Focusing on the Patient in Drug Development

A major component in the success of a therapeutic product is whether the patient actually takes the medicine—an aspect that is particularly pressing in patients with chronic and long-term illnesses. It has been well reported that non-adherence to medication regimens is significantly burdensome on healthcare systems and, ultimately, countries’ economies.

One way to improve patient adherence to medication is by ensuring the dosage form is patient-centric or user-friendly. “Patient centricity is gaining more focus and importance in drug development,” confirms Anil Kane, PhD, MBA, senior director, global technical scientific affairs, pharma services, Thermo Fisher Scientific. “Problems related to polypharmacy and patient non-adherence is critical to healthcare. Patient-centric pharmaceutical drug product is increasingly important to gain patient adherence in pediatrics, adults, and geriatric patients.”

Patient-focused thinking

Ideally, a dosage form should incorporate multiple facets to be patient-friendly, notes Anita Solanki, lead—White Papers, Formulation R&D (Pharma and Nutra), ACG Group. “The ideal, patient-friendly, dosage form is one that is easier to swallow, non-invasive, with no taste or odor, has quicker brand recall and is easier to handle and carry, with suitable packaging materials,” she says. “So, throughout the process—right from dosage form selection, through to excipient selection, appearance, packaging, and material selection—every step needs to be considered with the patient in mind.”

Cornell Stamoran, PhD, vice president of Strategy and co-chair of the Catalent Applied Drug Delivery Institute, also agrees that key decisions made during drug development, such as molecule design, formulation, dose form, device selection, and packaging, can have an impact on how successful the therapy will be for patients. Stamoran confirms that through discussions with physicians and payors that were performed at Catalent’s Applied Drug Delivery Institute there is a belief “that those companies that more effectively incorporate patient-focused thinking into drug product design, and provide evidence from the resulting clinical trials, will increasingly have a significant advantage in the market, and ultimately this will benefit patients.”

In terms of dosage form, Thomas B. “Brad” Gold, PhD, vice president, Pharmaceutical Development, Metrics, specifies that oral solid dosage (OSD) forms are preferred by both patients and payers. As such, OSD forms are dominant within drug development and payer schedules. However, despite the fact that OSD forms are preferred there are still issues with compliance, he continues.

“Depending on the formulation and treatment course, orally administering a drug to patients can become problematic and contribute to poor health outcomes,” Gold adds. “Individual dose size, as well as dose frequency all have an impact on the patient centricity of an oral drug. Fortunately, formulation and manufacturing techniques like amorphous solid dispersions, multiple unit particle systems, mini-tablets and other systems are helping to make OSDs more patient centric than ever.”

Kane concurs that OSD forms continue to be market-leading, not least because of the preference by patients, but also due to cost savings and efficiencies afforded by the dosage form in the small molecule market. “Strategies for improving adherence need to consider the characteristics of the individual therapeutic regimens according to the needs of the patients. In particular, geriatric, and pediatric populations have special needs/preferences that should be considered when designing drug products,” Kane continues. “Introducing patient centricity into drug product design offers the opportunity to meet the needs and preferences of patients.”

Providing some examples, Kane explains that through employing taste-masking to create palatable dosage forms will allow for improved patient compliance in geriatric patients with dysphagia, which is a difficulty in swallowing. Other considerations to improve patient centricity of a dosage form for the pediatric segment of the population include the size of the dose, ease of administration, volume of fluid required to be administered, and dosing frequency or frequency of administration, he confirms.

“Innovative ideas and formulation strategies including fixed-dose combinations, modified release dosage forms, orodispersible formulations, multi-particulates, and minitablets in capsules or tablets will support patient-centric drug products,” Kane states.

For Ali Rajabi-Siahboomi, vice president—chief innovation officer, Colorcon, another factor to consider is the potential differentiation between tablets, which is being sought by regulators and marketers. “Unique designs are important because they help make medicines memorable to patients, caregivers, and pharmacists, and may also make a tablet easier to take. From an anti-counterfeiting perspective, unique designs also make products more difficult to copy,” he says. “There are production benefits as well. A unique design offers differentiation on the packaging line, making it easier to visually spot a mix-up and prevent product recalls before it even happens.”

Furthermore, Rajabi-Siahboomi reports that the FDA has also acknowledged the advantages of better design, a fact that is apparent through its guidance documents—Safety Considerations for Product Design to Minimize Medication Errors and Size, Shape, and Other Physical Attributes of Generic Tablets and Capsules (1,2). “In essence, too many products and dosages look the same and there’s a risk dispensers may distribute the wrong product, or people taking more than one medicine (increasingly common in our aging population) will get their pills mixed up,” he explains. “Regulators now expect companies to consider aspects such as size, shape, color, and differentiation between dosage strengths before marketing. Similar-looking dosages, especially plain white tablets, may not get through the regulatory process. Your tablet needs to be different.”

Adding to these thoughts, Hanns-Christian Mahler, CEO of ten23 health agrees that regulatory focus on patient-centricity will further increase, albeit to ensure the therapeutic product is being used adequately and to understand potential liabilities when products are used incorrectly. “Additionally, connectivity (to e.g., smartphones) and sustainability are in my view key topics in relation to patient-centricity for drug/device combination products,” he says.

In concurrence, Rajabi-Siabhoomi also points out that the connection with smartphones or smart medicines can provide a significant opportunity with on-dose authentication technology. “Imagine a patient scanning their pill with a smartphone that not only confirms the identity of the medicine but also connects the patient directly to support materials, such as the product leaflet, information that explains the benefits of the medicine, the importance of adherence, what to do if there are side effects, and even the ability to opt into reminders to take the medicine at the right time,” he states.

Final thoughts

As was specified earlier, poor adherence to a therapeutic regimen not only impacts the effectiveness of a drug but also has a financial impact on healthcare systems, economies, and patients. “Prescription drug medication adherence is a growing concern for public health, and poor adherence to therapy has been associated with poor health outcomes and higher costs for patients,” notes Kane.

Solanki believes that the influence of patient-centricity on the pharma industry will continue to grow and is leading to a big shift in priorities during the development of most dosage forms. “The patient centricity trend is fueling the focus on developing patient-friendly dosage forms. This trend has to be accomplished whilst maintaining optimum therapeutic efficacy and safety,” she says. “Concept transformation has led to the integration of patient-centricity into all aspects of R&D—including formulation design and dosage form development.”

However, there is more work to be done, according to Stamoran. “Through Catalent’s Applied Drug Delivery Institute, we have been advocating for an intentional, patient-focused approach to these areas for the last 10 years. There are important signs of progress, but there remains substantial opportunity to improve patient usability of both new and existing treatments,” he states.

Mahler hopes that patient-centricity and product usability remain priorities for the pharma industry and even that they grow in relevance. “After all, patient-centric drug development all about the patient and to enable them to have an effective and safe treatment,” he concludes.

References

1. FDA, Size, Shape, and Other Physical Attributes of Generic Tablets and Capsules, Guidance for Industry, fda.gov (June 2015).
2. FDA, Safety Considerations for Product Design to Minimize Medication Errors, Guidance for Industry, fda.gov (April 2016).

About the author

Felicity Thomas is the European editor for Pharmaceutical Technology Group.

Article Details

Pharmaceutical Technology
Vol. 46, No. 6
June 2022
Pages: 27–28

Citation

When referring to this article, please cite it as F. Thomas, “Focusing on the Patient in Drug Development,” Pharmaceutical Technology 46 (6) 2022.