought to market a single drug for sleepingsickness, Chagas disease, leishmaniasis, malaria, TB, or otherviral, bacterial, or fungal infections (excluding HIV/AIDS). iiiThe UNICEF/UNDP/World Bank, WHO Special Programme forResearch and Training in Tropical Diseases (TDR) was a majorplayer and had been operational for 25 years; most neglecteddisease products registered were done so with support fromTDR, even though funding remained chronically low.While certain research institutions such as the US NationalInstitutes of Health have always been major funders ofinfectious disease R&D, particularly for HIV/AIDS, governmentsby and large have not filled the vacuum left by industry,particularly for the “most neglected” diseases. Instead, publicsector research was increasingly focused on diseases affectingprimarily developed countries, actively pursuing possibilitiesfor commercialization. At this time, philanthropic actors likethe Bill & Melinda Gates Foundation, Wellcome Trust, andRockefeller Foundation were beginning to get involved andstarted playing an increasingly large role in funding public/private partnerships, leading to concerns that governmentswere abdicating their responsibility and letting the action offoundations act as an “alibi for government inaction.” iiiIn 2012 A broader range of actors and initiatives, butinsufficient coordination and sustainable funding: Over thepast decade, new R&D initiatives have been launched by abroad range of stakeholders including academic groups andemerging countries, including India, Brazil, and South Korea.A majority of pharmaceutical companies are now engaged3

DNDi/Anita KHEMKA2003, xii a policy process at the WHO levelhas been underway and culminated in theApril 2012 report of the independent WHOConsultative Expert Working Group onR&D Financing and Coordination (CEWG),which provided a blueprint for action. Thereport recommended that all countriesinitiate formal negotiations towards aglobal framework – an R&D Convention– that would strengthen coordinationand financing of R&D and ensure the costof R&D was de-linked from the price ofproducts in order to meet the needs ofdeveloping countries.Despite a clear recognition by allstakeholders that market incentives arefailing to generate biomedical innovationthat meets the needs of certain patientpopulations, primarily poor people livingMother with child suffering fever in kala azar endemic area, Bihar, Indiain developing countries – and to guaranteeaccess to the fruits of this innovation –the WHO Prequalification Programme, while not explicitlydesigned as an R&D enabling mechanism, has played athe urgent, concerted action needed fromgovernments to address this situation has not yet materialized.major role in facilitating regulatory approval of medicines inIn November 2012, countries met at the WHO to discussdeveloping countries, thereby increasing access – includingconcrete next steps in relation to the WHO CEWG adapted formulations for pediatric HIV drugs and fixed doseUnfortunately, the only concrete agreement made was incombinations – for patients most in need. There is a clearrelation to monitoring health R&D through establishing aneed to closely monitor and assess these new mechanismsGlobal Health R&D Observatory within the WHO. This is anwith regard to their impact both on innovation and access.important and necessary first step, but this is nowhere nearwhat is required to address the magnitude of the challenge.Toward a SustainableInstead of pushing forward with a real plan to address theNeeds-Driven System forcontinued lack of suitable and affordable vaccines, drugsGlobal Health R&Dand diagnostics, all countries have really pledged to do isIto continue observing the situation. There is a disconnectn 2001, the MSF and Drugs for Neglected Diseases Workingbetween the recognition of the scale and urgency of theGroup report concluded with a series of recommendations toproblem, which is widely shared, and the fact that proposalspolicy-makers. iii Many of these – a call for WHO to lead an R&Dfor transformative change have been pushed back forpriority-setting exercise to identify the most pressing gaps; aanother four to governments to develop comprehensive solutions tothe R&D crisis, including possibly through an R&D treaty; a This is for increased, reliable and long-term funding for neglecteddisease R&D; and a call for measures to ensure affordabilityand equitable access to the fruits of innovation – remainstartlingly relevant today.After ten years of debates, international negotiations and thepublication of numerous expert reports, the imperative is clearfor governments to take concrete action and spur biomedicalinnovation in areas neglected by the current innovationAt the global level, over the past decade, efforts to put theinnovation crisis on the international political agenda havesystem. They cannot shirk their responsibility to put in place asustainable global framework for essential health R&D.been slow-moving. Since the inception of the Commission onIntellectual Property Rights, Innovation and Public Health in What is needed is public leadership, notably from WHO and6

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