How pharma can support doctors who treat rare diseases

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Where do doctors see patients with rare diseases, how do they keep up with treatment advances and what can pharmaceutical companies do to help?

Those were just some of the questions addressed by exclusive research carried out by Cambridge, Massachusetts-based healthcare market research company MedPanel for this issue of pharmaphorum’s Deep Dive magazine.

The cohort of 100 doctors that took part in the online research are among those at the frontline of treating patients with rare diseases. On average 75% of their professional time is spent on patient care.

The survey focused on five rare diseases: multiple myeloma, lupus, haemophilia, spinal muscular atrophy (SMA) and cystic fibrosis patients (CF). The doctors within the survey that see these patients specialise in rheumatology (25% of respondents), haematology (22%), neurology (18%) and oncology (17%).

Where do doctors see rare disease patients?

Looking at the most common pathway that patients with rare diseases follow when they come into the care of the survey’s respondents, the single most common route to a rare disease specialist was via referral from a non-specialist provider. Indeed, in the UK this is essentially the only pathway that can be followed.

US-based doctors treating rare diseases said that a few of their patients would be self-referred or come to them based on a recommendation from another patient with the same condition (about 8-10%). Smaller numbers of patients are still steered to them by advocacy groups or referred by a hospital (about 4%). Nevertheless, 72% of patients seen in the US were referred from a non-specialist provider.

How do rare disease doctors keep up with new developments?

Almost 90% of respondents feel they are keeping up to date with treatment options for the rare diseases they deal with, and the same proportion feel that pharmaceutical manufacturers support their efforts to stay current on treatment options.

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When it comes to the type of support that these physicians lean towards, journals and conferences/seminars are almost universally relied upon to help 98% of the physicians questioned remain current in their specialty.

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Online searches were cited by over 70% of the physicians surveyed, and for an average 70% of doctors it was interactions with their professional peers, such as grand rounds, that were their favoured means of staying up to date.

Other channels seen as important were information provided by rare disease advocacy groups (relied upon by 20-30% of doctors) and information from pharmaceutical companies (30-40%).

What challenges do these doctors face?

Limited treatment options was the greatest challenge noted by the survey’s doctors treating rare diseases, so it’s no surprise that they viewed the most exciting development on the horizon as being new treatment options, with biologics and immunotherapy finding particular favour.

Drilling down into what they would wish to see from medicines, there was a wish for safer treatments that can be taken on a long-term basis, while a common complaint was the lack of biomarker availability to help guide either treatment decisions or patient monitoring.

The second most frequently mentioned challenge was with the disease complications that they see, noted by 29% of respondents. Meanwhile, 10% of respondents focused on ‘patient access challenges’, in terms of getting patients to disease experts or getting them access to newer therapies.

Compliance, traditionally an issue well-understood by the pharmaceutical industry, was seen as less important for the doctors in this survey, with just 6% of them saying it presented a challenge, mainly in terms of patient fatigue with treatments and their long-term disease.

Market access issues, such as the cost of drugs, were also identified as issues for some. “In the UK, getting the drugs we want in the order and combinations that we want,” noted one respondent, adding: “In the world [the challenge is] making CAR-T cheaper and less toxic.”

Is greater rare diseases education needed?

The majority (84%) of doctors that took part in the survey were clear that more education about rare diseases is needed, with patients and non-specialist caregivers identified as those most in need of greater information about these conditions.

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However, about half (55%) of the physicians surveyed admitted that they themselves would also benefit from more education about rare diseases, with that result found to be more pronounced among those doctors engaged in treating multiple myeloma, 61% of whom thought they needed more education.

Interestingly, respondents rarely cited the general public as a group that needs more education about rare diseases.

 

 

 

 

Do doctors work with rare disease patient groups or pharma?

The doctors in this study spent none of their professional time working with patient advocacy groups within rare diseases, and 43% went so far as to avoid any involvement at all, although 13% said they would have a low-key involvement.

However, they do have some potential exposure to the pharmaceutical industry. Two percent of respondents attend or present at medical conferences and 10% take part in research activities.

With that in mind, opinion was mixed as to whether the pharmaceutical industry should be involved with rare disease patient groups, with 26% of respondents saying they should not against 15% who thought they should.

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Looking at the smaller group in favour of pharma involvement with rare disease patient groups, the thinking was that sharing unbiased information would be the best way to do so.

How could pharma help?

Asked how pharmaceutical manufacturers could support non-specialist healthcare providers’ educational needs, the provision of appropriate educational materials was mentioned by 70% of those surveyed as a way pharmaceutical manufacturers could provide education support.

Second to that, making online webinars available to non-specialist healthcare providers was cited by over 55%, followed by organising – and sharing – evidence-based outcome information (about 50% of respondents).

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A sizable minority (40%) thought that having sales representatives or medical science liaisons interact directly with healthcare providers other than physicians was the best way to support their educational needs, but it was still the least frequently selected option.

On the question of how pharmaceutical manufacturers could better support doctors in their efforts to stay up to date on treatment options, offering educational grants was the most frequently chosen method of doing so. Such grants were chosen by 94% of UK physicians, but only 66% of their US counterparts.

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Providing more clinical study reprints from well-respected medical journals was the second most frequent choice.

However, increasing the number of sales rep or MSL visits was a relatively unpopular option for how manufacturers could better support doctors in their quest to stay up to date on treatment options, selected by only 19% of those surveyed.

The research

The research, produced exclusively for pharmaphorum by MedPanel, involved 100 ‘rare disease treaters’, with the numbers split evenly between the US and UK.

The doctors that took part in the online survey during January 2019 treat one of five rare diseases: haemophilia, multiple myeloma, spinal muscular atrophy (SMA), cystic fibrosis (CF) or lupus.

Respondents had at least two years’ medical practice and most of those who took part in the study had been in practice for 17 to 20 years.

Conclusion

The MedPanel poll of doctors was clear that most feel they keep up to date with treatment options for the rare diseases that they treat, and that the pharmaceutical industry supports their efforts to stay current.

Around 7,000 rare diseases have so far been identified, but treatments for them remain few. So, it’s no surprise that the doctors in our survey said that limited options were the greatest challenge that they faced.

Improving that situation remains the biggest contribution the pharmaceutical industry can make. But in the meantime, as the survey showed, help with education and – for some at least – working with patient groups would benefit from pharma’s focus.

Could talking to patients be the biggest innovation in clinical trial recruitment?

This artcile was first published in March 2019 and is written by Havas Lynx Faze, a specialist patient recruitment and engagement agency focused on delivering better patient experiences within clinical trials’

  • 80% of trials are failing to recruit the required number of patients
  • The way the industry speaks to patients has not shifted in years
  • What companies are missing when it comes to patient recruitment

     

    imagesClinical trial recruitment is in crisis, with 80% of trials failing to recruit the required number of patients on time. There are few instances in life where we are happy achieving a score of two out of ten, so why are we allowing this to happen in an industry where the end result is a delay in getting life-changing treatments to patients (not to mention billions of dollars of additional costs)?

    At the root of this problem is an industry that has been slow to adapt to drastic change over the past 20 years. If we compare the number of trials registered on clinicaltrials.gov there were 13,460%1 more trials registered in 2018 than there were in the year 2000. To put this into perspective, the number of registered trials has increased six times more than Amazon sales over the same period of time. Unfortunately, we haven’t changed our approach at the same pace.

    In a time where Google, Apple, Airbnb, Uber etc. have transformed nearly every industry to be completely customer-experience centric we often still take an approach that avoids considering the end customer all together. Instead we focus on the feelings and needs of investigative sites and HCPs missing the fact that the patients are now the ones in control.

    A new type of competition

    The growth in the number of trials has been coupled with the exponential growth in the use of the internet and smartphones, which has put patients, caregivers, family members and friends in control of their healthcare. There is now an unprecedented amount of noise in the competition for a patient’s attention. With the average person thought to see as many as 3,000 advertising messages a day across all media, the competition for share of voice is no longer just between competing studies, but has expanded across alternative and holistic therapies, as well as powerful consumer brands such as Apple, Facebook, Amazon and Nike.

    Speaking to patients like a consumer brand

  • The competition has shifted, but the way that the industry is speaking to patients has not. It is important to remember that patients are people, and that a unique set of rules does not need to apply when talking to them.

    “Do you have asthma? Are you 18-35 years old?
    You might be eligible for our study”.

    This is the industry standard for attracting patients to a study. This is the equivalent of John Lewis saying “Are you 35-55 years old? Do you need some food and consumer goods? You can get some here”, during their annual Christmas campaign. Although the idea sounds amusing, it loses its charm when you think that it is this sort of activity that is keeping medicines from the people who need them and taking budget away from companies planning to re-invest in their pipeline.

    This poses the question, what are we missing in patient recruitment?

    Patients are being left out; left out of the thoughts of those designing a trial and left out of the discussions during a trial. We need to talk to patients, understand their motivations, their challenges and their requirements. These insights are invaluable, and lead to the creation of patient-centred trials. The current status quo is to create clinical trial protocols based on the insights of industry professionals who often do not have experience as a patient, or healthcare professionals who see each patient once or twice a year. Would you ask a CEO what their staff like to do on the weekends? Although they may be at the top of the game within their respective industries these qualified professionals are often too far removed from the patients to understand their everyday needs.

    Patient experience design

    At Havas Lynx Faze we believe that a clinical trial protocol should never be written without going through a very simple process – mapping out the entire patient journey from start to finish. This includes highlighting all the hoops a patient will need to jump through in order to successfully make their mark on the future of medicine. This mapping considers all of the visits they will attend, the procedures they will endure, and the data capture forms they will need to complete. But it does not stop there. We must also look at the journeys that they will make, the expenses that they will incur, the things that they will need to remember and the impact on the people around them.

    Mapping out the challenges and working out practical solutions which allow a patient to participate in a clinical trial without having to drastically change their daily life is fundamental in the creation of an attractive trial. This can be, and should be, achieved before a protocol has been approved. Working with patients directly, taking their input and feedback, and then feeding it into the design in order to work around the intricate (but often avoidable) challenges. Interrogating the patient journey in this way does not always change a study design; often it simply introduces supportive solutions to prevent the challenges from causing problems and delays. Examples of this include arranging transport for patients through UberHealth or using prepaid study credit cards to cover study expenses so that patients are never out of pocket due to their involvement.

    The key is understanding basic human needs. It is easy to overthink the requirements in a world where sophisticated technology appears to be the answer to all our problems, but in reality, it is the simple things that make a study more convenient, less costly and ultimately less of a burden on a patient’s life. Ultimately improving all of these things will increase the overall probability of success.

    Work with patients, don’t make them work for you

    There is no excuse for a protocol to ever be approved before a patient has been involved. The solution to everything being late is not to rush but to get it right the first time. How often do we hear that taking the time to get something right in the first instance is more efficient and cheaper than rushing and having to fix it in the long term once it has gone wrong? Clinical trials are no different.

    When you consider the enormous costs involved in launching a clinical trial, the idea of not talking to those that are going to be involved is ludicrous. This becomes even more absurd when you think about the different countries and markets in which the trials are conducted, and the huge variation in practices, lifestyle and culture between them. It is fundamental to understand that the way you speak to someone in the US healthcare system can be entirely different to how you would speak to someone who is receiving treatment through the NHS in the UK, not to mention the disparity with the Asian markets or in Russia (where culture, media channels and media consumption vary greatly).

    Often when people talk about involving patients and patient-centric initiatives, what they really mean is they conducted an exercise where patients are sat down and asked what they would like. This usually consists of asking arbitrary questions like “Do you like apps?”, “Do you want a website?” or “Do you read posters?” While these questions are not bad, per se, we are relying on the patients to validate the expected challenges and solutions that were predetermined without their input. As Henry Ford once said, “If I’d have asked people what they wanted then they would have asked for a faster horse”. We know that patients are experts in their own experience, in their challenges and in their motivations, so we need to talk to them in order to understand every parameter and then use them to create tools and solutions tailored to these specific needs.

    How can you be patient-centric without talking to a patient and making them central to your approach? Patients should be involved from the outset, not because it sounds good to say you have spoken to them, but because it is madness not to. Compare this to any consumer market – no consumer brands make their products without thinking of their customers, they design them for their customers and then improve them based on their feedback. Involving customers isn’t seen as a noble thing to do, it just makes business sense. We see no reason why clinical trials should be any different.

    Patient-focused trials

    The magic comes when you unpick the real challenges a patient has, as the solutions to seemingly complex problems are often so logical and simpler than you would imagine. Whether it is help explaining some overly complicated inclusion criteria, making it easy for a patient to get in touch, ensuring someone phones them back, or simply even reminding them of their appointments, often the hard work is done in understanding the real problem (meaning that the associated solutions can be so much simpler).

    This is not to say that there aren’t huge innovations coming. There is an array of novel and innovative options aiming to improve the performance and efficiencies of trials as a whole. Looking to the future there is scope for improved trial success through alternative trial designs such as siteless and adaptive trials. These designs will revolutionise the patient experience, eliminating the logistical challenges that we are seeing today, and achieving this simply by flipping the traditional model on its head and moving into a new age where the patient is the focus.

    But before we jump ahead to these transformative and technical solutions, we must walk before we can run. We must get it right for patients today by making our approach to trial recruitment a little more human in order to benefit the patients of tomorrow.

    For more information about Havas Lynx Faze and how they can help you get patients contact Mark Wilkins @ mark.wilkins@havasfaze.com

    The original article link is below https://deep-dive.pharmaphorum.com/magazine/pharma-research-rare-diseases/havas-lynx-faze-clinical-trials/

Government may drop clinical trials of orphan drugs

Orphan drugs are medicines used in the treatment of rare diseases with less than five lakh patients in a country, but are believed to be expensive.

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Published: 27th February 2019 07:48 AM | Last Updated: 27th February 2019 07:59 AM  | A+ A A-

By ENS Economic Bureau
HYDERABAD: In a significant move, the Central Drugs Standard Control Organisation (CDSCO) has decided to relax rules including a possible waiver of clinical trials for orphan drugs used in the treatment of rare diseases.

Proposed as part of the New Drugs and Clinical Trials Rules-2018, expected to be notified in March, the CDSCO intends to provide a pathway so that even with limited data, orphan drugs can be approved, with or without a trial. In other words, a partial or full waiver of clinical trials is in the offing for specific drugs, provided such tests are conducted elsewhere, particularly in advanced nations like the US or Europe. As per Indian norms, any drug, old or new, has to conduct clinical trials before proceeding with the commercial launch.

“The reason is that the number of patients (with rare diseases) may be 500-1,000 (in India) and with such low numbers, it’ll be difficult for them (clinical trials) to be economically viable. The new rules give regulatory freedom to launch (orphan drugs),” said Chandrashekar Ranga, deputy director-general, CDSCO.

Orphan drugs are medicines used in the treatment of rare diseases with less than five lakh patients in a country, but are believed to be expensive.

Speaking to media on the sidelines of BioAsia 2019, Ranga said the matter of approvals will now be given in a few weeks. Currently, as per the notification, there are no regulatory pathways for approval of orphan drugs. The upcoming rules will offer a constant pathway for approvals and reduce the cost and time to market the drug at affordable prices.

However, pharma companies often complain that seldom was this deadline adhered to, causing severe delays in approvals. From next month, when the rules are notified, companies can proceed with the trials after 45 and 90 days even if they do not get the regulatory nod.

Meanwhile, dismissing reports about the falling number of clinical trials in the country, Ranga said that in reality, trials have been on the rise over the past two to three years.

Boost to drug market

The upcoming rules on clinical trials will offer a constant pathway for approvals and reduce the cost and time to market the drug at affordable prices
The new rules are expected to reduce the time taken for approval of clinical trials to 45 days for domestic companies and 90 days for foreign companies from the current 120 days

2/28/2019According to him, the Indian drugs regulator is also considering waiving off Indian clinical trials of such medicines that are already applied in advanced economies like the US and Europe.
The incoming rules on clinical trials are expected to reduce the time taken for approval of clinical trials to 45 days for domestic companies and 90 days for foreign companies from the current 120 days.

Indian biosimilar market to be worth $40 billion by 2030

The Confederation of Indian Industry (CII) & Sathguru Management Consultants released a position paper on Indian Biosimilars Industry.

“Indian Biosimilars Industry: Roadmap to Actualize Global Leadership” at the 2nd National Biotechnology Conclave 2018 – Driving Inclusive Growth for Indian Bio-economy Vision- 2025 organised by CII in Delhi.

 

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Biosimilars present a very attractive opportunity for Indian pharma companies going beyond small molecule generics ridden with intense competition and price pressures.  Based on analysis of currently approved biologic drugs and global pipeline, in the most optimistic scenario, we estimate that the global biosimilar market will be worth of $240 Billion and Indian biosimilars market will be worth of $40 billion by 2030.  Growth is being driven by increasing market maturity in Europe, more recent forthcoming environment for regulatory approvals in US and high unmet clinical need across RoW markets.

With record number of domestic approvals, active engagement in semi-regulated markets and growing footprint in regulated markets, the Indian biosimilar industry is poised at the cusp of growth. There are more than 70 biosimilars approved in the country today.  The revised Indian guidelines released in 2016 have not only resulted in a rigorous framework for approvals in India, but have also paved the way for leaner bridging effort to enter global markets. At this juncture, it is imperative to nurture the vibrant industry landscape and support the industry in value realization.  Major challenges to combat to secure a sustainable future for Indian-made biosimilars and key recommendations are:

1.    Expanding markets in domestic landscape:  Biosimilars as a means to overcoming affordability barriers is a promise that is a promise that is yet to be translated to reality in several countries including India.  Though substantial level of price erosion has been breached, overall market size is currently unattractive as volume penetration remains significantly low.  For instance, one of the more mature assets in India, Trastuzumab biosimilar had overall sales of about Rs. 286 crores in 2017 translating to less than 10% penetration when assessed on the basis of overall clinical need. Price control has had insignificant impact on expanding access as overall cost of care in indications such as oncology still remains out of reach for majority of the population.

Hence, is it is important to consider other models of aggregated and negotiated buying for public health so that blanket price control can be avoided.  It is critical that affordable biologics are included in national coverage programs to expand access, especially at a time when landmark public health programs such as Ayushman Bharat PMJAY are rolled out.  Finally, as done in certain European countries with high success in biosimilars adoption, a multi-stakeholder approach has to be engaged to drive greater awareness, clinical adoption and market expansion.

2.    Strengthening upstream technology backbone: Sustainable success in the segment will hinge on production economics, especially as prices erode and unit cost and enhanced capex recovery become critical.  There is urgent need to strengthen industry’s backbone in upstream technology development, especially in areas such as high yielding clones and industry-academia platforms should be explored.  For near term impact, globally benchmarkable non-dilutive funding and fiscal incentives for technology acquisition should be considered.

3.    Paving the way for sustainable leadership in global markets: As regulated markets embrace biosimilars and move towards higher market maturity, it is optimal time for the industry to expand thrust on regulated markets, that will constitute majority of the near term global markets by value.  However, given the significant level of binary risk per asset (clinical development investments of about $100 to $150 million per asset), key to defraying risk and expanding investment appetite will lie in more aggressively pursuing strategic partnerships with global as well as domestic counterparts.

The Biocon-Mylan partnership is a great testimony to strategic value of such partnerships and has resulted in Indian developed biosimilars being among the first wave of approved assets in regulated markets.  For RoW markets, the WHO PQ presents great promise as a mechanism that can provide aggregated access to multiple markets that are relatively less mature.  India should encourage fast tracking of the WHO PQ pilot while pursuing regulatory reciprocity in select markets until such aggregated possibility is a tangible reality.  Finally, as done through bodies such as PHARMEXIL in small molecule drugs, a multi-stakeholder approach to enhance global reputation and support global market access will greatly accelerate market expansion in the fragmented RoW markets.

German Regulators: 1,300 Trials Imperiled by “Hard” Brexit

Some 1,300 trials in the UK may have to stop their work if the UK leaves the EU without some kind of new regulatory regime in place, officials at Germany’s Federal Institute for Drugs and Medical Devices say in a new advisory published last week.

German regulators are warning trial sponsors that changing a representative requires “substantial amendments” to a trial’s regulatory regime. Sponsors should file a single amendment focused on their post-Brexit move to the continent and let any other regulatory questions lie for now. It will probably take 20-35 days, on average, to process the new filings, the institute says.

If sponsors are only changing their legal representative, “it is possible to submit this amendment for several clinical trials of one single sponsor by way of a collective amendment,” the advisory says.

Read the regulatory update here: https://bit.ly/2txx3Ri.

Three Reasons Why Mobile Technology Is A Secret Weapon For Patient Engagement And Study Success

The use of mobile technology in clinical trials is becoming increasingly prevalent, most notably in collecting electronic clinical outcome assessment (eCOA) data. Its role, however, in improving patient engagement throughout an entire study is a lesser known area, and an opportunity for differentiation.

 

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Three reasons stand out why mobile technology aids patient engagement so significantly:

Firstly, mobile is everywhere. Everyone knows how integral mobile technology has become to our daily lives, but you may not realize quite how ubiquitous it really is. An average person spends five hours a day on their smartphone, with more than half picking up their smartphone within moments of waking up in the morning. In fact, studies have shown that smartphone users view their device over 500 times per day. Consequently, over a typical 16-hour waking day, mobile technology offers more visibility to patients than any other platform. Tapping into this direct line to patients allows sponsors and CROs to subtly introduce trial activities (e.g. protocol instructions, video guides on home self-injection procedures) seamlessly into the normal flow of patients’ lives. By doing this, sponsors not only help patients understand what they must do in a study, they feel empowered and more invested in its success, which ultimately reduces the incidence of drop out from a study. Being so embedded is an opportunity the industry cannot afford to miss.

Secondly, patients expect technology. Today’s consumers are experienced—and skeptical. No one today would comfortably enter their credit card details into a website that looks like it was built in the 1990s. On the other hand, a modern and sleek website with proper security protocols, forms, and communication, fosters confidence in how personal data is being managed, ultimately making people more likely to proceed. Now imagine instead of making a purchase online, we are asking patients to trust us with their health, using investigational therapies—and then we present to them stacks of paper binders and outdated, clunky software. This cannot inspire confidence in people who expect and demand a clean technology from the most basic of their daily tasks in order to  have trust.

For the pharma industry, developments are in play that can optimize the patient experience beyond simply delivering a range of apps. Leading technology innovators are involved with integrations of several third-party services into one app to create one simple interface for patients (and sites and study teams). Such integrations that meet the needs of patients include ePRO and eCOA data capture, eConsent, patient engagement, patient payments, courier and travel services, and home health nursing. This eliminates an excess of disparate technological interactions with websites, apps, SMS alerts or emails which, despite best intentions, often overwhelm patients rather than reduce the burden of participation. Such seamless interaction, bringing systems to patients that reflect and respect patient-centricity, cannot be underestimated for the value of technology in building confidence in how well studies are managed. Patients have enough to deal with managing their health situation, so minimizing logistical burdens by combining as many solutions as possible into a single app, not only eases the daily burden of participation, it also meets—or exceeds—the expectation of a quality trial.

Finally, mobile technology lets you get creative. Beyond the known benefits of visit reminders, medication compliance alerts, and study notifications, mobile technology can deliver exciting new possibilities. For example, in a recent smoking cessation study, gamification was used to engage with the adolescent patient population. A game involved an adventurer searching for a lost city and the further patients progressed in the study, the more of the story they were able to see with each new chapter revealed at each study visit. A simple yet effective concept, the game proved a powerful engagement tool for patients. This type of content can also be culturally adapted to demonstrate sponsor awareness of subtle cultural difference.

Controlled image and audio capture is another exciting opportunity for researchers, given the high resolution of cameras on most smartphones today. Utilizing such technology can be particularly helpful to assess dermatological conditions such as psoriasis, or to better evaluate reactions to injection-based therapies, such as vaccines. Guides on capturing clinically accessible images for study teams can also be developed as a built-in feature to support patients through such aspects, similarly with audio and capture of precisely calibrated sound recordings to assess conditions such as Parkinson’s or Friedreich’s Ataxia where speech or phonation patterns can be impacted by the disease.

Other possibilities include lay summaries and access to data that don’t risk bias or unblinding, cultural interpretation of images (e.g. using manga cartoons in Japan), trial updates such as final patient completion and regulatory submission, as well as simple expressions of gratitude towards patients. Each of these helps the patient receive not only more value from their involvement in the study, but equally importantly feel more valued by the study.

In summary, much is being done to push the boundaries on what is possible in patient engagement by best-of-breed vendors, going beyond standard industry features. There is a huge opportunity to engage patients in clinical trials through mobile technology and greatly improve study outcomes. With growing evidence that patient engagement is not only synonymous with increased patient retention, but part of the actual conduct of study, is it really something that any sponsor can afford not to make a strategic priority, both to improve the patient experience and their own ROI in clinical trials worldwide?

Patients Are Eager for Trials But Aren’t Getting the Word, Survey Finds

Patients are eager to participate in clinical trials, but they’re having trouble finding the information they need — not just on social media, but from their own doctors, a new survey suggests.

Many respondents said they’d be interested in joining trials — nearly three-fifths said they would be willing to share genetic information or their health records as part of a trial — and one in five said they had proactively asked their doctors about trials, but only 15 percent had ever enrolled in one. Fewer than one in five said their doctor had suggested trials for them.

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The survey was conducted by Inspire, a healthcare-focused social media platform, which surveyed nearly 9,500 of its more than 1 million patient and caregiver members about a range of issues, including awareness of clinical trials.

There also seems to be something of a gender gap in patient readiness for trials. Nearly one-quarter of men said their doctor had suggested a trial for their diseases, but fewer than one-fifth of women received similar advice.

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This is the fourth year Inspire has surveyed patients and caregivers about their treatment. The ongoing lesson, Inspire Research Manger Hannah Watson Eccard says, is that people “are more than recipients of medical care. They are active partners who work with healthcare professionals to make medical decisions and find the best option for them.”

For some patient advocates, the Inspire survey is wake-up call to the trials industry.

“We need to do a better job of initiating conversations about new treatment options — especially clinical trials,” says Dana Dornsife, founder of the Lazarex Cancer Foundation, a California nonprofit that helps patients overcome obstacles to trial enrollment.

Dornsife says she and her colleagues “receive calls daily from cancer patients for whom standard care isn’t working and can’t find the information they need as they investigate clinical trial options.” The group is in the midst of a project it calls IMPACT, a three-year pilot program aimed at improving enrollment in trials.

“The system is fragmented and flawed and leaves many patients out,” she says. “And if a patient does find a clinical trial for which they’re eligible, they’re often blindsided, in many cases, by insurmountable travel costs just to get to the clinical trial. “

Keep Investigator’s Brochure Updates Clear, Concise and Timely, Experts Advise

Investigator’s brochures (IBs) are meant to be a living document, updated at least once a year to keep up with progress and developments in the trial.

But scheduling and drafting updates can prove challenging, says Tiffany Guckin, associate director of regulatory affairs for Invicro, an imaging services and software company.

 

IB means - Investigator's Brochure
IB means – Investigator’s Brochure

The FDA doesn’t stipulate when investigators and sponsors should submit updates to their IRBs and the agency, but it does require sponsors to submit an annual report to the agency within 60 days of the anniversary of the date an IND became active. Scheduling the two updates together in advance is an efficient way to keep reporting requirements on track, she says.

Most of the information in a clinical trial’s annual report is contained — often in greater detail — in the investigator’s brochure, so it makes sense to produce those documents in parallel, Guckin says. She notes, however, that an IB would need to be updated immediately if any crucial safety-related information came to light.

Beyond timing, Guckin says investigators often struggle with keeping IB updates clear and concise, balancing the need to provide all the relevant information with the need to produce a document that an IRB can quickly and easily digest. She suggests including with each IB revision a brief, easy-to-read summary of changes since the last iteration of the brochure. This summary can perform double-duty, she adds, as it can likely be copied and pasted directly into the annual report. She also suggests investigators track changes to make it abundantly clear what has been revised in the new version of the brochure.

Lindsay McNair, chief medical officer for WCG, agrees that transparency is key. “When an IB is revised,” she advises, “provide a clear notation of what data or sections have been changed or updated so the reader knows what to focus on, especially when assessing whether the risk profile has changed.”

As an investigative product moves through development, McNair says, it’s important to revise the sections of the IB containing clinical and non-clinical study results, rather than simply flooding those sections with more data. As the results of more studies become available, investigators can offer a brief summary of past results that have become comparatively less important. For instance, the results of single-dose animal toxicity studies can be minimized once data from chronic-dosing studies are available, she says.

McNair, who has served on a number of IRBs, says sponsors also could be clearer about which possible adverse events would be considered “expected” when drafting the original IB. The regulatory guidance says that serious adverse events will be considered unexpected — thus triggering the filing of an IND safety report to the FDA — if they’re not listed in the IB. “Which sounds straightforward,” she says, but can be “surprisingly hard to determine in practice.” Often, an IB will include safety data on the product related to other conditions, or in combination with other drugs, which may have different expected safety profiles.

Guckin emphasizes the need for coordination and interdepartmental cooperation when preparing annual reports and IB updates. “I suggest that if you have multiple IND applications, you send the due dates out to all the involved departments at the start of the year,” so they can be prepared for regulatory affairs to reach out to them for information.

And when an IB is updated, the newly revised document should be sent to all clinical site investigators involved in the trial.

CDER Drug Trials Report Shows Declining Enrolment

CDER released its fourth annual Drug Trials Snapshots report, which shows a significant drop in the number of participants in pivotal novel drug trials that led to agency approvals.

The overall number of participants dropped by more than half from approximately 106,000 in 2015 to 44,000 in 2018, according to the latest report.

Clinical trial retention

Mark Summers, president, patient engagement, WCG Clinical, noted that clinical studies — especially those for cancer drugs — don’t need as many people as they used to.

“In areas like oncology everything’s moving toward biomarkers. You don’t need as large a population. That could be a factor as well,” Summers said, cautioning that the report doesn’t speak for overall participation in clinical studies because it only covers the enrollment rate of pivotal studies for FDA approved drugs.

Another significant trend was a shift in the trial demographics toward inclusion of more female and black or African American participants. The overall percentage of women increased from 40 percent in 2015 to 56 percent in 2018, while black or African American participation doubled from 5 percent in 2015 to 10 percent in 2018. Hispanics were not recorded in the first two snapshot reports but accounted for 14 percent in 2017 and 2018.

“In recent years, the representation of certain subgroups, such as women and people of racial and ethnic minority groups, has become of greater interest to the general public,” CDER director Janet Woodcock said.

The trend for participants aged 65 and older was generally upwards, with a spike in 2017 to 32 percent, but dropping off last year to just 15 percent representation. The spike may have been the result of several trials that were focused toward geriatric patients, such as Portola Pharmaceuticals’ Bevyxxa (betrixaban) for prevention of thromboembolism.

CDER approved 59 novel drugs last year — either new molecular entities or biologics — ranging from oncology drugs to a preventive migraine drug to treatments for bacterial skin infections, smallpox disease and epilepsy, among others.

The report includes data on the ethnicities, age and sex of participants in each approved novel drug trial. Some trials differed significantly in terms of their demographics. For example, participants in the pivotal trial for Amicus Therapeutics’ Fabrys disease treatment Galafold (migalastat) were nearly all white (97 percent) and more than half were female (64 percent).

On the other hand, the key trial for Theratechnologies’ Trogarzo (ibalizumab-uiyk), an HIV-1 treatment, was more evenly distributed, with whites, black or African Americans, Asians and Hispanics accounting for 55 percent, 33 percent, 10 percent and 25 percent of the participants, respectively, with a 15 percent female makeup.

Read the report here: https://bit.ly/2DXLzHU.

Doing More With Less: The Road To Success For Clinical Trials

Conducting a clinical trial can be both a time consuming and costly affair for pharmaceutical companies. If one is to be successful, they must have their affairs in order, from navigating protocols and regulations to the basic budgeting.

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As the Vice President of Clinical Development Operations at Endo, Rosie Filling’s job is to do just that. Since taking the role in February 2018, she’s been responsible for protocol concepts through corporate social responsibility (CSR), and ultimately through any regulatory submission Endo may be compiling.

A biologist by training, Filling has more than 20 years of experience in the clinical trial industry, exposing her to all functional groups responsible for the execution of a clinical trial.

On behalf of Clinical Research News, Lee Yuan spoke with Filling about the challenges that come with outsourcing clinical trials, what innovations excite her, and what she’s looking forward to as the industry progresses.

Editor’s note: Lee Yuan, a Conference Producer at Cambridge Healthtech Institute, is planning a track dedicated to Managing Outsourced Clinical Trials at the upcoming Summit for Clinical Ops Executives, SCOPE, in Orlando, February 18-21. Filling will be giving a co-presentation at the program. Their conversation has been edited for length and clarity.

Clinical Research News: What do you feel are the biggest challenges in outsourcing clinical trials today, and why?

Rosie Filling: Currently I think the biggest challenge a lot of management is continuing to hear is knowing how to save costs while maintaining quality. It’s definitely that model of “do more with less.” I think as an industry we are constantly being challenged to evaluate cost saving measures, and that leads us to many different questions. Do we change our trial designs? Do we execute our clinical trials differently? Going through the evaluation process is best for the program as well as for your company. Trials are continuing to become more and more expensive. Some of that is certainly related to the requirements that are being enforced by certain regulatory bodies. But as our trials are becoming more and more complex, and when building budgets, it’s very easy for many of us to use previous estimates that we may have from our past experience, etc. Now, we really have to take a step back and look at the many different factors that are driving the additional costs and how those can be offset.

What innovations in the trial space have you most excited, and why?

I think there’s a lot going on out there. One thing in particular, I think, that’s exciting and has been certainly creating some noise over the past several years, is patient engagement. I still think we have come a long way over the past five to seven years in terms of both patient engagement and eCOA [Electronic Clinical Outcome Assessment], but I still think we have a lot of growth potential in that area. I’m excited to see where that goes.

Another one is the oversight of our CROs [Contract Research Organizations] and, in particular, our CRAs [Clinical Research Associates]. Having efficient processes to ensure that we have appropriate oversight, that we’re giving appropriate oversight, and not causing more complexity or harm to the clinical trials is crucial.

In general, what are you excited about in the industry?

Right now I think there needs to be a lot of focus on true partnerships. I think if you ask any CRO or any vendor out there, and even some pharma companies, are you really executing in a true partnership model? I think you’d be surprised with the responses that you would get. I think it’s important, and I like to speak about investing the time and effort in building relationships. Don’t give up too soon. Stick it out with your vendors; try to make it work. I have been fortunate to set up different models at many different pharmaceutical companies, and while my function is to ensure that we execute our portfolio with the greatest quality at the best cost, I have also instilled a culture in these places to ensure that our suppliers are set up to succeed and deliver for our teams as well.

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