Patient Advocacy Archives - Marksman Healthcare

How Well are we Able to Quantify ROI from Patient Centric Activities?
In recent years, patient-centric initiatives are becoming key factors among healthcare companies, as they are increasingly becoming aware of the fact that the key to growing business and improving customer health is to better focus on the needs and concerns of the patient, rather than attending to just the product approval. The objective behind patient-centric drug development and other associated initiatives is to involve patients and the healthcare community as partners in order to bring about a sense of ownership in the success of new medical treatments. (1,2) There are four core principles that frame the growing arsenal of patient centric initiatives: (3)
1. Relevance: Unmet medical needs identified in collaboration with and based on input from patients and their healthcare support network
2. Pragmatism: Agendas and clinical trial designs recognised and accommodated in real-life patient and healthcare community needs and experiences
3. Feasibility: The burden of study participation minimised and supported by initiatives that improve convenience
4. Interactivity/Participation: Patient’s voice responded with the support from patient community, and giving the patient community an opportunity to be actively involved throughout the research process by amplified planning and execution
Adherence to the above principles facilitate patient-centric drug development to encourage the sharing of important information and drug development risk among a broader community of external partners e.g., academic and basic research groups, co-development sponsors, development operations alliances, and patient advocacy groups. (3)

A growing number of researchers are adapting to patient-centric initiatives across multiple studies, which can eventually be used while centrally monitoring and coordinating activities. This can further promote harmonization and can also better assist in communicating lessons learned from earlier implementations and from peer companies. Such activities can also help in deriving consensus metrics to evaluate the impact of various initiatives on organizational and study-level processes and performance. (3,4)

Return on investment (ROI) expectations are required to consider a reasonably long-term view. This is because researchers need enough time to collect experience with patient-centric initiatives, learn from mistakes while continuously processing their use. Ideally, target measures from multiple representative studies should be gathered two to three years prior to and after the implementation of initiatives. Stakeholders use some key implementation and ROI metrics to measure three broad areas, viz. reach; patient/study volunteer feedback; and performance. (3)
- Reach measures (e.g., number of pilot initiatives, number of planned initiatives, etc.) are usually quantitative, having an aim to assess the extent of adoption and usage within organisations, along with the number of patients and study volunteers who have participated in a given initiative.
- Patient/study volunteer feedback measures (such as ratings, etc.) are more often qualitative in nature; however, several organisations do implement a few quantitative feedback measures as well. Qualitative measures examine the subjective reports of satisfaction; sense of involvement in a given study or in association with a specific initiative, and the perceived relevance of specific clinical trials; while quantitative measures determine efficiency of patient/study volunteer participation in facilitating change. (3,4)
- Performance measures (viz. screen failure rates, number of procedures per visit or protocol amendments, etc.) are quantitative and they largely compare studies, whether or not they include the usage of patient-centric initiatives or not. (3)
We feel that it is too early to conclude on this aspect, as there is inadequate data demonstrating the extent and impact of patient-centric initiatives, across the industry. Needless to mention that patient-centricity movement is certainly inspiring the drug development enterprise to challenge and transform the traditional drug development prototype by putting the patient at its core.

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References
1. Lopez Kunz B. Trial Return On Engagement: Quantifying Benefits Of Patient-Centric Initiatives. Clinical leader. June, 2017.
2. A patient-centric approach: Patient-centric initiatives in drug development. March, 2017.
3. Getz KA. ROI for patient-centric drug development. Applied Clinical Trials. August, 2015.
4. Gets K. The transformative promise of patient centric R&D. May, 2015.
5. Markowitz FE. Involvement in mental health self-help groups and recovery. Health sociology review : the journal of the Health Section of the Australian Sociological Association 2015; 24(2):199-212.
MarksMan Healthcare

July 3, 2018

Patient Advocacy, Patient Centricity, Patient Outcomes, Patient Reported Outcomes
Taking Patients as Partners in Research

The purpose of medicines is to improve patients’ lives. Despite the increasing number and scope of patient involvement initiatives, there is no accepted master framework for systematic patient involvement in pharma-led medicines development. Patient engagement is more productive, but inconsistent and fragmented on a broader level. Incorporating the patient perspective in healthcare research is strongly promoted by policy makers, funding bodies and international regulators. Many theoretical benefits from patient involvement in research have been reported, such as improving the relevance of research questions, improving the recruitment of study participants, and increasing the chances for funding and dissemination of results. In addition, there is an increasing recognition of the essential role of patients in outcome research.

Patients have a personal experience of disease that is not available to most researchers, but that complements researchers’ analytical skills and scientific perspective. Patient or consumer involvement in research is therefore recommended, with theoretical benefits being that research grounded in relevant clinical need, patient perspectives, and patient priorities will enhance study design, practicality, recruitment, data interpretation, and dissemination. Patients can be involved in research by identifying and prioritizing topics, reviewing grant applications, analyzing and interpreting data, and disseminating findings. Involvement can mean consultation, collaboration, or consumer-led research.

Involving patients in research projects improves both the methodology and outcomes of the research, and offers invaluable additional insights. Contributions by patients to the design, implementation and evaluation of research lead to effectiveness, credibility, and often to more cost efficiency as well. It is essential to ensure that high quality research brings real benefits for patients and their daily lives.

Findings from these types of studies, where patients are considered partners, suggest that personal feedback from researchers as well as patients indicates that including the patient perspective in scientific projects is not easy. And although many people involved are convinced of the potential benefits of patient participation, they are still struggling with the question of ‘how to do it’.

Patient involvement can only be successful if patients are sufficiently prepared and supported to make a valuable contribution. Not only do patients need adequate support, but researchers also need help with practical tools and information about the conditions that make patients’ participation worthwhile. The involvement of patients in research should be an active and equal engagement between patients and researchers right from the start of the project. Increasing number of studies are now being conducted involving patients in the research. For many reasons patient representatives are engaging with researchers to improve methodology and research outcomes, to give credibility to the results and to acknowledge the fact that for ethical reasons patients should have a say in health care and health research when it is expected that decisions in these areas will have an impact on their daily life. In the last decade patient involvement has been shown to be beneficial in different contexts of research. In particular, in the development of Patient Reported Outcomes, patients play a role in addition to that of study participants. They become collaborative partners in a process of co-production.

Effective patient engagement is a time consuming process and significant investment. The research team and patient partners need to be open to the risks and be flexible in this work together. Mutual trust and integrity are key components to keep open conversation flowing and offer the possibility of allowing the patient voice to impact research studies, which can be incredibly valuable in providing end-use of research results. Future studies with a direct impact on patient-centered outcomes research would directly benefit from engagement with patients as full-team members in their research programs.

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MarksMan Healthcare

January 30, 2017

Patient Advocacy, Patient Reported Outcomes, PROs
Patient Engagement in Healthcare Decision Making

Modern healthcare system is very complex, and many patients struggle to obtain, process, communicate, and understand even basic health information and services. Many patients lack health literacy, or a true understanding of their medical conditions and find it difficult to follow instructions on how to take care for themselves or to adhere to treatment regimens. Above all, due to paucity of time, many practitioners fail to provide the information that patients need to make the best decisions about their own care and treatment.

‘Patient engagement’ is often used as an umbrella term to cover the multiplicity of interactions that patients have with their healthcare system. Terms such as ‘patient empowerment,’ ‘patient participation’ and ‘patient involvement’ are sometimes substituted for the term ‘patient engagement.

“Patient activation” refers to a patient’s knowledge, skills, ability, and willingness to manage his or her own health and care. On the other hand, “Patient engagement” is a broader concept that combines patient activation with interventions designed to increase active involvement and promote positive patient behavior, such as obtaining preventive care or exercising regularly.

Patients’ participation in decision making process in healthcare industry has become a political necessity in many countries and healthcare systems around the world. Literature reveals that participation of patients in healthcare has been associated with improved treatment outcomes and quality of life. With enhanced patient participation and considering patients as equal partners in healthcare decision making, people are encouraged to actively participate in their own treatment process and follow their treatment plan. Furthermore, a growing body of evidence demonstrates that patients who are more actively involved in their health care experience not only show better health outcomes, but also incur lower costs. As a result, many public and private health care organizations are employing strategies to engage patients, by educating them about their conditions and involving them directly in making decisions about their care.

In India too, there is an increasing awareness about this concept. With an aim to promote and facilitate patient engagement with healthcare providers and policy makers, Indian Alliance of Patient Groups (IAPG), an umbrella body of 12 patient groups working in the areas of rare disorders and chronic diseases, was launched in Mumbai in 2015. With member groups spread across India, IAPG has drawn up a broad action plan in accordance with their mission to facilitate and ensure a safe, effective, reliable patient-centered health system in India that provides timely access to the best available interventions for prevention, diagnosis, treatment and care. The action plan includes: promoting patient engagement in health policy decision making, supporting access to the best available care across the country, promoting health literacy and ensuring the availability of health information, advancing choice and empowerment of patients and their advocates, building a strong voice in support of building a responsive, patient-centered national and state healthcare system.

Although the concept of patient engagement promises to deliver significant value for health care providers, patients and other health care stakeholders, many gaps still exist for patient engagement to make an impact at large. This perspective towards engagement may help policy makers in setting research and funding agendas that can really improve the quality, relevance, ethical dimensions and implementation of the research and ensure that research resources address the issues which are most important to patients affected by health care problems.

Furthermore, this approach can help in drafting guidelines for planning interventions to foster patient engagement in a way, that is really fine-tuned with the specific phase of the patient’s experience. It may also contribute to understanding the real value of patients being involved in the care process according to a patient-centered approach.

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MarksMan Healthcare

August 29, 2016

Patient Advocacy

Indian Alliance of Patient Groups, patient empowerment, Patient Engagement, patient involvement, patient participation
“Voice of the Patient” in Improving Drug Access
Drugs which might seem very effective in clinical trials, sometimes fail in the market due to non compliance and acceptability issues from the patients. On the other hand, a drug with known and serious side effects is sometimes well accepted by the population, despite the risk involved.

An eye opener in this regard is the case of the Tyasarbi, the drug against Multiple Sclerosis. It was approved in 2004 by the US FDA, and withdrawn hardly 3 months post its launch, after being associated with cases of PML. However; the response of Tysabri in non responsive MS patients was so radical, that in spite of knowledge of life threatening and serious side effect as PML the patients were willing to take a risk with the drug, as opposed to allowing progression of MS. This was evident as representatives of MS patients and their relatives testified to the drastic improvement shown by the drug in patients of MS. There was a strong opposition to the withdrawal of the drug by the patients, who due to the non availability of the drug were being sentenced to progression of MS and ultimately a vegetative life. Thus in 2006 FDA ultimately decided that the side effects of Tysabri though serious, were acceptable considering the benefit accrued to the patient and the risk of non availability of the drug. Subsequently the drug was also approved for treatment of non responding patients of Crohn’s Disease.

Another case was the role played by patients is the case of patient led advocacy group for Duchenne’s Muscular Dystrophy (DMD). A group of more than 80 representatives from the Duchennes community held discussions on their expectation from drugs, the risk they were willing to take for clinical trials and at what risk of non curative retardation of disease was acceptable to them. A guidance document intended for the industry and regulatory authorities was then submitted to the FDA to give a direction to drug development in the field. FDA in turn got actively working with the pharmaceutical company Serepta on the development of the drug Eteplirsen for DMD.

These incidences led US FDA to come up with a unique initiative that allows patients to voice their opinions regarding their disease, how it impacts their life, their opinions on the current treatments available and what they expect from new drugs and therapies in the future. This program is aptly called as Patient Focused Drug Development (PFDD) program. The reports of these meetings are released to the public and industry, being aptly named as ‘Voice of the Patient’ reports.

PFDD have the following positive impact in the pharmaceutical world:
- It helps to give the patients’ viewpoint to the type of drugs needed in the market.
- The interaction provides the stakeholders, that is the patients & disease advocacy groups, the clinicians and researchers, and the payers, and the biopharmaceutical industry, with an opportunity to utilize this information in bringing about a positive change in the drug development process.
- Involving patients with drug development in the early stages would enable the researchers to get a better insight to the problems and possibly, newer avenues for drug discovery.
- Patient’s experiences would help in development of Patient Reported Outcomes (PRO) and help in Clinical Outcome Assessment(COA) in clinical trials.
- Since payers are increasingly depending on Real World Evidence to support their decisions, having a feedback from the end users of the drug well before the launch, gives payers an insight into how the drug will be accepted post its launch.
- For the success of any pharmaceutical product there has to be a market need, scientific opportunity to produce a drug and reimbursement or insurance coverage. PFDD ensures that the industry is made aware of the needs of the people or what they expect from the drug.
- It would help the industry to cater to the needs of the patients and also reduce the time taken for them to gain access to new drugs.
I believe that with the processes for systematic representation of patient’s voices, the regulatory authorities will now have a holistic outlook to how the drugs affect the patient’s life, the need for newer therapeutic options for diseases helping faster access of patients to drugs and clinical trials.

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MarksMan Healthcare

February 2, 2016

Patient Advocacy

Patient Advocacy, Patient Focused Drug Development, Voice of the Patient