State Efforts to Collect Child Outcomes Data for the Part B-619 and Part C Programs Under the Individuals with Disabilities Education Act

The Part B-619 and Part C Programs under the Individuals with Disabilities Education Act (IDEA) provide preschool special education and early intervention services, respectively, to children with disabilities birth through age 5. Recent requirements in IDEA require states to monitor the implementation of these programs through a series of indicators, one of which focuses on the outcomes that infants, toddlers, and preschoolers make as a result of program participation. Known commonly as child outcomes, these data will be used to evaluate the effectiveness of the Part B-619 and Part C programs. The purpose of this investigation was to determine how Part B-619 and Part C programs are collecting high-quality child outcomes data, what barriers these programs are facing in this collection, and how programs are addressing these barriers. Telephone interviews were used to gather descriptive information from a national sample of Part B-619 and Part C coordinators. The clarity and scope of the interview was improved through Dillman's (2000) pretest procedures. The final interview consisted of open-ended questions and was standardized to elicit consistent information from each respondent (Patton, 1990). The results of the study showed that state Part B-619 and Part C programs used similar methods to support the accurate and reliable collection of child outcomes data. Most frequently noted methods included training, a statewide measurement approach, and data review. Despite these methods, 18 types of barriers emerged from collection efforts. Barriers primarily pertained to data quality and the transmission of child outcomes data from local programs to the state. States chose to address barriers through the methods used to support high-quality collection efforts, most notably training and the use of communication and collaboration. Findings suggest that states have established a structure to coordinate the collection of child outcomes data statewide. These efforts focused primarily on improving the quality of these data. Barriers related to the quality of the data emerged despite these efforts, which further indicate the need for ongoing support to sustain high-quality collection efforts. These findings emphasize the importance of training and continuous monitoring to ensure the quality of child outcomes data in statewide collection efforts

Disease pathogenesis, treatment effectiveness, and co-morbid burden among adult patients with primary immune thrombocytopenia (ITP)

Background Primary immune thrombocytopenia (ITP) is an autoimmune disease involving autoantibody-mediated platelet destruction, suboptimal platelet production, and T-cell-mediated platelet lysis. These processes cause a decreased peripheral blood platelet count (< 150 x 109/L), resulting in an increased susceptibility to bleeding events. While the course of primary ITP is often acute (< 6 months) among children, it is generally chronic in adults. Despite a marked increase in epidemiological research over the past decade, unresolved questions remain with regard to disease pathogenesis, treatment effectiveness, and co-morbid burden among adults with primary ITP. Objectives 1. To launch a registry for adults with primary ITP in the United Kingdom (UK) 2. To evaluate associations of candidate single nucleotide polymorphisms (SNPs) with primary ITP 3. To assess the effectiveness of 111In-labelled platelet studies in predicting outcomes from splenectomy 4. To document health-related lifestyle concerns among patients 5. To gauge the effectiveness of Helicobacter pylori (H. pylori) eradication in elevating platelet counts 6. To characterise associations of primary ITP with both arterial and venous thromboembolic events (TEs) Data sources 1. The UK Adult ITP Registry (17 centres, 327 patients) 2. The UK Adult ITP Registry-affiliated, 111In-Labelled Platelet Study Database (117 centres, 256 patients) 3. The Wellcome Trust Case-Control Consortium (WTCCC) 1958 British Birth Cohort (3,000 individuals) 4. The General Practice Research Database (GPRD) (500+ centres, 4 million+ patients) 5. The UK ITP Support Association Health-Related Lifestyle Survey (790 patients) 6. Systematic reviews of published epidemiological studies Methods and Results SNP Study Caucasian patients from the UK Adult ITP Registry were gender-matched (1:3) with healthy controls from the WTCCC 1958 British Birth Cohort. Six functional candidate SNPs in cytokine or cytokine receptor genes were measured in cases and retrieved for controls from the European Genome-phenome Archive. Associations were evaluated using logistic regression models. A statistically significant per allele odds ratio (OR) of 1.34 (95% confidence interval [CI], 1.03- 1.75) was observed for TNFA -308 g>a, implicating increased disease susceptibility among carriers of the rare allele. 111In Study The effectiveness of autologous 111In-labelled platelet sequestration studies in predicting short (1-3 months), medium (6-12 months), and long-term (last follow-up) complete response (CR; count < 100 x 109/L) to splenectomy in patients with primary ITP was evaluated using multivariable logistic regression models. Significant adjusted (gender, age at splenectomy, and mean platelet lifespan) ORs were uncovered for short (7.47 [95% CI, 1.89-29.43], medium (4.85 [95% CI, 1.04-22.54]) and long-term (5.39 [95% CI, 1.34-21.65]) CR in patients with purely or predominantly splenic versus mixed or hepatic splenic platelet sequestration, highlighting the utility of platelet sequestration studies as an adjunct predictive tool prior to splenectomy. H. pylori Eradication Study A systematic literature review was conducted of studies evaluating the effects of H. pylori eradication on platelet count in patients with primary ITP. Twenty-five studies were identified, encompassing 696 evaluable patients. The weighted mean complete response (count > 100 x 109/L) and overall response (platelet count > 30 x 109/L) were 42.7% (95% CI, 31.8%-53.9%) and 50.3% (95% CI, 41.6%-59.0%), respectively. Observed responses were higher in countries with a higher prevalence of H. pylori infection and in patients with milder thrombocytopenia. These findings support the benefits of H. pylori detection and eradication in patients with primary ITP. Health-Related Lifestyle Study A 43-question, closed-field questionnaire was used to identify health-related lifestyle concerns among patient members of the UK Adult ITP Support Association. Completed surveys were returned by 790 (44.7%) members, with nearly one-third of adults reporting having an elective surgery delayed due to a low platelet count and experiencing difficulty in obtaining travel insurance. Notably, 12.5% of all patients reported “always” or “often” missing work or school due to fatigue. These results highlight several promising avenues for future health-related quality of life (HRQoL) research. Thromboembolic Event Studies Using the GPRD, 1,070 adults with primary ITP were matched (1:4) with 4,280 ITP-free controls by age, gender, practice, and observation time. Comparative risks of TEs were assessed using Cox proportional hazards models. Over a median time of 47.6 months (range: 3.0-192.5 months), adjusted hazard ratios of 1.58 (95% CI, 1.01-2.48) and 1.37 (95% CI, 0.94-2.00) were found for venous and arterial TEs, respectively. A similar investigation was conducted comparing age and sex-stratified incidence rates of TEs among patients in the UK Adult ITP Registry with population-based estimates for the general adult population, yielding significant standardised rate ratios of 2.43 (95% CI, 1.01-5.83) and 2.45 (95% CI, 1.48-4.06) for venous and arterial TEs, respectively. These results collectively highlight an increased risk of venous and arterial TEs in adults with primary ITP. Conclusions Primary ITP is a pro-inflammatory (i.e., TH-1-mediated), pro-thrombotic disease in adults. Available evidence supports testing for and eradication of H. pylori infection in patients with primary ITP and the utility of autologous 111Inlabelled platelet sequestration studies in identifying patients likely to respond to splenectomy. The development of a prospective, international registry will help assemble the sample sizes needed for promising further research, namely genome-wide disease association studies and investigations into the effects of the eradication of strain-specific H. pylori infection on platelet count, the precise relative risk of non-response to splenectomy in patients with mixed or hepatic platelet sequestration, and the associations of TEs with primary ITP in antiphospholipid antibody-negative

Transformative Models to Promote Prescription Drug Innovation and Access: A Landscape Analysis

The patent-based pharmaceutical innovation system in the US does not incentivize the development of drugs with the greatest impact on patient or public health. It has also led to drug prices that patients and health care systems cannot afford. Three alternate approaches to promoting pharmaceutical innovation have been proposed to address these shortcomings. Delinkage models involve payments for drug innovation based on public health value rather than on a per-use basis. Public manufacturing models call upon governments and nonprofit organizations to lead drug discovery, development, and production. Public-private partnership models entail publicly-funded organizations working closely with for-profit partners on drug development and price-setting. Each model exhibits promise in promoting prescription drug innovation and access. This paper reviews these transformative models in detail, examining their key characteristics, advantages, and limitations

Battling Over Patents: The Impact of Oil States on the Generic Drug Industry

In the 2018 case of Oil States Energy Services v. Greene’s Energy Group, the U.S. Supreme Court upheld the constitutionality of inter partes review, a non-judicial proceeding for challenging patents that was created by Congress as part of the 2011 Leahy-Smith America Invents Act. By establishing inter partes review, Congress hoped to rebalance patent policy to make it faster and less costly to invalidate erroneously granted patents in all fields of technology. In the pharmaceutical industry, generic drug companies have embraced inter partes review, filing hundreds of challenges in the first five years after its creation, with moderate success. Biologics, which make up a growing class of pharmaceutical products, are sometimes covered by dozens or scores of patents. As more of these complex therapeutics are developed and approved, inter partes review is expected to play an increasingly important role

Regulatory Solutions to the Problem of High Generic Drug Costs

Recent reports have highlighted dramatic price increases for several older generic drugs, including a number of essential products used to treat deadly infectious diseases. Although most of these medicines have been widely available at reasonable prices for decades, some manufacturers have seized on unique features of the pharmaceutical marketplace to seek substantial profits. In this Perspective, we examine limitations in current price regulation among public and private payors and consider several reforms that could address the problem of expensive generic drugs through improved competition

COVID-19 vaccine boosters for all adults: An optimal U.s. approach?

By 20 October 2021, the U.S. Food and Drug Administration (FDA) had amended its Emergency Use Authorizations for immunocompetent adults who previously received the Pfizer-BioNTech, Moderna, or Johnson & Johnson COVID-19 vaccines. For the 2-dose Pfizer-BioNTech and Moderna vaccines, the FDA permitted a single booster dose for adults aged 65 years or older and adults aged 18 to 64 years at high-risk for severe COVID-19 or at high risk for occupational or institutional COVID-19 exposure. For the single-dose Johnson & Johnson vaccine, the FDA permitted a single booster dose for all adults aged 18 or older. These eligibility schemes were endorsed by the Centers for Disease Control and Prevention shortly after FDA approval

Comparing generic drug markets in Europe and the United States: prices, volumes, and spending

Our study indicates that there are opportunities for cost savings in generic drug markets in Europe and the United States. Regulators should make it easier for generic drugs to reach the market. Regulators and payers should apply measures to stimulate price competition among generic drugmakers and to increase generic drug use. To meaningfully evaluate policy options, it is important to analyze historical context and understand why similar initiatives failed previously. Context: Rising drug prices are putting pressure on health care budgets. Policymakers are assessing how they can save money through generic drugs. Methods: We compared generic drug prices and market shares in 13 European countries, using data from 2013, to assess the amount of variation that exists between countries. To place these results in context, we reviewed evidence from recent studies on the prices and use of generics in Europe and the United States. We also surveyed peer‐reviewed studies, gray literature, and books published since 2000 to (1) outline existing generic drug policies in European countries and the United States; (2) identify ways to increase generic drug use and to promote price competition among generic drug companies; and (3) explore barriers to implementing reform of generic drug policies, using a historical example from the United States as a case study. Findings: The prices and market shares of generics vary widely across Europe. For example, prices charged by manufacturers in Switzerland are, on average, more than 2.5 times those in Germany and more than 6 times those in the United Kingdom, based on the results of a commonly used price index. The proportion of prescriptions filled with generics ranges from 17% in Switzerland to 83% in the United Kingdom. By comparison, the United States has historically had low generic drug prices and high rates of generic drug use (84% in 2013), but has in recent years experienced sharp price increases for some off‐patent products. There are policy solutions to address issues in Europe and the United States, such as streamlining the generic drug approval process and requiring generic prescribing and substitution where such policies are not yet in place. The history of substitution laws in the United States provides insights into the economic, political, and cultural issues influencing the adoption of generic drug policies. Conclusions: Governments should apply coherent supply‐ and demand‐side policies in generic drug markets. An immediate priority is to convince more physicians, pharmacists, and patients that generic drugs are bioequivalent to branded products. Special‐interest groups continue to obstruct reform in Europe and the United States

Patient-reported treatment burden of chronic immune thrombocytopenia therapies

<p>Abstract</p> <p>Background</p> <p>Chronic immune thrombocytopenia (ITP) is a debilitating autoimmune disorder that causes a reduction in blood platelets and increased risk of bleeding. ITP is currently managed with various pharmacologic therapies and splenectomy.</p> <p>This study was conducted to assess patient perceived and reported treatment side effects, as well as the perceived burden or bother, and need to reduce or stop treatment, associated with these side effects among adult patients with chronic ITP.</p> <p>Methods</p> <p>A Web-enabled survey was administered to members of a US-based ITP patient support group. Patients reported demographic and clinical characteristics, ITP treatments' side effects for treatments received since diagnosed, level of bother (or distress), and need to reduce or stop treatment, associated with side effects. Current and past exposure was assessed for five specific treatment types: corticosteroids (CS), intravenous immunoglobulin (IVIg), anti-D immunoglobulin (anti-D), rituximab (RT), and splenectomy (SPL), as well as for other patient-referenced therapies (captured as "other").</p> <p>Results</p> <p>The survey was completed by 589 patients; 78% female, 89% white, mean age 48 years (SD = 14.71), and 68% reported a typical low platelet count of < 50,000/μL. Current or past treatment with CS was reported by 92% (n = 542) of patients, 56% (n = 322) for IVIg, 36% (n = 209) for anti-D, 36% (n = 213) for RT, and 39% (n = 227) for SPL. A substantial proportion of CS-treated patients reported side effects (98%, <it>P </it>< 0.05), were highly bothered by their side effects (53.1%, <it>P </it>< 0.05), and reported the need to stop or reduce treatment due to side effects (37.8%, <it>P </it>< 0.05). Among patients reporting side effects of treatment, significant associations were noted for the number of side effects, aggregate bother of reported side effects, and the need to stop or reduce treatment (all <it>P </it>< 0.05).</p> <p>Conclusions</p> <p>Current ITP treatments, particularly corticosteroids, are associated with multiple bothersome side effects that may lead to patients stopping or reducing therapy. Open, informed and complete communication between clinician and patient regarding both the benefits and the side effects of ITP treatment may better prepare patients for their prescribed regimens.</p

Influence of drug safety advisories on drug utilisation: an international interrupted time series and meta-analysis

OBJECTIVE: To evaluate the association between regulatory drug safety advisories and changes in drug utilisation. DESIGN: We conducted controlled, interrupted times series analyses with administrative prescription claims data to estimate changes in drug utilisation following advisories. We used random-effects meta-analysis with inverse-variance weighting to estimate the average postadvisory change in drug utilisation across advisories. STUDY POPULATION: We included advisories issued in Canada, Denmark, the UK and the USA during 2009-2015, mainly concerning drugs in common use in primary care. We excluded advisories related to over-the-counter drugs, drug-drug interactions, vaccines, drugs used primarily in hospital and advisories with co-interventions within ±6 months. MAIN OUTCOME MEASURES: Change in drug utilisation, defined as actual versus predicted percentage change in the number of prescriptions (for advisories without dose-related advice), or in the number of defined daily doses (for dose-related advisories), per 100 000 population. RESULTS: Among advisories without dose-related advice (n=20), the average change in drug utilisation was -5.83% (95% CI -10.93 to -0.73; p=0.03). Advisories with dose-related advice (n=4) were not associated with a statistically significant change in drug utilisation (-1.93%; 95% CI -17.10 to 13.23; p=0.80). In a post hoc subgroup analysis of advisories without dose-related advice, we observed no statistically significant difference between the change in drug utilisation following advisories with explicit prescribing advice, such as a recommendation to consider the risk of a drug when prescribing, and the change in drug utilisation following advisories without such advice. CONCLUSIONS: Among safety advisories issued on a wide range of drugs during 2009-2015 in 4 countries (Canada, Denmark, the UK and the USA), the association of advisories with changes in drug utilisation was variable, and the average association was modest