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Editorial: Public-Private Partnerships as Drivers of Innovation in Healthcare

This article was originally published on Frontiers in Medicine. Authors: Remco L.A. de Vrueh, Jon S. B. de Vlieger, and Daan J. A. Crommelin

A partnership driving healthcare innovation

Editorial on the Research Topic
Public-Private Partnerships as drivers of innovation in healthcare

The format: from bilateral to multilateral

Around the turn of the century, a rather simple classification of public-private-partnerships (PPPs) in the world of medicine development sufficed. These PPPs consisted primarily of bilateral collaborations between pharmaceutical companies and academic institutes. Since then, these “simple” bilateral PPPs have been complemented by different and more diverse types of PPPs. On the one hand, PPPs emerged such as the Medicines for Malaria Venture (MMV) or the Drugs for Neglected Diseases Initiative (DNDI) with as major drivers charities, country donors, industry, and academic groups. These so-called product development partnerships (PDPs) focus on developing products for specific communicable diseases impacting health of patients in less affluent countries. On the other hand, Pharma-PPPs, such as the Innovative Medicines Initiative (IMI), emerged that focused on jointly tackling specific -precompetitive- issues in medicine development. The major players in the last category consisted of the pharmaceutical industry (large pharma), small, and medium sized enterprises (SMEs), academic institutes and–again- governmental funding programs (12). Since then the background of participating stakeholders of PPPs has greatly diversified. Important new stakeholders joined the PPP consortia, including patient organizations, regulatory bodies, health technology assessment agencies, insurance companies, and IT-companies (see articles in this special issue, e.g., Aartsen et al.) All have their unique incentives to join, which makes the PPP concept more difficult to define and to evaluate in terms of its benefits. Nowadays, many PPP-flavors exist and the number and diversity continues to grow. Contributions to this special issue exemplify this current development in the PPP-world.

Added value: in the eye of the beholder or more concrete impact measures?

Early on, questions were raised about the assessment of performance and success-failure of PPPs (13). Performance indicators to look at were identified as: the input, the process, the output, the short-term outcome, and impact. See Figure 1 for details. The basis for this methodology was already developed and tested in other fields. What makes the Pharma-PPP case so special are the long timelines–years- to measure “impact.” The classical PPP projects have a typical running time of 4–6 years. The long-term outcome-and impact e.g., in terms of concrete new medicines can only be measured many years after finishing the project and on top of that there are many “diluting” contributing factors in the post-PPP years. Moreover, simply looking at the number of medicines developed based on the activities of a PPP significantly underappreciates the additional impact from knowledge transfer, ongoing collaborations, patents, spin-off companies formed, and last but not least the educational aspect PPP initiatives offer (See Figure 1). The true impact of the first generation of PPPs now becomes visible and we can review that according to the key performance indicators set out from the start [cf. (45)].

Table representing the reported performance indicators to be considered in a research public-private-partnership (PPP) performance measurement system
Figure 1. Reported performance indicators to be considered in a research PPP performance measurement system, classified into 5 categories. Figure adapted from (2).
2. De Vrueh RL, Crommelin DJ. Reflections on the future of pharmaceutical public-private partnerships: from input to impact. Pharma Res. (2017) 34:1985–99. doi: 10.1007/s11095-017-2192-5
PubMed Abstract | CrossRef Full Text | Google Scholar

In that light, there is one question that was often raised in the early days and that can now be answered, i.e., the concern about the quality of the research output -read publications- of PPPs. Several studies made it clear (34) that the impact of publications measured in terms of impact factor of scientific journals and number of citations of IMI and TI Pharma consortia was comparable–if not higher- than of articles published through “regular” academic groups efforts.

Sustainability: to stay or to perish?

What is the chance for a consortium to survive after finishing the first funding round? Before answering this question it should be clear whether the project, topic-wise, is supposed to be continued at all? Some projects simply do not have a horizon beyond their running time. They are set up to solve a particular -often concrete- problem. However, what if a prolonged existence is foreseen? Experience teaches us that then already in an early stage the question of sustainability should be addressed. For instance, in case infrastructure has been built up, such as databanks or test facilities, further strategies to continue activities after the first funding round should be subject of discussion early on. The article by Aartsen et al. in this special issue discusses various sustainability strategies developed for IMI projects in detail and lists “lessons learned.”

Evolution: PPP Quo Vadis?

The adoption of the “open innovation model” by the pharmaceutical industry has given the PPP concept a big push. Originally, the public partners were mainly academic and national or international public funding organizations. The large pharmaceutical industry with or without SMEs took care of the private side. Over time, the background of stakeholders in PPP consortia has diversified. Patient organizations and health insurance companies joined the consortia. Regulatory bodies such as EMA and FDA are becoming partners as well, although these institutions are very cautious to safeguard their independence from large pharma and other private stakeholders. Big IT organizations such as Google and Amazon (cloud-computing services) expanded the spectrum on the private side (Moreno et al.) as did medical device-diagnostics companies such as Siemens, Agilent, and Philips in the context of IMI. This expanding source of partners will change the character of PPP consortia. Also, the scope of activities evolved. As partners in first PPPs were jointly exploring science and collaboration in a truly pre-competitive field, a shift toward projects where partners share their strategic assets is now observed. E.g., in the IMI—European Lead Factory (see this issue: Karawajczyk et al.) industry decided to share some proprietary assets allowing competitors and public partners to boost their drug discovery programs. It demonstrates that the PPP concept has become a trusted way of working and partners now seem comfortable to evolve the model with activities closer to their core business.

These recent developments raise the question whether the original, rather narrow definitions of a PPP as mentioned at the beginning of this editorial will properly describe the PPPs in medicine development in the future. Partners outside pharma now join the game and change the dynamics and “culture.” The walls between the classical “silos” disappear rapidly.

The remaining question is then. PPP concept in the world of medicine development: Quo Vadis?

References

1. Denee TR, Sneekes A, Stolk P, Juliens A, Raaijmakers JA, Goldman M, et al. Measuring the value of public–private partnerships in the pharmaceutical sciences. Nat Rev Drug Discov. (2012) 11:419. doi: 10.1038/nrd3078-c1

PubMed Abstract | CrossRef Full Text | Google Scholar

2. De Vrueh RL, Crommelin DJ. Reflections on the future of pharmaceutical public-private partnerships: from input to impact. Pharma Res. (2017) 34:1985–99. doi: 10.1007/s11095-017-2192-5

PubMed Abstract | CrossRef Full Text | Google Scholar

3. Gunn M, Lim M, Cross D, Goldman M. Benchmarking the scientific output of the Innovative Medicines Initiative. Nat Biotechnol. (2015) 33:811–2. doi: 10.1038/nbt.3305

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4. TI Pharma. New Tracks to Medicines. (2014). Available online at: http://www.lygature.org/files/atoms/files/TI%20Pharma%20Report%20-%20New%20Tracks%20to%20Medicines.pdf

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5. CTMM. Translating Science Into Better Healthcare. (2016). Available online at: http://www.lygature.org/sites/www.lygature.org/files/atoms/files/CTMM%20Translating%20Science%20into%20Better%20Healthcare.pdf

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Keywords: editorial, public-private parternships, healthcare, innovation, medicine

Citation: de Vrueh RLA, de Vlieger JSB and Crommelin DJA (2019) Editorial: Public-Private Partnerships as Drivers of Innovation in Healthcare. Front. Med. 6:114. doi: 10.3389/fmed.2019.00114

Received: 02 May 2019; Accepted: 07 May 2019;
Published: 31 May 2019.

Edited and reviewed by:Michel Goldman, Free University of Brussels, Belgium

Copyright © 2019 de Vrueh, de Vlieger and Crommelin. This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) and the copyright owner(s) are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.

*Correspondence: Daan J. A. Crommelin, d.j.a.crommelin@uu.nl

Improving the Translational Medicine Process: Moving Patients From “End-Users” to “Engaged Collaborators”

This article was originally published on Frontiers in Medicine. Authors: Manuela Battaglia1,  Pat Furlong2, Nico Martinus Wulffraat3, and Felicitas Bellutti Enders4

  • 1Diabetes Research Institute, IRCCS San Raffaele Scientific Institute, Milan, Italy
  • 2Parent Project Muscular Dystrophy, Hackensack, NJ, United States
  • 3Department of Pediatric Immunology, Wilhelmina Children’s Hospital, University Medical Centre Utrecht, Utrecht, Netherlands
  • 4Allergy Unit, Department of Dermatology, University Hospital, Basel, Switzerland
patient involvement in translational medicine

Translational medicine works through the definition of unmet medical needs, their understanding and final resolution. In this complex and multi-disciplinary process patients have always been regarded as “end-users” or no more than “data provider.” Considering that the translational practice is nowadays highly inefficient (i.e., large intellectual and economical resources are wasted with limited impact on people health) here we propose to reverse the process: start from patients, engage them, and keep them at the center. A new partnership needs to be formed between the patients and the health care professionals, as well as the treating physicians, to make the most out of the current “health resources.” New patient-centric approaches are emerging but they remain isolated phenomena often difficult to implement. Here—with this perspective—we aim at thinking differently and learning from new experiences. We will provide some successful examples of change, and we will discuss new approaches to create a radical change in the way translational medicine is managed and how this would significantly impact people health and health care systems.

Introduction

The European Society for Translational Medicine has defined translational medicine as an interdisciplinary branch of the biomedical field supported by three main pillars: benchside, bedside, and community. The goal of translational medicine is to combine disciplines, resources, expertise, and techniques within these pillars to translate efficiently and effectively scientific research findings relevant to human diseases into knowledge that is beneficial for patients via new drugs, devices, or treatment options (1). Accordingly, translational medicine is a highly interdisciplinary field and includes academia, industry, and regulatory institutions. However, patients (who are the direct beneficiaries of translational medicine) are often excluded by this complex process.

In this perspective paper, we will discuss the impasse of translational medicine, the role that patients should have in this process (with concrete examples of success) and the future directions with the aim at fostering a science that really impacts on patients’ life.

The Impasse of Translational Medicine

Translational medicine is a process fundamental for the society as it aims at developing new interventions beneficial to the patients. However, translational medicine is at a historic moment of crisis. The process is becoming unsustainable in spite of enormous technological advances, since the technological explosion has not been accompanied by a reinforcement of quality in experimental designs, especially in the discovery phases. However, there is no clear path neither for clinicians nor for scientists regarding the process of how a discovery leads to an approved drug. The high level of failure of clinical trials in Phase II/III swallows up economic resources, generates exhaustion among researchers and clinicians and, most importantly, induces frustration among patients who see their hopes for a new drug to treat their disease, disappear (2). The high failure of clinical trials can be due to the inadequate study design, incorporating endpoints that provide limited or misleading information regarding the efficacy of the test agent, to the limited reproducibility of data, or also due to the high variability between tested subjects regarding their genetic background or the heterogeneity of their disease and also their comorbidities (3). Although it is now clear that even the failure of well-designed studies benefits both researchers and healthcare systems by, for example, generating evidence about disease theories and demonstrating the limits of proven drugs (4).

The Food and Drug Administration (FDA) publishes every year a list of all the drugs released on the market 1. Backtracking the initial publication on the mechanism or molecule leading to drug development shows—for the drugs released between January and September 2018—a median interval of 10 years (range: 5–37 years) before the drug is reaching the patient. This is becoming unsustainable as it creates tremendous social distress. At a time of global financial crisis, citizens perceive that vital resources are not being used efficiently and scientists fear to enter in a career path that is uncertain and not properly rewarded.

Thus, there is a great need to reconsider the translational medicine process and we do believe that moving the patients from “end-users” to “engaged collaborators” would transform them into agents of change. The standard business model is indeed to speak to the consumer. Apple for instance understands its consumer: it must first identify the customer, talk about the product and ask if the intended consumer would value the product. Is it any wonder why Apple is the first billion-dollar company? They know their consumer! In translational medicine, this concept is ignored. Patients are the ultimate users of health technologies and they can advocate and promote models for patient involvement among other stakeholders. Nothing will facilitate the dialogue among scientists, clinicians, and society more effectively than the creation of a pathway, constructed together, and bound by a common objective. This should lead to improved translational medicine efficiency and reduced waste of resources and energies.

Current Advances

The doctor-patient relationship in western countries has significantly evolved over the years. Prior to the last two decades the relationship followed a paternalistic model, where the patient sought help and the doctors used their skills to choose the necessary interventions or treatments to restore or improve patients’ health. Decisions of the doctors were silently complied by the patient (5). The social system has been challenged over the last 20 years: society has changed (being now multicultural), access to information is broader (social), media allow easier contact between patients and thus facilitated creation of patient’s organization. Therefore, critics have emerged, demanding a more active, autonomous and thus centered role for the patient who advocates greater control, reduced physician dominance, and more mutual participation.

This has led to the idea of the Shared Decision Medicine (SDM), which is a process promoted by the Institute of Medicine (IOM) as part of the strategy to improve the quality of health care in the United States. The IOM recommended that healthcare should be customized based on the patient’s needs and values, the patient should be given adequate knowledge and control to make decisions that affect his/her health, patients and healthcare providers should communicate and share information, and patients should receive information that allows them to make informed decisions. To this end, SDM is the joint involvement of patients and healthcare providers in making healthcare decisions that are informed by the best available evidence in regards to possible options, potential benefits and harms, and that consider patient preferences and values. SDM ensures patients get no more and no less of the care they need and want (67). However, despite attention to principles and competences, there remains a lack of clear guidance about how to accomplish SDM in routine practice. Studies have not yet addressed the question about the impact on professionals. There might be the need to coach patients to be able to assess the value, risks, benefits, and burden of interventions. For organizations, a consistent shared decision-making might change patient experience evaluations and lead to a “satisfied patient” and fewer complaints or even legal issues. Clear outcome measurements of shared decision-making are needed as they would provide a more substantive evidence base to guide implementation (6).

Another, more recent, approach to bring the patients closer to the science that could impact their life is the “plan S.” Research funders from France, the United Kingdom, the Netherlands, and eight other European nations have unveiled a radical open-access initiative; they will mandate that, from 2020, the scientists they fund must make resulting papers free to read immediately on publication. The scientific papers would have a liberal publishing license that would allow anyone to download, read it or otherwise reuse the work leading to a science no more locked behind paywalls and freely available for everybody (8).

Increasingly, funding opportunities for translational biomedical research require studies to engage community partners, patients, or other stakeholders in the research process to address their concerns. However, there is little evidence on strategies to prepare teams of academic and community partners to collaborate on grants. A well-planned and feasible educational program designed to help community organizations and academic institutions to build infrastructure for collaborative research projects using a partnered approach is needed and some institutions are already investing in this important activities (910).

Industry is today also very open to view patients as close collaborators and aims at connecting with them throughout the innovation journey, starting with validation of new concepts to the design of patient-centric trials. The customer journey is a term from marketing, describing the 5 cycles, which a client passes through before he decides to buy a product, or in medical terms, before he decides for one or the other therapy. Five phases mark this journey: awareness, favorability, consideration, intent to purchase (or in medical terms, intent to treat), conversion (decision to treat). For most pharma companies, this represents a major shift in thinking. It requires putting not the product but the customer at the center of the launch, and addressing customers’ emotional and behavioral needs as well as their clinical ones.

There are also tangible examples of success on how to move the patients at the center of the translational medicine process. Here we report two specific cases.

The Remarkable Story of a Mother and the Parent Project Muscolar Dystrophy

When doctors diagnosed her two sons, Christopher and Patrick, with Duchenne muscular dystrophy (DMD) in 1984, Pat Furlong didn’t accept the therapeutic nihilism, the fatalistic message from their doctor “there is no hope and little help.” DMD is the most common, lethal genetic disease diagnosed in childhood; it is an aggressive and ultimately fatal muscle wasting disease that primarily affects boys and it results in a progressive loss of muscle strength. Individuals with DMD lose ambulation in the early teens, require ventilation in the mid-teens and die before reaching the 3rd decade. Families who receive the diagnosis are in a race against time. They await new knowledge and scientific breakthroughs, possibilities to slow the degeneration. As of today, steroids are used to slow the decline, but there are no cures for DMD. In 1994, together with other parents, Pat Furlong founded Parent Project Muscular Dystrophy (PPMD) to change the course of the disease and ultimately end DMD. PPMD is today the largest most comprehensive non-profit organization in the United States focused on finding a cure for DMD 2. In her quest for a cure, she first realized that there simply wasn’t enough research into the disease and too many questions being left unanswered. Her first efforts focused on small investments in academic research and leveraging those investments. Due to the rarity of the disease (1 in 4600−5600 boys) and hence lack of potential profits there had been little interest at the onset from major pharmaceutical companies. Early in the fight, PPMD realized that the greatest source of advancement in basic science surrounding DMD would be through an investment by the National Institutes of Health (NIH) and related agencies. In 2000, the Duchenne community, through PPMD, employed a firm to lobby on their behalf in Washington DC and scored major legislative success with the introduction of legislation, intended to require government agencies such as NIH to significantly increase its investment in and coordination of research into the muscular dystrophy’s. That same year, at the insistence of PPMD, NIH held a scientific workshop on Duchenne, bringing in scientists from all over the world to advance the cause. This was a workshop of major significance in which attending scientists’ and researchers came to the realization that with the knowledge of the genetic basis of the disease and through multidisciplinary collaboration, something could be done to improve the quality of life and extend the lifespan of boys with DMD. On the tails of the earlier success with NIH, PPMD continued its Washington DC advocacy agenda and achieved another stirring victory. In December 2001, the Muscular Dystrophy CARE ACT was signed into law. This legislation dramatically increased NIHs investment in Muscular dystrophy research (from ~$17 millions to over $750 millions), including the funding of six Centers of Excellence. All of that, in addition to the earlier orphan drug act of 1983 incentivizing companies to invest in rare disease research, resulted in significant breakthroughs and new knowledge to fully characterize the pathology of DMD and to encourage industry interest in targeting relevant pathways. Today there are more than 40 ongoing clinical trials, whereas in 1999 there was 1 trial. Additionally, today there are more than 45 pharmaceutical companies investing in DMD. Current market estimates an 8-Billion-dollar investment in drug development. PPMD is currently working with FDA to develop a Master Protocol to enable access to trials across the Duchenne community, potentially leading to combination therapies and reach the highest priority of families (Figure 1).

Patient advocacy has come of age. Foundations focused on a specific disease provide substantial investments in research, organize the patient community, collect data to better understand disease progression, support the development of biobanks, inform regulatory interactions and assist patients navigate the healthcare environment. Advocacy efforts lead the ecosystem of research, therapy development, access and reimbursement. Her sons lost their battle with DMD in their teenage years, but Pat Furlong continues to fight—in their honor and for all the community to this day.

The Development of a Patient Council Within a Clinical Department

Another, yet different, example of success comes from the Department of Pediatric Rheumatology at the Wilhelmina Children’s University Hospital in Utrecht (The Netherland) focused on the study and cure of Juvenile idiopathic arthritis (JIA). JIA is the most common chronic rheumatic disorder in children and is a major cause of short-term and long-term disability. JIA is defined as having an inflamed joint before the age of 16 without a clear cause that persists for more than 6 weeks; it is a chronic disorder, which if neglected, can lead to serious complications.

In developing a network for biological research for patients with Childhood Arthritis doctors and scientists at the Wilhelmina Center of Excellence strongly think that input from and collaboration with patients and patient organizations is crucial. Patients, their parents, doctors and researchers all share the same common goal, namely that progress in basic science is translated in real tangible products for patients with childhood arthritis. In 2013 a patient council was formed in this Department. Together with professionals the JIA patient council explore research priority setting by reviewing the research topics, safety and efficacy of immunizations, as well as stopping medications. In addition to this, a jointly written application was obtained for a project with focus groups for patients that was also led by a parent. The patient council selected a topic which was the most frequent concern expressed by patients: the uncertainty patients feel due to the impact of the unpredictable course of their disease (pain, relapses) in their activities of daily life (activities at school for younger children and later work, sports and social contacts). Focus groups further analyzed the effects of the unpredictable course of the disease. Information was written for websites and two youtube movies were made. The group made of Dutch organizations of patients, parents and clinicians will collaboratively develop a research agenda for JIA, following the James Lind Alliance (JLA) methodology 3. The JLA is a non-profit making initiative established in 2004 and it brings patients, caregivers and clinicians together in Priority Setting Partnerships (PSPs) to identify and prioritize the top 10 uncertainties, or unanswered questions, about the effects of treatments. The aim of this is to make sure that health research funders are aware of the issues that matter most to patients and clinicians. In this process the input from clinicians, patients and their caregivers will be equally valued. Additionally, focus groups will be organized to involve young people with JIA. The involvement of all contributors will be monitored and evaluated. In this manner, the project will contribute to the growing body of literature on how to involve young people in agenda setting in a meaningful way.

This approach, despite still at its infancy, will inform researchers and research funders about the most important research questions for JIA and this will hopefully lead research agenda for research that really matters (11).

Current Obstacles and Future Directions

The examples provided show how patients and their care givers can be the catalysts of a change that is highly needed in translational medicine but they remain, as per today, sporadic cases led by unique human beings or by particularly inspired institutions. Many obstacles remain. Qualitative research showed that the involvement of patients and caregivers is challenging: real co-design does not happen by itself (12). First, specific educational programs are needed to improve the process of shared decision-making, for both partners, the patient and the physician. These programs are missing and importantly clinicians are often limited in their time-management. Educate and engage patients is a time-consuming process but health insurances—as well as hospitals—push more and more to reduce the time spent with patients, as costs of medication, exams, and personnel are dramatically increasing.

Scientists are even farther away from this process, as they often do not have direct contact with the patients. Current criteria for promotion in the medical field still rely heavily on individual research output such as high impact publications, h-index, grants, and invited lectures. There is tremendous pressure and on top of this pressure, there is really no space for a patient-centric view that needs time, patience and dedication. Especially in a system where these activities are not properly recognized and, as a consequence, rewarded. To change this, institutions need to ensure that their tenure and promotions systems are able to evaluate and recognize the contributions investigators conducting translational medicine make. Many institutions are working in this direction and, for instance, signed the Declaration on Research Assessment (DORA). DORA recognizes the need to improve the ways in which the outputs of scholarly research are evaluated. The declaration was developed in 2012 during the Annual Meeting of the American Society for Cell Biology in San Francisco4[4]. It is a worldwide initiative covering all scholarly disciplines and all key stakeholders including funders, publishers, professional societies, institutions, and researchers. It is a first step toward assessing research based on its own merits rather than on the basis of the journal in which the research is published.

In conclusion, translational medicine is a very complex branch of medicine. The constant challenges of teaching, researching, publishing, and competing for limited sources of funding, coupled with pursuing career aims and ambitions, can seem daunting. On top of this, we are also adding the patient-centric4 view, which adds another level of complexity. However, we believe that once the obstacles are overcome, the real inclusion of patients in the process of translational medicine will improve healthcare delivery to patients.

Author Contributions

MB conceived the topic, contributed to the topic discussion and wrote the manuscript. PF and NW contributed to the topic discussion and contributed to manuscript writing. FB contributed to the topic discussion and wrote the manuscript.

Funding

MB is supported by research grant from JDRF, NIH and the Italian Ministry of Health.

Conflict of Interest Statement

The authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest.

Footnotes

1. ^https://www.centerwatch.com/drug-information/fda-approved-drugs/drug/100246/lutathera-lutetium-lu-177-dotatate

2. ^https://www.parentprojectmd.org/

3. ^http://www.jla.nihr.ac.uk/about-the-james-lind-alliance/about-psps.htm

4. ^https://sfdora.org/

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Keywords: translational medicine, patient-centric approaches, shared decision medicine, the innovation journey, patient advocacy

Citation: Battaglia M, Furlong P, Wulffraat NM and Bellutti Enders F (2019) Improving the Translational Medicine Process: Moving Patients From “End-Users” to “Engaged Collaborators”. Front. Med. 6:110. doi: 10.3389/fmed.2019.00110

Received: 10 October 2018; Accepted: 03 May 2019;
Published: 21 May 2019.

Edited by:Wu Yuan, Johns Hopkins Medicine, United States

Reviewed by:Shi-Cong Tao, Shanghai Sixth People’s Hospital, China
Marian Klinger, Opole University, Poland

Copyright © 2019 Battaglia, Furlong, Wulffraat and Bellutti Enders. This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) and the copyright owner(s) are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.

*Correspondence: Manuela Battaglia, mbattaglia@telethon.it

Present Address: Manuela Battaglia, Telethon Foundation, Milan, Italy

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My professional development in recent years. It’s a real opportunity to be part of the global medical team of Sanofi consumer healthcare. Indeed I have joined Sanofi at the local level by working on transversal projects, medico-marketing, pharmacovigilance, and clinical studies.
Today I have a specific medical position, with close interaction with regulatory and pharmacovigilance teams. As a partner, I give my best, and I also learn from others to face future challenges.

  • What is your favorite hobby and/or activity outside of your work?

 Football, scrabble, cinema, reading

  • What do you enjoy most about being a Medical Affairs Professional?

As a physician, my greatest satisfaction is to contribute to the well-being of the patient through an assessment of products based on their efficacy and safety datas, and to propose a risk mitigation plan. In addition i like the medical strategy because it helps to identify the real needs of patients and to propose the appropriate actions.

  • What has been the most beneficial aspect of the IFAPP Academy Program?

This program gives a broader view of the medical affairs function, and the skills required. The role of the medical affairs professional is well highlighted in all activities of the pharmaceutical industry, and this helps to better understand how we can be a key partner of other functions.

  • How has your view of the medical affairs profession altered and/or improved?

I limited the medical affairs to my personal experience but this program has shown me the evolution of the profession and the added value of our role in the life of the drug. Some topics that were abstract are now concrete for me. It is a specialty that deserves to be recognized in view of the complexity of the various skills required to be a professional in medical affairs. This profession has a major role in all decisions concerning the life of the product, from the discovery of the molecule to the monitoring and innovation of the mature product. The professionals must be multi-skilled and have a critical judgment in all discussions hence the extensive knowledge provided by this program.

  • Any additional comments that you would like to make about the Program?

This program is an opportunity to exchange with experts and other professionals from the pharmaceutical industry through webinars and forums. In addition to deepening our knowledge, it is a professional network that is created.

Thanks to Sanofi which is a partner of this program, and thanks to the IFAPP team for your expertise.

International Perception of Competence, Education, and Training Needs Among Biomedical Professionals Involved in Medicines Development

The below excerpt is from an article originally published on the Frontiers in Pharmacology website. To check out the full article click here.

Medical Affairs Books

The development of new medicines today, requires a multi-professional workforce, both in industry and the clinical research arena. Pharmaceutical physicians (PPs) and medicines development scientists (MDS) need a certain level of competence, achieved through on-the-job experience, with a postgraduate education foundation and continuous professional development programs. In order to assess the self-perception of competence, education and training needs, an on-line questionnaire based on the seven domains of competence, developed by IFAPP-PharmaTrain, was prepared and distributed among PPs and MDS members of IFAPP’s affiliated professional associations in countries with facilities for postgraduate education. The data collection was run over a fixed period of three months in Japan, Italy, Brazil, and Spain during 2017. Results indicate low but variable levels of perceived competence for the various domains as well as seniority in the job. All respondents declared a significant need for continuing professional development in all domains. These results corroborate and support the continuous efforts, put in place by IFAPP and the PharmaTrain Federation, to foster the development of accredited education and training among professionals involved in medicines development.

Introduction

For some time now, the biopharmaceutical industry has been the key link between basic biomedical discovery and the emergence of novel medicines that prolong or improve life. However, the industry faces several ongoing and emerging challenges, including technical knowledge gaps, limitations in clinical testing, lowered productivity, higher development costs, increased regulatory requirements, growing payer pressures and patent expiration.

The lack of an adequately sized and appropriately trained multi-professional workforce, both in the industry and the clinical research field, is also a significant part of the problem. There is a perceived mismatch between the profiles and abilities of graduates from academic programs in healthcare professions, and the changing needs of the various health systems around the world. As a possible solution to achieving a transformative learning, an outcomes-based education, or competency-based education (CBE), has been proposed (Silva et al., 2013). Competent professionals would be able to perform their specific responsibilities effectively, such as bringing and maintaining new medicines to the marketplace. A need for competency-based education and training has been identified in the United States, Europe, and Latin America (Dubois et al., 2016).

These respective professional groups have been left with the responsibility to define the competencies needed to perform their function effectively. Competencies can be clustered in domains and can be learned through proper postgraduate education or continuing professional development (CPD) (Sonstein et al., 2014).

The International Federation of Associations of Pharmaceutical Physicians and Pharmaceutical Medicine (IFAPP)1 and the PharmaTrain Federation (PharmaTrain)2 assumed the task of producing the defined core competencies to orientate Pharmaceutical Medicine and Medicines Development as a discipline and profession. Three areas, seven domains and 57 core competencies at the cognitive level, were identified (Silva et al., 2013). The domains have been summarized in a Statement of Competence.

In addition to serving as a template for job profiles and portfolios, the domains can be used to identify general education and training needs. Based on these premises, an international survey among members of the IFAPP national member association was designed using an online questionnaire. Stakeholders were asked about their self-perception of competence and the need for education and training. The results were then assessed to identify gaps, in order to address the potential need for future development of pharmaceutical physicians and medicines development scientists.

By:
Kyoko Imamura1*
Domenico Criscuolo2
Anna Jurczynska3
Gustavo Kesselring4
Peter Stonier5
Tatsushi Tsuda1 and 
Honorio Silva6

Copyright © 2019 Imamura, Criscuolo, Jurczynska, Kesselring, Stonier, Tsuda and Silva.

The IFAPP Academy welcomes Ana B. Salgado as the new Director of Strategic Planning

Ana B has extensive professional experience in Medical Affairs, Marketing, and senior Business Management. We look forward to her contributions to the sustained growth of the Academy and achieving our mission and goals.

Ana B. Salgado, IFAPP Academy Director of Strategic Planning

Dr. Ana B. Salgado is a highly motivated and energetic pharmaceutical business executive with extensive global experience.

A physician by training, after three years of practicing medicine in the emergency room setting, Dr. Salgado started her pharmaceutical career as Medical Affairs Manager in Roche- Colombia. Her high affinity for change and continuous learning took her career into marketing and the commercial business. She joined Pfizer, where she occupied positions of increasing responsibility in marketing at a country level (Colombia) and later, at regional (Latin America, Europe) and global levels. She worked on products and portfolios across several therapeutic categories including anti-infectives, anti-inflammatories, and neuroscience in collaboration with multi-disciplinary teams around the world. Dr. Salgado was responsible for the launch of Celebrex in Latin America, several neuroscience launches in Europe (Zeldox, Relpax, Lyrica) and she led the Global Team for Aricept (in an alliance collaboration with Eisai – Japan).

Expanding her knowledge into the commercial business, Dr. Salgado served in business operations roles at regional and global level. She became the Chief of Staff for the Established Products/ Emerging Markets Business Unit at Pfizer, a business unit with a $25B yearly turnover. In these roles, Dr. Salgado led and participated in several large and complex corporate initiatives including the mergers of Pfizer with Warner Lambert, Pharmacia and Wyeth; several company reorganizations and the creation of the Established Products Business Unit. Before leaving Pfizer, Dr. Salgado led a global transformation to a customer centric – digitally enabled marketing model.

Dr. Salgado received her medical degree from Pontificia Universidad Javeriana in Colombia. She is currently pursuing a Certificate in Financial Planning.

IFAPP Academy 2019 Cohort Excelling!

The 2019 Cohort has just completed the first of six Modules required to earn their Medical Affairs in Medicines Development Certification (CMD). In the past month students have learned about the purpose and identity of medical affairs, received an introduction to pharmacology, overviewed the clinical trial process, networked with their fellow students during webinars and lesson discussions, and much more!

We are proud to announce that we have a few students who received perfect scores for their work in Module 1! This means they attended all lectures, webinars, and achieved full marks in participation for each activity. This is by no means a small feat. Their dedication to the Program has not only exceeded expectations through their academic achievements but also through their interactions with their fellow students.

We look forward to sharing with you the continued success of each one of the students in this Cohort.

Want to learn more about the IFAPP/King’s College London, Medical Affairs in Medicines Development, Certification Program?

The International Federation of Associations of Pharmaceutical Physicians and Pharmaceutical Medicine (IFAPP) Academy provides online Continuing Professional Development for Medical Affairs Professionals.

The IFAPP Academy is a non-profit organization whose mission is to promote, develop, and implement educational activities that support Pharmaceutical Medicine by enhancing the knowledge, expertise, and skills of pharmaceutical physicians and medicines development scientists worldwide. Partnered with King’s College London, the Academy offers Professional Certification for students successfully completing the course. Click here to learn more about the benefits of Professional Certification from the IFAPP Academy.

IFAPP Academy Meeting in Budapest

Budapest 2018

In November 2018, the IFAPP Academy was invited by Sandor Kerpel-Fronius to hold the annual face-to-face Steering Committee meeting at the prestigious Semmelweis University in Budapest, Hungary.

Just a few of the Academy’s Board members and operational team

The IFAPP Academy Board Members, stakeholders, IFAPP’s secretariats, and the operations team gathered the first evening in Budapest to share a light dinner, a couple of drinks, and some good conversation.  

The first morning of meetings kicked off with Professor Sandor Kerpel-Fronius, faculty at the Semmelweis University, giving us a history of the University, insider tips to the city, and an overview of the meeting agenda. 

Professor Sandor Kerpel-Fronius inviting us inside the Semmelweis University for our first meeting.

The Academy was excited to welcome Tim Aldwinckle, UCB representative, to the meeting. He was able to provide some great perspective in several areas of the discussion.

After a full day of presentations, the team enjoyed an amazing dinner at Budapest’s Museum Restaurant.

The IFAPP Academy meeting attendees gather for dinner at the Museum Restaurant

Day 2 of the meeting – Gustavo Kesselring and Xavier Luria discussed the strategic collaborations that the IFAPP Academy will be fostering in 2019. Peter Stonier also presented the updated contract with King’s College London. The afternoon was concluded with coffee and the recording of video testimonials starring some of the key representatives of the Academy.

It was a great few days in Budapest and wonderful for the team to be able to spend some quality time together and workout the logistics for a successful 2019 and beyond!

Check out some more of the pictures from the Budapest Steering Committee Meeting: